Ireland’s Cell and Gene Therapy Map: Illuminating the Island’s Growing Ecosystem
Introduction
Last week’s launch of the Irish Society for Gene and Cell Therapy (ISGCT) website marked a milestone in building visibility for advanced therapies across the island of Ireland. It comes at a time when Ireland’s ecosystem - spanning research, clinical delivery, and manufacturing - is accelerating rapidly.
At Lonrú Consulting, we see this as the right moment to shine a light on the opportunities Ireland presents for enabling technology providers. To make this landscape more tangible, we’ve built a new resource: Ireland’s Cell and Gene Therapy Map - an interactive tool that brings the island’s ecosystem into focus.
Ireland’s Cell and Gene Therapy Map
Ireland’s Cell and Gene Therapy Map is a first-of-its-kind interactive ecosystem map showcasing:
Research centers advancing discovery and translational science.
Clinical sites delivering stem cell transplants and CAR-T therapies.
Manufacturing facilities producing GMP-grade advanced therapy products.
Key players and consortia driving collaboration and innovation.
Explore the interactive map below.
The tool enables users to click through academic hubs, GMP facilities, consortia, and companies, discovering why each node matters for tool providers. Filters make it easy to focus on capabilities such as GMP, analytics, non-viral delivery, or clinical trial readiness.
Why It Matters for Enabling Technology Providers
The map demonstrates that Ireland offers more than clinical delivery or traditional biopharma manufacturing. From Takeda’s stem cell manufacturing facility in Dublin, to CCMI’s GMP-licensed ATMP site in Galway, to St. James’s CAR-T program, and HiTech Health’s new CDMO capacity, Ireland is becoming a proving ground for enabling technologies.
For providers of analytics, automation, delivery platforms, and quality systems, Ireland is a place to:
Pilot tools with translational centers and GMP labs.
Validate technologies in live manufacturing and clinical environments.
Partner with consortia like ISGCT and Can-Vas to link into funding and clinical trials.
Lonrú’s Perspective
With Ireland’s Cell and Gene Therapy Map, we’ve built a resource that not only catalogs facilities, but highlights why each node matters for tool and platform providers.
This aligns with our ethos: Navigating complexity, illuminating innovation. By combining insights, interactivity, and data-driven clarity, we aim to help enabling technology providers see Ireland’s position in the global CGT landscape.
Looking Ahead
With the relaunch of ISGCT and the launch of our interactive map tool providers can see that Ireland is ready to step forward as a connected hub for cell and gene therapy.
For enabling technology providers, the opportunity is clear: engage early, validate locally, and scale globally.
CRISPR Clinical Trials: Global Shifts and New Directions
At Lonrú Consulting, we believe clarity comes from illumination. That’s why we’re excited to introduce our newest interactive tool: the VantagePoint™ CRISPR Clinical Trials Dashboard. Built by Lonrú Studios™, our custom analytics and visualization arm, the dashboard transforms raw data into insight - helping our clients see where the field is heading, and why it matters for enabling technologies.
What the data shows
By exploring the latest dataset of CRISPR-focused clinical trials, we can see both where research is accelerating globally and the new directions shaping the next wave of gene editing:
Therapeutic momentum is shifting
The majority of ongoing CRISPR trials are concentrated in oncology and hematology, but the pipeline is diversifying. We’re now seeing increasing activity in metabolic indications, as well as ophthalmology and rare genetic disorders.Ex vivo editing remains dominant, but boundaries are moving
Most active programs continue to leverage ex vivo approaches (particularly in T-cell and hematopoietic stem cell therapies). However, a growing number of early-phase trials are testing direct delivery platforms - signalling opportunity for providers of delivery vectors, lipid nanoparticles, and targeting systems.Global expansion is accelerating
Many assume the US is at the heart of CRISPR clinical development, but the data tells a different story. China now leads in sheer volume of CRISPR trials, outpacing the US and Europe. It’s no coincidence that some of the biggest bio-pharma partnerships announced this year have been sourced in China. For enabling technology providers, this underlines the strategic importance of engaging with Chinese innovators and preparing for globally distributed development pathways.Emerging trends in editing technologies
The field is no longer just underpinned by CRISPR-Cas9. We see exploratory trials beginning to reference base editing and prime editing, pointing to a diversification of platforms that will demand new supporting tools, analytics, and quality frameworks.
What’s new in the past month?
The trials updated in the past month offer a good snapshot of these new directions; from late-phase genetic programs to unconventional nutrition-linked studies:
Vertex Pharmaceuticals has registered a new Phase 3 program (NCT05951205) focused on a single-dose intervention in genetic disease. This underscores how established leaders are moving rapidly toward pivotal studies, reflecting both confidence in the science and a push for near-term approvals.
Haikou Affiliated Hospital of Central South University (China) has initiated an in vivo CRISPR trial targeting airway basal stem cells (NCT07018804). While still small in scale, it highlights China’s continued leadership in first-in-human in vivo approaches.
Quadram Institute Bioscience (UK) has launched a non-classical intervention trial (NCT07142759) exploring Vitamin D biofortified foods in a CRISPR trial context. While not a traditional therapeutic editing program per se, it signals a growing interest in how adjacent lifestyle and nutritional factors intersect with gene-editing research. This broadening scope is worth watching as it could open new categories of enabling technologies and cross-disciplinary collaborations.
Why this matters:
Together, these updates show that the CRISPR pipeline is diversifying in strategy. From pivotal late-phase trials to unconventional nutrition-linked studies, the field is probing both mainstream and novel directions. For enabling technology providers, this means opportunities will span beyond traditional vectors and QC tools, into areas where gene editing intersects with adjacent health domains.
Why this matters for enabling technology providers
These patterns carry direct implications for Lonrú’s clients:
Manufacturing & process innovators should anticipate increasing demand for scalable ex vivo workflows alongside flexible platforms capable of supporting in vivo expansion.
Analytics & QC tool developers have a growing opportunity to differentiate as editing modalities diversify.
Delivery technology providers are moving into sharper focus as sponsors test in vivo approaches.
In short: as CRISPR clinical trials broaden, the enabling ecosystem becomes the key to acceleration.
A tool to guide strategy
The new dashboard offers a clear vantage point. By surfacing shifts in indications, geographies, and editing approaches, it helps stakeholders identify where their solutions align with unmet needs and where strategic investment is headed.
At Lonrú Consulting, our mission is to empower the technologies behind CGT breakthroughs. Through VantagePoint™ Insights and custom dashboards from Lonrú Studios™, we illuminate complexity and help our clients act with confidence.
👉 Explore the dashboard for yourself below, or click here to view our full suite of interactive dashboards: VantagePoint™ Interactive Dashboards.
The Arc of Innovation in Cell & Gene Therapy: A Decade of Milestones and Signals Ahead
For much of the 2000s, cell and gene therapy was synonymous with viral delivery. AAV, lentiviral, and retroviral platforms defined the way the field engineered cells, delivered payloads, and approached manufacturing. These systems enabled the first wave of approvals and proved that genetic medicine could be real, but they also brought with them limitations—complex scale-up, safety concerns, and payload constraints.
The last decade has marked a clear shift. New modalities—non-viral, protein-only, mRNA-only, and in vivo approaches—have begun to reshape expectations for what therapies can look like and how they can be made. That is where our lens begins: in 2016, with the infusion of CRISPR-edited T cells. Not because it was the first milestone, but because it marked the start of a new era in which the dominance of viral vectors began to give way to a more diverse landscape.
Looking back, we can trace a clear progression:
2017 cemented viral validation with Luxturna and the first CAR-T approvals.
2020 redefined delivery as mRNA vaccines demonstrated at global scale that non-viral modalities could be safe, effective, and manufacturable.
2023 brought the first durable in vivo base editing data, showing that protein-only systems could drive meaningful clinical outcomes.
2024–2025 is set to be remembered for strategic bets: Interius entering the clinic with in vivo CAR-T, and AstraZeneca acquiring EsoBiotec to gain a foothold in reprogramming.
Each milestone tells its own story—but together they map a larger narrative: viral dominance giving way to a more diverse delivery landscape, and a steady march from ex vivo manipulation toward in vivo reprogramming.
Why this matters now
For enabling technology providers—the companies building the assays, platforms, and processes that make therapies possible—these shifts are not abstract. They determine where demand will concentrate, which capabilities will command premiums, and what regulators will expect as standards. The move toward protein-only, RNA-only, and in vivo modalities means that tools once designed for viral vectors are no longer sufficient.
Signals ahead
The next five years will test whether early promises translate into durable platforms.
By 2027, pivotal RNP-only programs in blood disorders could establish protein-only delivery as a credible alternative to viral editing.
By 2029, the first approvals of mRNA-only therapies in oncology and autoimmune disease, alongside in vivo CAR-T, would confirm that the delivery ecosystem has truly diversified.
Explore the narrative
We created The Arc of Innovation Timeline as a companion to this story - an interactive way to walk through the milestones, compare modalities, and see how today’s shifts set the stage for tomorrow’s breakthroughs. It reflects the philosophy behind our VantagePoint™ services: transforming complex data into clear, engaging insights that don’t just inform, but illuminate. For us, making data interactive is about helping enabling technology providers see the bigger picture and act with confidence.
Explore The Arc of Innovation Timeline below.
A Whistle-Stop Tour Around the Cell And Gene Therapy World
When you step back and take a truly global VantagePoint™, it’s remarkable how diversely and dynamically the cell and gene therapy (CGT) ecosystem is evolving. Different regions are leaning into their strengths: infrastructure, regulatory experimentation, workforce training, or cost disruption. For those building the enabling technologies behind CGT; the reagents, digital tools, automation platforms, and analytics, this creates a mosaic of opportunity.
At Lonrú Consulting, we wanted to give you a snapshot tour; east to west of the latest regional dynamics shaping CGT, and why they matter for tool providers. To make this more dynamic, we’ve built an interactive globe that lets you spin from Singapore to São Paulo, click into a region, and see the highlights in context. Scroll down to explore the journey and engage with the map.
Around the World: Regional Cell & Gene Therapy Highlights
How to explore
- Drag to rotate. Wheel or pinch to zoom.
- Click a pin to open details. Popups appear above pins.
- Use ⟵ ⟶ to step through regions. Auto View resets to AU/NZ.
Asia-Pacific
Australia
Australia is building sovereign CGT manufacturing strength. Facilities in Melbourne and Brisbane are focusing on viral vector supply and GMP cell manufacturing. Vendors in automated bioprocessing, digital quality control, and real-time analytics will find strong demand. Workforce training initiatives reinforce adoption of closed-system and scalable platforms.
New Zealand
New Zealand’s CGT presence is smaller but growing through clinical trial networks and government-backed biotech initiatives. Tool providers with portable manufacturing and data-light QC solutions can position early, especially as NZ leverages collaboration with Australian hubs.
Singapore
Singapore has established itself as a Southeast Asian CGT hub. Investments in ACTRIS, Biopolis, and REMEDIS are structured to help projects move smoothly from pilot scale to GMP. This creates appetite for MES systems, electronic batch records, and analytics tools that reduce tech transfer friction.
China
China is home to a rapidly expanding CGT pipeline across oncology, ocular, and metabolic programs. NMPA approvals are rising, which drives demand for genome editing platforms, delivery systems, and CMC characterization tools.
Japan
Japan’s conditional approval pathway accelerates regenerative therapies but requires real-world evidence. Vendors with decentralized manufacturing solutions, potency assays, and hospital-ready quality systems are well positioned.
South Korea
South Korea is scaling with policy support and new facilities. Growth projections exceed 15 percent annually, opening opportunities for automated expansion tools, comparability assays, and cold-chain logistics solutions.
India
India’s NexCAR19, the first indigenous CAR-T, showcases a cost-disruptive model. Hospitals require affordable closed systems, in-country QC, and vendor platforms that allow efficient tech transfer. This makes India a value-driven market for enabling technologies.
Thailand
Thailand’s conditional approval framework for ATMPs signals regulatory openness. Vendors offering decentralized manufacturing tools and pharmacovigilance-friendly platforms will find traction.
Taiwan
Taiwan offers first-in-human approvals in as little as 30 days and has deep CAR-T experience. This is an ideal environment for early evaluation of compact processing systems, integrated data packages, and analytics platforms.
Middle East
Israel
Israel is a powerhouse for CGT startups and rapid clinical iteration. Compact bioprocess hardware, digital analytics, and novel delivery tools can find quick uptake and serve as proving grounds for global scale.
United Arab Emirates
The UAE’s Abu Dhabi Stem Cells Center (ADSCC) is running trials in cell therapies, while G42 Healthcare invests in AI and advanced clinical research. This signals demand for analytics, automation, and quality systems beyond traditional cleanroom builds. Vendors offering QC labs, turnkey closed systems, and digital solutions are well positioned.
Saudi Arabia
King Faisal Specialist Hospital in Riyadh is leading CAR-T clinical trial development. Vision 2030 prioritises biotech and regenerative medicine, backed by sovereign wealth funding. Demand signals include automated CAR-T expansion platforms, digital QC, and workforce training aligned with rapid scaling ambitions.
Qatar
Sidra Medicine in Doha is developing regenerative medicine programs and rare disease genomics. Government R&D investment in precision medicine is strong, with opportunities for vendors in genome editing tools, sequencing analytics, and rare disease-focused QC solutions.
Africa
Nigeria
Nigeria has the world’s largest sickle cell patient population. With the global approval of gene-editing therapies like CASGEVY®, attention is turning to Nigeria as a market for future access. The challenge is to translate breakthrough therapies into resource-limited health systems. For enabling technology providers, this signals urgent demand for simplified, portable, and low-cost platforms that could make advanced therapies deliverable at scale.
South Africa
South Africa has the continent’s strongest clinical trial infrastructure, with hubs in Johannesburg and Cape Town and facilities linked to Wits and Stellenbosch universities. Opportunities include viral vector analytics, workforce training platforms, and in-process QC tools for hematology and oncology trials.
Egypt
Egypt has invested in stem cell and gene therapy R&D with government support for ATMP clinical frameworks. Partnerships with European bodies are growing. Vendors in process development, QC, and modular cleanrooms can find early adoption pathways.
Europe
United Kingdom
The UK is leading with new frameworks for point-of-care and modular manufacturing. Compact cleanroom pods, digital QMS, and automation tools that integrate directly into hospitals are in high demand.
Ireland
Ireland is scaling through NIBRT’s new ATMP plant and public funding for newborn cell therapy. Vendors in robotics, analytics, and workforce training can connect directly into this ecosystem.
Germany
Germany’s Berlin and Munich clusters are central to European CGT manufacturing. Robotics-ready platforms, viral vector scale-out technologies, and MES solutions are priorities.
France
France is strengthening its hospital-linked GMP clusters in Paris and Lyon. Vendors with QC analytics and compact processing hardware will find strong pull-through here.
Spain
Catalonia and Andalusia are expanding infrastructure and clinical trial capacity. This is creating demand for comparability analytics, digital QC, and trial-ready processing platforms.
Sweden
Sweden’s Karolinska Institutet and Testa Center are key innovation hubs. The country is a launchpad for automation and closed-system process technologies.
Norway
The Oslo Cancer Cluster is becoming a trial hub for CGT. Vendors supporting trial logistics, cold-chain integration, and biobank systems will find an active customer base.
Denmark
Copenhagen’s life science cluster is investing heavily in ATMP readiness. Robotics, automation, and advanced process control tools are a natural fit.
Italy
Italy’s CGT activity is clustered in Milan and Naples. Consortia-driven programs need potency assays, QC analytics, and hospital-integrated manufacturing solutions.
Austria
Austria’s Vienna ecosystem is entering the ATMP race. Modular suites, electronic batch records, and advanced analytics platforms can accelerate uptake.
The Americas
Brazil
Brazil’s CGT field is growing under evolving ANVISA regulation but remains cost-constrained. Vendors who can deliver closed automation and in-process analytics that reduce cost of goods will resonate.
United States
The US remains the global leader with continued ATMP approvals, including pz-cel for RDEB. Scale-up and scale-out platforms, potency assays, and chain-of-identity systems are now essential infrastructure.
Canada
Canada is combining OmniaBio and CCRM with federal investment in AI and robotics. This creates demand for automation, MES integration, and QC technologies that support advanced manufacturing.
Closing Reflections
Our world tour underscores that CGT is global, yet not uniform. Each region presents its own opportunities and challenges, and for tools and enabling technology providers the lesson is clear: align with local drivers, but keep a global perspective.
At Lonrú Consulting, we specialize in helping enabling technology companies identify and act on these opportunities. Our VantagePoint™ suite brings together market intelligence, validation frameworks, and positioning strategies to ensure technologies not only launch but scale successfully in the dynamic CGT landscape.
The Business of Reaching Patients: ESGCT 2025 Preview
2025’s most important cell and gene therapy (CGT) conversations focus on how we pay, where we manufacture, and who gets access to therapies. ESGCT’s programme quietly concentrates several talks that sit right at that junction. Below are the sessions we’re watching for signals on real-world access, sustainable economics, and the operating models that will define CGT’s next chapter.
1. Clinical Trials & Commercialisation Workshop
(Tuesday, a.m.)
Theme: pricing, access, and launch realism.
Expect a candid sweep from regulatory mechanics to payer expectations and patient‑group perspectives. Two standouts:
Reimbursement models for ATMPs: a practical tour of contracts (outcome‑based, annuity, hybrid) and the data demands they impose on sponsors.
Advanced therapies in rare disease: research to commercial developer‑side lessons on first approvals, launch phasing, manufacturing readiness and payer evidence.
What to listen for: Where multi‑year outcomes contracts are actually landing; how to “design for reimbursement” early (endpoints, real-world-evidence capture, and post‑market commitments built into the plan).
2. In Vivo Gene Therapy
(Wednesday, 15:30–17:30)
This block is a litmus test for one‑and‑done value narratives. The way durability, safety, and quality of life are framed here will influence payer appetite and trial design across the board, especially for programs signaling pivotal readiness. Watch for language around post‑approval data, registry infrastructure, and the end of lifelong chronic therapy in specific conditions.
3. Manufacturing I: Delivery Technologies
(Wednesday, 18:00–19:30)
Session highlight: INV30 A Strategic Platform Roadmap for Commercializing Gene Therapy - Cesar Trigueros (Viralgen).
This is likely to read as an operating model talk with a view to CMC: platform efficiencies, indication sequencing, COGS sensitivity, QC and comparability, and what “commercial‑ready” actually looks like for vector manufacturing in 2025.
Also worth catching in the same session: practical levers that move the unit economics viral vector cell‑line engineering, downstream simplification, and analytical automation, for sponsors who need cost curves that stand up to HTA scrutiny.
4. Accessibility of Gene Therapy
(Thursday, 15:30–17:30)
A rare, concentrated discussion of how to reach more patients: logistics, site qualification, patient access, and payer readiness. Expect pragmatic perspectives from hospital systems and regulators on topics like:
Shipment, chain‑of‑identity, and turn‑around constraints that still block scale.
What “fair access” and “practical affordability” look like for ultra‑rare populations.
How to structure evidence generation so that pricing debates become data‑driven rather than aspirational.
5. Manufacturing II: Cell Therapies
(Thursday, 18:00–19:30)
Session highlight: INV60 Decentralized, innovative point‑of‑care CAR‑T manufacturing platform - Ruiz Astigarraga (Galapagos BV).
This is the business model experiment many have been waiting to see at scale: push manufacturing closer to the bedside to compress vein‑to‑vein, increase throughput, and reduce logistics risks.
Listen for:
What the real time gains are (collection → release).
How digital release, comparability, and QA work across a network of centers.
The site‑level economics (capex/opex, staffing, training) and who pays for what.
Related talks in this block - TIL manufacturing, cryo dynamics, and LVV platform learnings - fill in the operational detail that determines whether “point‑of‑care” is a headline or a workable system.
6. National Initiatives in Gene & Cell Therapy
(Thursday, 18:00–19:30)
This session maps country‑level frameworks for scaling academic manufacturing, coordinated trials, and patient access and how infrastructure and philanthropy‑backed models may intersect with national health systems to broaden availability.
What to listen for:
How Germany, Spain, Italy, France, the UK and Sweden are organizing capacity and standards.
Whether cross‑border data and release protocols are converging enough to reduce duplication and cost.
The role of hospital exemption and academia‑industry hybrids in earlier patient access.
7) Regulatory Requirements for CTAs
(Friday, 11:00–13:00)
With senior voices from agencies, expect clarity on:
First‑in‑human expectations for manufacturing and control.
How the evolving EU legislative landscape interacts with CGT development timelines.
What regulators want to see in post‑approval evidence to support sustained access and reimbursement.
8) EuroGCT Lunchtime Symposium
(Friday, 13:30–14:30)
Policy and regulatory strategy focused on translation and equitable delivery. Watch for signals on registries, real-world-evidence, and center‑of‑excellence concepts that could become the “plumbing” for outcome‑based payment and cross‑site learning.
Lonrú Clients: What this means for CGT enablers
Design for access, not just efficacy. If decentralized and rapid‑release models gain ground, demand rises for modular cleanrooms, integrated analytics, networked QA, and software that makes batch review and chain‑of‑identity auditable across sites.
Plan the contract before the trial. Build outcomes capture, follow‑up cadence, and real‑world endpoints into protocol design to support annuity or outcomes‑based payments later.
Academia is a serious buyer. National initiatives and philanthropic ecosystems (Italy is a prime example) are creating structured channels for tools, analytics, and training - especially where public infrastructure underwrites early access.
The sessions to circle in your diary
Clinical Trials & Commercialisation Workshop (Tuesday, a.m.) pricing, contracting, and the patient perspective.
In Vivo Gene Therapy Plenary (Wednesday, 15:30–17:30) framing “near‑market” for payers.
Manufacturing I (Wednesday, 18:00–19:30) INV30 Cesar Trigueros (Viralgen) on a commercialization roadmap.
Accessibility of Gene Therapy (Thursday, 15:30–17:30) practical access and logistics.
Manufacturing II (Thursday, 18:00–19:30) INV60 Ruiz Astigarraga (Galapagos BV) on decentralized CAR‑T.
National Initiatives (Thursday, 18:00–19:30) include Italy alongside Germany, Spain, France, the UK, Sweden.
Regulatory Requirements for CTAs (Friday, 11:00–13:00) policy reality check.
How Lonrú can help
Lonrú’s VantagePoint™ services support companies turning these signals into action:
Insights: market and policy intelligence to anticipate the access path.
Validation & Accelerate: go‑to‑market design, including networked manufacturing and payer‑ready evidence plans.
Connect & Scale: partnerships with national initiatives, academic centers and investors; pricing and scale frameworks aligned to real‑world delivery.
CRISPR Safety: Where Tools Companies Can Lead
The recent Genes & Diseases review by the CGT Catapult’s team on non-clinical safety considerations for CRISPR/Cas genome editing is a valuable reference for anyone in the enabling technology space. It sets out the practical safety and regulatory challenges that stand between promising science and first-in-human use. For companies developing tools and platforms, it offers a clear view of where targeted solutions can become part of the industry’s essential infrastructure.
At Lonrú Consulting, we work with CGT-enabling technology providers to identify exactly these kinds of opportunities. Using our VantagePoint™ framework, we help clients align product development with market demand, validate their technology for partner and regulator confidence, and plan for commercial growth. In the sections that follow, we examine the primary safety concerns highlighted in the recent CRISPR/Cas review, explore the corresponding opportunities for tools companies, and indicate where our VantagePoint™ solutions can support those companies in advancing their mission.
The recent review by Toofan et al. outlines six key safety considerations for CRISPR/Cas editing, we outline opportunities for tools companies to address each point.
1. Off-Target and On-Target-But-Unintended Edits
A starting point for many developers is the challenge of controlling where and how genome edits occur. The review details the potential for unwanted changes both at and away from the target site, ranging from single base substitutions to megabase-scale deletions and rearrangements. Regulatory bodies increasingly expect comprehensive datasets that quantify these effects and show how they are being mitigated.
Opportunity for tools companies: Develop AI-assisted gRNA design systems that take into account chromatin state and sequence context, engineer high-fidelity Cas variants, and provide GLP-ready detection platforms such as NGS, ddPCR, and single-cell sequencing.
VantagePoint™ Fit: Insights to analyze adoption rates and emerging technical standards, and Validation to format and present results in a way that satisfies regulatory expectations.
2. p53-Mediated Toxicity and Tumorigenicity
The second major concern is the unintended cellular response to double-strand breaks. The review describes how activation of p53 pathways can result in apoptosis, or in some cases, selective survival of p53-deficient cells with a higher likelihood of tumorigenicity. This risk profile can vary by target site and cell type, making it a key focus for preclinical safety assessment.
Opportunity: Create assay kits to measure p53 activity in edited cells, develop DSB-free editing technologies such as base or prime editors with companion validation tools, and design genomic stability assays for iPSC-derived and primary cell therapies.
VantagePoint™ Fit: Accelerate by guiding high-value applications for DSB-free platforms, and Illuminate by helping you communicate the safety value of your solution to potential partners and regulators.
3. Immunogenicity to Cas Proteins and Delivery Vectors
Another recurring issue in the paper is the immune system’s response to both the editing protein and the delivery vehicle. Many individuals already carry antibodies or T cells reactive to Cas proteins, and delivery methods such as AAV and LNP can trigger strong innate or adaptive immune responses. These reactions can undermine efficacy and limit repeat dosing.
Opportunity: Engineer Cas variants with reduced immune recognition, identify alternative orthologs from under-exposed bacterial species, and develop immune-profiling services to assess patient compatibility with specific delivery systems.
VantagePoint™ Fit: Connect by linking your technology to immune assay providers and therapy developers, and Scale by embedding immune risk management into development pipelines that aim for repeat administration.
4. Delivery System Limitations
The choice of delivery method shapes both the feasibility and safety of a genome editing approach. AAV vectors face strict size limits and potential high-dose toxicities, adenovirus offers larger capacity but carries a higher risk of immune activation, and LNPs can be unstable or inefficient for certain tissue types. The review notes that efficient, targeted delivery remains one of the biggest barriers to translation.
Opportunity: Create modular delivery systems that overcome payload constraints, develop hybrid viral/non-viral approaches, and offer AI-driven biodistribution modeling for early-stage planning.
VantagePoint™ Fit: Insights to track market shifts in delivery technology adoption, and Validation to provide preclinical data packages that can support licensing or partnership agreements.
5. Bio-distribution, Persistence, and Clearance
Where the editing system goes in the body, how long it remains active, and whether it lingers in non-target tissues are all central to non-clinical risk assessment. These parameters can change significantly depending on vector type, dosing, and administration route. For cell-based products, especially allogeneic or iPSC-derived, long-term tracking is a critical requirement.
Opportunity: Develop tracking technologies that are applicable from preclinical to clinical settings, create predictive PK/PD modelling platforms, and provide integration site mapping services.
VantagePoint™ Fit: Scale by positioning your product as a standard solution for generating long-term safety data, and Accelerate by enabling developers to present robust bio-distribution findings early in regulatory interactions.
6. Regulatory-Grade Non-Clinical Packages
Finally, the review emphasizes the need for non-clinical data packages that are tailored to the product, indication, and intended patient population. Both FDA and EMA stress a risk-based approach, with GLP compliance where relevant, and a focus on answering the most critical safety questions for each use case. Delivering this efficiently can shorten timelines and reduce cost.
Opportunity: Offer ready-to-use regulatory data generation templates, integrate validated assay panels with in silico and in vitro modeling, and create audit-ready dashboards for submissions.
VantagePoint™ Fit: Illuminate your technology as a tool for faster regulatory readiness, and Validation to show how your offering aligns with agency requirements without adding unnecessary study burden.
Final note:
The safety challenges discussed in the review are defining factors in whether a therapy advances to the clinic. For enabling technology companies, each represents a space where a well-designed tool or platform can become essential to the development process. The VantagePoint™ approach gives you a way to identify these openings, build the case for your solution, connect with the right partners, and create a clear path from innovation to market impact.
A global view on point of care manufacturing regulations after the UK’s leap
The UK’s Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations 2025 discussed in last week’s post, flipped the regulatory lens from post-code to process. By creating two new licenses; one for fixed hospital suites and one for relocatable micro-factories, with a single control site supervising every node through a live quality-management master file, the MHRA has written the world’s first clear pathway for turning cell, gene and other ultra-short-shelf-life products from a centralized craft into a distributed service.
For developers, that means the last, failure-prone hours of a CAR-T or CRISPR-edited product can now happen meters from the patient instead of hundreds of miles away. It also means regulators will review data-integrity safeguards, remote batch-release protocols and digital twins with the same rigor they once reserved for stainless-steel reactors which raises the bar overall, but shortens the critical vein-to-vein time. In the sections that follow we set the UK’s new framework beside emerging approaches in the United States, Japan and China to see which markets are closest to matching the MHRA’s lead and to identify where gaps remain.
Bringing the factory to the patient: we’re tracking the rules that make it possible.
United States: a framework taking shape
Across the Atlantic the FDA is edging toward a similar destination through its CDER FRAME initiative. Since 2022 the Agency has floated discussion papers on Distributed Manufacturing and Point-of-Care Manufacturing of Drugs, held dedicated workshops and, in November 2023, published a stakeholder action plan. The timetable now includes three draft guidances, the first of which (addressing control-strategy verification under 21 CFR 211) landed in January 2025. The concept mirrors the UK model (central QMS, remotely located units) but, for now, manufacturers must patch together existing CGMP provisions and request case-by-case advice while FDA finalizes its rule book.
Japan: a decade of hospital manufacture, newly widened
Japan actually legalised bedside manufacture back in 2014. Under the Act on the Safety of Regenerative Medicine (ASRM) physicians can prepare autologous cells in registered in-hospital suites, regulated site by site, while the Pharmaceuticals and Medical Devices Act (PMD Act) governs commercial products. That twin-track system is now tightening: an ASRM amendment coming into force on 31 May 2025 pulls in vivo gene therapy into the highest-risk tier and demands stronger conflict-of-interest controls and data oversight by Certified Committees for Regenerative Medicine. Japan therefore offers a functioning clinical pathway without mandating the real-time, multi-site master-file structure that the MHRA requires.
China: a maturing dual-track experiment
China’s regulators split advanced-therapy oversight between the National Health Commission (NHC), which lets accredited hospitals run investigator-initiated cell-therapy trials, and the National Medical Products Administration (NMPA), which handles industry INDs and licences. Draft NHC rules piloted in 2021 were finalized in September 2024, standardizing digital supervision and ethics governance for hospital-run trials, while the NMPA continues to refine 2017 and 2021 technical guidelines for gene-modified cells . The model already enables bedside manufacture, but licenses remain site-specific and provincial, and there is no single, central control-site concept, leaving data harmonization and nationwide scalability as open questions.
What this signals
Taken together, these moves show convergence on a core idea: personalized therapies can be finished where the patient is, yet controlled as if they were still made in one building. The UK supplies the first full legal scaffold; the FDA is drafting its solution, Japan is extending an established hospital channel into gene therapy, and China is aligning its fast-growing hospital track with national standards.
For therapeutic innovators the lesson is clear: design digital-first quality systems that can stream data from dozens of small reactors, bake remote intervention into CMC plans, and pick an early pilot jurisdiction in London, Boston or Tokyo to prove the model.
For technology innovators, the engineers building closed, automated cell-processing suites, rapid sterility assays, inline analytics, electronic batch-record platforms and AI-powered deviation handling, the opportunity is just as immediate. Hospitals will invest in turnkey, compliance-by-design micro-factories that drop into existing real estate, streaming validated data back to a control site and that can be overseen, paused or released by a Qualified Person half a continent away. Vendors that can wrap hardware and software into a tamper-proof, cloud-native ecosystem, complete with digital twins, secure SOPs and encrypted audit trails will find the UK an unrivaled proving ground and a springboard for the FDA’s forthcoming guidance.
MHRA’s Point‑of‑Care Manufacturing Framework
The UK’s MHRA unveils first-of-its-kind framework allowing hospitals to manufacture personalized cell and gene therapies on-site.
A New Era for Cell & Gene Therapy
Just last week, the UK Medicines and Healthcare products Regulatory Agency (MHRA) quietly did something revolutionary: it switched the regulatory focus for advanced therapies from where they are made to how their quality is controlled. The Human Medicines (Amendment) (Modular Manufacture and Point of Care) Regulations 2025 create the first dedicated legal pathway that allows hospitals, and even mobile clean‑room units, to complete the final stages of cell‑ and gene‑therapy manufacture right at the patient’s bedside.
What the new rules actually say
The legislation introduces two new manufacturing licenses. A Manufacturer’s License (Point of Care) covers fixed hospital suites, while a Manufacturer’s License (Modular Manufacture) captures relocatable or mobile micro‑factories. In both cases a single control site (e.g. a centre of excellence or a technology provider) holds the license, maintains the quality‑management system and supervises every distributed site in real time through a master file. The framework applies not only to autologous CAR‑T and gene‑edited cell products but also to 3‑D‑printed implants, medicinal gases and a handful of other short‑shelf‑life products that have previously struggled to fit into the conventional GMP regime.
Crucially, the MHRA has also tightened historic exemptions that allowed clinicians to skirt full GMP by talking about “simple mixing at the bedside.” If you are performing anything that looks like manufacture e.g. cell expansion, genome editing, final formulation, aseptic fill, you now need to follow the new license route. In return the field gains regulatory recognition that some therapies cannot survive a cross‑country drive to a central facility, let alone an international flight.
Why therapeutic developers should take notice
For developers of cell and gene therapies the POC framework is more than an administrative tweak. In fact, it reshapes CMC, clinical development and launch strategy in one move. Processes that were previously split between a primary facility and a courier can now be divided between a central hub for complex manipulations and a hospital suite for the high‑risk, time‑critical finishing steps. That change alone can shave days off vein‑to‑vein time, reduce cryo‑shipping failures and unlock adaptive trial designs that rely on immediate dosing.
Regulatory‑CMC teams will need to engage early with the MHRA to agree the contents of the POC Master File, the location of batch‑release testing and the digital systems that underpin real‑time oversight. The agency has signaled a willingness to use its Innovative Licensing and Access Pathway (ILAP) to accelerate such conversations, but developers should expect probing questions about data integrity and remote intervention protocols.
Commercial leaders, meanwhile, must rethink launch footprints. A therapy that can be finished on‑site will naturally gravitate toward NHS trusts with established GMP capacity, shifting the centre of gravity away from a single national facility toward a federation of hospital networks. Intellectual‑property management also rises up the agenda: closed, automated systems, secure SOPs and encrypted audit trails become proprietary assets that differentiate one therapy from another.
Implications for the enabling‑technology ecosystem
If you build the tools that power advanced‑therapy manufacture, the UK has just become an essential proving ground. Hospitals will seek compact, closed, automated suites that can drop into existing infrastructure without a multi‑million‑pound refurbishment. Those systems must stream batch data back to the control site, plug directly into electronic batch records and trigger remote release testing that takes hours rather than days.
Software providers face parallel opportunities and responsibilities. The distributed model only works if every site can be seen, in real time, by the license holder and, by extension, the MHRA. Expect hospitals to demand digital twins, inline analytics and AI‑assisted deviation handling as standard, not as premium add‑ons. Rapid microbial sterility tests, single‑use bioreactors optimized for autologous volumes, and augmented‑reality training modules will all find a readier market now that the regulatory path is clear.
Go‑to‑market strategy should also shift. NHS trusts, not just biotechs, become primary customers. Vendors that can wrap hardware and software into a turnkey, compliance‑by‑design offering will have a compelling first‑mover advantage. Success in the UK will provide a template that other regulators are likely to emulate, turning early adoption into global leverage.
The Lonrú perspective: clients navigating first and fast
At Lonrú we see three immediate priorities. First, therapeutic developers should map their existing manufacturing architecture and pinpoint which unit operations could be safely migrated to a point‑of‑care environment. Second, both developers and tool providers should initiate early scientific‑advice dialogues with the MHRA to clarify expectations around digital quality management, remote batch release and control‑site governance. Finally, stakeholders must invest in robust tech‑transfer packages, complete with remote‑support protocols and tamper‑proof data pipelines since the distributed model is only as strong as its weakest node.
The UK has opened the door to a future in which genuinely personalized medicines can be made, tested and delivered within the same clinical encounter. Those who step through early will help define the standards the rest of the world adopts and will gain invaluable operational insight and skills along the way. Lonrú Consulting stands ready to guide developers and technology providers alike through this new landscape, from strategy and competitor landscaping to hospital‑channel execution.
Beyond NPV: Quantifying Patient Benefit in Cell & Gene Therapies
Picture an investment committee reviewing two first-in-class gene-editing programs. Financial models show identical risk-adjusted NPVs, yet one promises four times the projected patient benefit. Which wins the vote? The story isn’t about any single foundation; it’s about a recurring dilemma in CGT financing where dollars and patient impact don’t always point to the same deal.
Traditional rNPV remains the lingua franca of venture finance, but CGT’s high R&D costs, small patient pools and curative ambitions make monetary returns unusually volatile. Layering patient impact alongside NPV offers a stabilising compass, ensuring capital flows toward programmes that move both the balance-sheet and the survival curve. Just last week, Philip Brainin (Associate at Sound Bioventures) published a new framework in Nature Biotechnology to measure the societal impact of venture investments in life sciences.
Quick Primer on SALYs & SADYs
Brainin’s framework proposes two patient-centric metrics tailored for investors. Science-Adjusted Life Years (SALYs) and Science-Adjusted Disability Years (SADYs) building on global health metrics like DALYs and QALYs but adjust for:
Probability of Scientific Success: SALYs are discounted by the likelihood a therapeutic candidate clears regulatory hurdles.
Equity Ownership: SALYs can be normalized per $ invested, per share class, or per co-investor share creating comparability across venture and public-private deals.
Where DALYs account for disease burden lost, SALYs forecast life-years gained a new measure grounded in both biology and market realism. SALYs and SADYs incorporate four key elements: (i) disease incidence, (ii) target-product-profile reach, (iii) change in likelihood-of-approval, and (iv) survival or disability benefit into a single number that discounts for scientific risk while scaling for real epidemiology.
NPV reaches first for finance levers: discount rates, price and market uptake, while the SALY/SADY construct substitutes phase-specific probabilities of success, incidence and disability weights. The side-by-side comparison in Table 1 below makes the contrast explicit and underscores why the two metrics work best together: NPV flags capital efficiency, while SALY/SADY illuminates the magnitude of patient impact.
Table 1. Financial levers dominate NPV calculations, whereas disease burden and probability estimates dominate SALY/SADY making the two metrics complementary rather than redundant.
Cell and Gene Therapies: The Ultimate Stress-Test
Because single-dose curative therapies concentrate an entire lifetime of revenue into an upfront payment, traditional rNPV estimates can fluctuate sharply with every adjustment to price assumptions. In contrast, SALYs ground the analysis in quantified patient benefit, providing a more stable indicator of programme value. For example, a one-time prime-editing treatment targeting just 400 patients may still generate several thousand discounted SALYs, demonstrating substantial societal impact even when the commercial total addressable market remains modest. That analysis may be crucial for foundations deciding between grants and equity, as well as HTA agencies assessing early economic value and impact funds seeking to prove additionality.
The matrix below illustrates how SALY and SADY calculations can re-rank programmes once patient-benefit intensity is considered. High-incidence mainstream drugs (e.g., an antibiotic for urosepsis) unsurprisingly dominate absolute SALY totals, yet several CGT examples; Casgevy for sickle-cell disease, Vertex’s VX-880 for type 1 diabetes, and Lexeo’s LX2006 for Friedreich’s ataxia deliver double- and triple-digit SALY counts from comparatively smaller patient pools.
Table 2. Annual Science-Adjusted Life-Years (SALYs) and Science-Adjusted Disability-Years (SADYs) for representative therapeutics spanning Brainin’s original examples and a selection of cell- and gene-therapy (CGT) assets†
† Incident U.S. patient population, mirroring Brainin’s convention.
§ Birth prevalence ≈ 1 : 2,024 (CDC) → ~1,830 births / yr; halving to those with ≥ 2 VOCs ⇒ 1,350.
¶ U.S. annual DMD diagnoses estimated at 300–400 boys / yr (1 : 5 000 male births).
†† CDC reports just over 18,000 youth diagnosed with T1D each year.
‡‡ FA affects 1/50,000 people; 4 M births × 1/50,000 ⇒ ~80 births; 60 % meet cardiac-gene-therapy criteria ⇒ ~50.
§§ GenSight estimates 15,000–20,000 new RP vision-loss cases per year in the US + EU; mid-point used for US.
Value for Patient Alliances & Impact Investors
The comparison highlights why impact-minded investors, foundations, and HTA agencies should weigh both population size and per-patient life-year gain rather than relying on rNPV alone: a modest-TAM curative can still generate a disproportionate share of risk-adjusted patient benefit. Moreover, quantified patient benefit can arm advocates with data for accelerated review petitions, outcome-based pricing arguments and R&D tax-credit campaigns.
Inserting SALY forecasts into horizon-scans lets bodies like NICE or FDA’s Health-Equity initiative flag high-impact indications earlier. Likewise, manufacturing grants, fee waivers or voucher tweaks can be recalibrated to favour high-SALY but commercially challenging diseases.
Implementation Playbook
Lonrú’s VantagePoint™ Suite can combine global burden of disease epidemiology with our proprietary phase-transition probability library to generate turnkey SALY/SADY dashboards. Beyond the analytics, Lonrú can partner directly with patient alliances to run evidence-gathering workshops, analyse and validate disability weights, and co-author advocacy briefs that translate SALY insights into concrete asks for regulators and payers. Lonrú Consulting operates as a neutral translator between financial, clinical and patient voices. By integrating Brainin’s novel SALY/SADY calculus into our advisory workflows, we can help stakeholders steer capital and policy toward truly transformative CGT programmes.
Bridging The Data Gap On The Pediatric CRISPR Highway
Reflections On The new CZI-IGI Centre for Pediatric CRISPR Cures
(Follow-up to our previous post “Building the U.S. Interventional-Genetics Interstate”)
Last week, the Chan Zuckerberg Initiative (CZI) and the Innovative Genomics Institute (IGI) unveiled a $20 million Center for Pediatric CRISPR Cures that will treat eight children using bespoke gene-editing protocols and share data and methodology with other academic centres, amplifying the impact to reach more patients. The funding cements a highway-scale build-out of personalised editing that began with Baby KJ’s bespoke therapy announced earlier this year. Below we zoom in on KJ’s blueprint, note the opportunities that remain for tools and service providers, focusing on the not yet mentioned data-infrastructure lane. And we’ll discuss how rapidly growing Databricks and Snowflake solutions could slot in beside the big-cloud contenders.
KJ’s blueprint was tuned to target the liver, requiring a liver-specific off-target panel, and relied on six-to-eight-week reagent lead-times and notably had no disclosed informatics backbone. Those gaps are build-out opportunities to accelerate the next phase: ligand-decorated or capsid-mimetic nanoparticles for bone-marrow / CNS delivery, bench-top mRNA/gRNA micro-factories with “factory-as-code” manufacturing systems, duplex long-read safety assays folded straight into IND templates, and overlaying it all, a federated data layer able to convert patient record streams into Beacon-discovery and real-world-evidence dashboards at interstate speeds informing the next patient-specific therapy.
Figure 1: Blueprint of the “next-gen” CRISPR-cure highway: from patient intake, through rapid design and micro-factory production, into a secure cloud backbone that feeds real-world evidence back to the very first node.
Who might “pour the concrete” for the missing data layer?
The heavy lifting will almost certainly happen on one of the big public-cloud platforms - it’s just a question of which one provides the right plug and play solutions.
Amazon Web Services already offers a specialist toolbox called AWS HealthOmics. Think of it as a ready-made workshop where research centres can upload genetic data and run standard pipelines without building their own servers. It even hosts an open-source workflow standard called GA4GH-WES (basically a universal “power outlet” for genomic software).
Google Cloud brings its Healthcare API which acts like a data-ingestion gate built around the hospital record format FHIR, plus BigQuery, a powerful spreadsheet-on-steroids that analysts love. Google has even published point-and-click guides for translating FHIR data into the research-friendly OMOP layout, so you don’t need an army of data engineers to get started.
Oracle is pitching Oracle Health Data Intelligence, a cloud warehouse that isn’t tied to any one electronic-health-record system but still has hooks into its own Cerner software. Recent upgrades add in-house artificial-intelligence services so users can, for example, predict which patients might respond to a treatment.
Whichever cloud supports the center, the infrastructure will still benefit from specialist “add-ons” to make day-to-day work easy for scientists and compliance officers:
Databricks Lakehouse which operates like a giant notebook for data crunching and can sit near the hospital edge. Teams can write code to clean raw files (that’s the “code-first ETL” piece), build machine-learning models, and then share polished tables using a no-copy hand-off called Delta Share.
Snowflake Healthcare & Life Sciences Data Cloud can act as the central hangar. It provides locked-down “clean rooms” where regulators or insurance companies can analyse data without ever downloading it which is ideal for privacy-sensitive real-world-evidence studies.
Databricks accelerates how quickly each site can generate validated data and scalable models for future custom therapies; Snowflake governs how broadly those insights can be shared and queried. That tandem; fast edge processing plus a secure, elastic core is what turns eight bespoke therapies in CZI/IGI’s center into hundreds of “recipe-ready” cures across multiple hospitals without rebuilding the stack each time. In practice, we could anticipate an “edge Databricks, core Snowflake” pattern: hospitals do their raw prep in Databricks, push the curated results into Snowflake, and Snowflake becomes the official registry - no matter whether the underlying cloud hardware belongs to AWS, Google, or Oracle.
What to watch for next
IGI is expected to disclose the eight-patient disease roster at ASGCT 2026; that list will reveal whether the liver-first foundation can stretch to immune or CNS targets. An RFP for a Beacon v2-compliant registry will tip the favourite cloud stack, and early FDA CBER pilots on distributed CMC will determine how quickly the cookbook and recipes can propagate between hospitals. Vendors that align their delivery tech, analytics panels or micro-factory kits with the eventual backbone will find themselves moving quickly along the newly established gene editing interstate highway, while slower movers will fight for on-ramps.
Who Follows Verve?
Cash Runway, Indication “White-Space”, and Where the Tools Ecosystem Can Play A Part
Lilly-Verve: A Wake-Up Call for the Gene-Editing Supply Chain
Eli Lilly’s agreement to acquire Verve Therapeutics for $1.3 billion delivered the first big-pharma endorsement of an in-vivo base-editing therapy aimed at a common cardiovascular risk factor. For developers this affirms that a well-differentiated programme can command a premium while still in early clinical stages; for the companies that supply lipids, vectors, analytical assays, single-cell QC or GMP processes the message is equally clear: the strategic value of an editing platform now rises or falls with the maturity of its tool-chain. In other words, big-pharma is prepared to pay early only when it can see a credible route to scale-up, and that route is paved by the specialist providers working behind the scenes.
Who We’re Tracking, and Why a Few Familiar Names Don’t Appear
Our lens concentrates on Beam Therapeutics, Intellia Therapeutics, CRISPR Therapeutics, Editas Medicine, Caribou Biosciences, Prime Medicine and Precision BioSciences. Each of these seven companies is publicly listed, still independent, and already has at least one drug-editing programme in the clinic or cleared for first-in-human testing. Recently purchased firms such as Capstan (now inside AbbVie), EsoBiotec (acquired by AstraZeneca) and Poseida (absorbed by Roche) no longer control their own financing cadence, while delivery-only or RNA-editing specialists like ReCode, ShapeTX and Korro tend to measure progress by platform milestones rather than therapeutic read-outs. Note, Precision’s ARCUS technology earns its place even though it is derived from a homing endonuclease rather than Cas9: it performs the same sequence-specific cut in an in-vivo setting and therefore competes for exactly the same opportunities.
White-Space Shots vs. Second-Wave Plays: The Current Indication Map
Within that group the spectrum of ambition is striking. Verve, now partnered with Lilly, and CRISPR Therapeutics are pushing into cardiovascular lipid disorders where no first-generation genome editor has yet secured approval, while Intellia is offering a one-and-done alternative to the chronic RNA-interference treatments already in play for ATTR amyloidosis. Intellia also reported fresh NTLA-2002 data yesterday, boosting investor sentiment but not altering its runway guidance. Programmes from Beam, Editas and Caribou focus on haemoglobinopathies or oncology indications that regulators have already seen in an autologous or first-wave CRISPR guise, making differentiation a question of efficiency, safety and patient access. Prime Medicine and Precision BioSciences step into ultra-rare chronic granulomatous disease and chronic hepatitis B respectively, both areas without an existing curative option. We have added Verve/Lilly to the indication table so we can compare how the acquired asset fits alongside the still-independent cohort.
Source: Lonrú Consulting’s VantagePoint™ analysis, July 2025. The post-Verve field of clinical-stage gene-editors and where each sits on the white space v second wave spectrum.
Charting runway next to market size, and why Verve serves as a useful benchmark
Mapping each company’s publicly guided cash runway on one axis and the projected 2030 market size of its lead indication on the other helps reveal who may seek partnership cash soonest and who can afford to take a longer view on tool selection. The Verve/Lilly transaction provides a helpful reference point: Verve entered the deal with roughly twenty-four months of runway remaining and was addressing a market comfortably north of twenty-five billion dollars. Any company that shows a shorter runway or a smaller commercial opportunity may need to demonstrate an even tighter command of its supply chain, or else invite partnership earlier than planned. For tool providers this plot highlights two complementary paths: larger-TAM, longer-runway players can commit to multi-year, higher-margin supply agreements, whereas shorter-runway firms may look for creative cost-sharing structures that still ensure uninterrupted development.
Source: Lonrú Consulting’s VantagePoint™ analysis (July 2025). Cash-runway figures drawn from latest SEC filings; 2030 market sizes from consensus analyst and disease-area forecasts.
From the supplier’s perspective the priorities differ by modality. In-vivo cardio and hepatitis programmes rely on ever finer lipid chemistry, capsid engineering and genome-wide off-target analytics; vendors that can package those capabilities together become attractive long-term partners. Ex-vivo haem-onc projects remain sensitive to cost of goods and therefore reward vendors who can combine closed-system cell hardware with lean vector manufacture or cost effective alternatives. Throughout, the tone is shifting from transactional reagent sales to collaborative co-development aimed at building IND-ready modules that regulators will recognise.
Signals to Watch and Practical Moves for Lonrú Clients
Regulators are already signalling an interest in longer durability and safety read-outs for single-shot LDL-C and HBV cures, a change that is likely to boost demand for high-resolution sequencing assays and longitudinal biomarker platforms. Macroeconomic uncertainty means companies holding less than eighteen months of cash could accelerate partnering or M&A conversations, potentially redrawing the competitive field in a matter of quarters. Meanwhile, any early clinical success from prime editing or ARCUS could reshuffle licensing appetites in much the same way Verve’s early cardio data did in 2024.
For Lonrú’s clients the immediate takeaway is to align commercial efforts with those scientific and financial inflection points. Cardiometabolic and hepatitis programmes offer the largest revenue head-room and will tolerate premium pricing if the tools accelerate path-to-market, whereas the haem-onc field will reward solutions that deliver efficiency and regulatory clarity at a competitive cost. Engaging earlier; ideally before a cash-short biotech reaches its fund-raising cliff, creates room for more balanced, longer-term collaboration that can survive the inevitable shifts that follow a strategic acquisition.
From Pipeline Fillers to Platform Plays
Yesterday morning, AbbVie dropped a $2.1 billion headline - announcing it will buy Capstan Therapeutics and, with it, a pre-clinical in-vivo CAR-T “toolkit” capable of re-programming immune cells inside the body. The deal isn’t about slotting a single late-stage asset into AbbVie’s post-Humira pipeline; it’s about owning the full technology operating system comprising Capstan’s targeted LNP delivery and its programmable mRNA payloads when combined can spin out dozens of future programs.
Yesterday’s news is the latest, and loudest, data-point in a year-long pattern: Lilly’s base-editing buyout of Verve, BMS’s prime-editing pact with Prime Medicine, and now AbbVie’s Capstan swoop all signal that Big Pharma’s shopping cart has moved from finished goods to modular tech stacks. In this Lonrú Lens post, we’ll unpack why platform control is eclipsing asset acquisition, how in-vivo versus ex-vivo strategies are reshaping CMC and analytics demands, and, most importantly, what this shift means for companies supplying the “picks and shovels” of cell-and-gene-therapy (CGT) manufacturing and quality. Expect a deep dive into the new M&A economics, the tooling gaps Big Pharma just inherited, and the strategic plays that CGT enablers should make before the next billion-dollar platform changes hands.
Why the pivot?
Patent-cliff paranoia: With blockbusters like Humira already off-patent, acquirers are chasing engines that can feed multiple franchises, not one-shot drugs.
CapEx vs Build-time: Buying a fully formed editing or delivery “OS” leap-frogs the 5-7 year slog of internal development.
Competitive moats shift upstream: If editing fidelity, delivery tropism or CMC automation become the differentiators, owning the platform locks competitors out of entire modality classes.
What a platform deal looks like in practice
The three deals in focus that have re-framed 2024-25 M&A illustrate just how differently “platform” can look in practice, and why the supporting tool chain must be equally diverse. AbbVie’s buy-in to Capstan gives it an in-vivo programmable-cell platform, where targeted lipid-nanoparticle (LNP) composition and real-time bio-distribution analytics are the gatekeepers of potency and safety. Lilly’s takeover of Verve centres on systemic base-editing, a population-scale play that makes high-throughput gRNA library design, on/off-target NGS panels and seamless digital batch-records non-negotiable. Bristol Myers Squibb’s alliance with Prime Medicine, meanwhile, is rooted in ex-vivo prime editing, pushing the need for closed, automated cell engineering suites and single-cell QC to handle multiplex edits at commercial scale. Read together, the deals prove that Big Pharma isn’t just buying optionality; it’s inheriting distinct, capital-intensive tooling gaps. In the next section we explore how specialised CGT vendors can turn those gaps into growth.
One size doesn’t fit all: each CGT platform archetype carries its own toolbox.
The ripple-effect for CGT tool providers
For companies that make the “picks and shovels” of cell-and-gene manufacturing, the new platform land-grab translates into a race for capacity, data fluency and regulatory credibility, simultaneously. Big Pharma’s acquisition teams have bought themselves ambitious editing blueprints, but they did not buy the supply-chain depth, digital infrastructure or quality modules needed to run those blueprints at scale. That shortfall thrusts specialised vendors into a more strategic role: LNP-analytics firms are being invited to embed directly into CMC work-streams; cloud-native e-batch-record providers are negotiating multi-asset master service agreements; microfluidic-hardware players are fielding requests for closed, GMP-ready pods that can be dropped into established manufacturing plants. In other words, tooling companies are no longer peripheral suppliers instead, they are fast becoming co-architects of the very platforms Big Pharma just paid billions to control. The winners will be those that can plug gaps today while designing modular solutions that won’t bottleneck the multi-indication pipelines these deals aim to unlock in the next five to ten years.
Three emerging CGT platform engines: in-vivo programmable cell therapy, systemic base editing and ex-vivo prime editing - ready to power a decade of multi-asset pipelines.
Strategic plays for tooling companies
To capture the white-space that Big Pharma’s platform buys have opened, CGT tool providers will need to stop thinking like commodity suppliers and start behaving like platform accelerators. The first step is commercial: price your offering against the value it unlocks across an entire multi-programme stack, not on a per-lot consumable basis. Next comes product architecture - design every module to plug seamlessly into either an in-vivo or ex-vivo workflow, because sponsors may soon straddle both. Data is the third pillar: milestone-heavy deals shift technical risk onto development partners, so the vendor that can surface lot-to-lot reproducibility, predictive release analytics and audit-ready batch records becomes indispensable. Finally, rethink deal structures altogether; in a world where the tool can determine whether dozens of assets reach the clinic, equity, royalties or shared IP could be worth more than a traditional supply contract.
Bottom line
Pipeline filler M&A is giving way to platform play M&A. For CGT tool providers, that means the next wave of growth will come from positioning your technology as critical infrastructure for these newly acquired operating systems. The winners will be those who can scale, standardise and data-enable the platforms that Big Pharma just paid billions to own.
Lonrú Lens will continue to track how these platform deals reshape the CGT value chain - and where enabling technology companies can capture value in 2026 and beyond.
The Non-Viral Delivery Surge: LNPs, Extracellular Vesicles & the Emerging Pipeline in Cell & Gene Therapy
What recent signals indicate
Each year, the American Society of Gene and Cell Therapy (ASGCT) annual meeting provides one of the clearest forward signals of where innovation is heading in CGT. With thousands of abstracts spanning academic, preclinical, and translational work, it acts as a near real-time readout of what platforms, targets, and tools are capturing attention and investment.
In April, we flagged a sharp uptick in lipid nanoparticle (LNP) interest across the 2025 abstract preview. Now, in the wake of Eli Lilly’s $1.3 B acquisition of Verve Therapeutics, that trend is no longer just academic. Verve’s GalNAc-functionalised LNP platform for in-vivo base editing offers a concrete signal that non-viral delivery is maturing into the clinic.
To be clear: many of these programs remain early-stage. It’s far from certain that in-vivo gene editing or exosome therapeutics will become the dominant modalities. But what’s emerging is a persistent and distinct trend line: away from viral vectors, and toward programmable, scalable, and re-doseable delivery platforms.
This post offers a tactical recap, from the data to the deals, and highlights what CGT tech providers should be doing now to stay ahead.
What’s Emerging? Key Technical Trends
From in-vivo CAR-T therapies to prenatal gene editing and designer extracellular vesicles, the technical conversation is evolving fast. This year’s standout sessions point to a distinct pivot: CGT developers are increasingly pursuing delivery systems that are programmable, re-doseable, and capable of bypassing traditional manufacturing bottlenecks. Each modality has different CMC and clinical implications, but all push the field closer to de-centralized, patient-centric models.
In-vivo CAR-T via targeted LNPs
Bypasses costly and logistically complex ex-vivo manufacturing and enables re-dosing potential.
Talk highlight: “Cell-targeted LNP‑based CAR‑T Therapy for B‑cell Malignancies”Prenatal gene editing using LNPs
Uses LNPs to treat congenital disease in utero, pushing boundaries of delivery and ethics.
Talk highlight: “LNP‑Based Delivery Systems for Prenatal Applications & Ethical Considerations”Designer extracellular vesicles (EVs)
Natural bio-distribution with tunable tropism; lower immunogenicity profile than synthetic carriers.
Talk highlight: “Scalable Manufacturing of MSC‑Derived EVs for Oncology”
Figure 1 | From syringe to hepatocyte in one step.
A single intravenous dose of ionizable-lipid nanoparticles (LNPs) delivers base-editing RNA directly to the liver, bypassing ex-vivo manipulation and shortening the therapeutic chain to needle-to-target.
Real-World Momentum via Deal Flow
Non-viral delivery platforms are no longer just for fringe programs. In the month following ASGCT, major deals and facility announcements like Lilly’s $1.3B buyout of Verve and BASF’s new excipient hub, reinforce the commercial weight behind this trend. From big pharma acquisitions to upstream investment in GMP infrastructure, the ecosystem is visibly recalibrating to support non-viral modalities.
Tactics for Enabling Technology Providers
For tech providers in analytics, mixing, excipients, or cleanroom design, the message is clear: adapt now to serve the next generation of therapies. Tactical moves like securing GMP lipids, modularizing mixing systems, or redesigning lipid analytics aren’t speculative, they’re prerequisites for relevance. Lonrú’s VantagePoint dashboards help prioritize what matters by product class and market stage.
How Lonrú Consulting Can Help
Lonrú helps enabling tech companies make strategic sense of CGT market shifts. Whether you're refining your value proposition, identifying high-fit partners, or plotting entry into the non-viral delivery space, we deliver clarity and actionability. Our approach blends market intelligence with customized partner strategy and commercial mapping ensuring you don't just track trends, you lead them.
Strategy Depth: We don’t just summarize the buzz, we decode its strategic consequences. Our VantagePoint™ Insights platform helps you see around corners.
Execution Alignment: Whether you’re building out your product roadmap or targeting new customers, we offer partner-mapping and strategy to align growth with opportunity.
Non‑Viral Commercial Strategy: If your technology enables non-viral therapeutic developers, we can help you map your market, position your offering, and sharpen your go-to-market plan.
Bookmark earlier reads
Through the Lonrú Lens - What to Watch at ASGCT 2025 (May 2025) for our conference preview that first spotlighted the looming LNP surge and non-viral delivery sessions.
From Bottleneck to Backbone: Building the U.S. ‘Interventional-Genetics Interstate’ (June 2025) for a deep dive into modular GMP build-outs, micro-batch production, and the equipment choices enabling rapid scale-up.
Meeting on the Med: A Pulse Check on CGT (April 2025) for field notes on industrialization pressures, COGs calculations and the analytics upgrades vendors must make to stay competitive.
From Bottleneck to Backbone: Building the U.S. “Interventional-Genetics Interstate”
Pioneer of next-generation gene editing, David Liu’s challenge at last week’s FDA cell and gene therapy round-table was stark: if the United States can compress concept-to-clinic manufacturing to “well under 90 days,” at least a thousand ultra-rare patients could receive bespoke in-vivo gene-editing therapies by 2030. The underlying science may be ready, but the physical and digital scaffolding that would let those therapies flow is not. Below is a high level review of where the U.S.’s infrastructure currently stands, where the cracks lie, and how proven international models can help finish the road in time.
A patchwork that almost works
Start-ups and academics have shown glimpses of what an agile infrastructure might look like. Philadelphia’s CHOP/UPenn Clinical Vector Core has produced 181 GMP vectors for 37 INDs touching 859 patients since 2007, showing what lean, mission-driven facilities can do over time. Vector production timelines remain a bottleneck for the field - Andelyn Biosciences’ 16-suite Columbus headquarters released its first clinical AAV lot in May 2025 ten months after tech-transfer began. Other modalities are emerging to address the shortcomings of vectors - in Boston, Landmark Bio’s 44 k ft² “full-spectrum” facility producing mRNA, LNPs and traditional vector products has been running since late 2022, providing eight re-configurable cleanrooms within walking distance of the Harvard–MIT medical cluster. Federally, the Bespoke Gene Therapy Consortium (BGTC) selected eight prototype diseases in May 2023 to demonstrate a repeatable regulatory-and-manufacturing playbook, while NINDS’ URGenT Network continues to fund IND-enabling work for ultra-rare neurologic disorders under its January 2025 funding notice (PAR-25-326).
Yet capacity is brittle. National Resilience, once the poster-child for “mega-factory” gene-therapy CDMOs announced yesterday, that it will close six of its ten U.S. sites because “capacity expansion has outpaced demand”. The message is clear: square footage alone does not guarantee throughput.
The gap between ambition and reality
Even with bright spots, most sponsors still wait nine months or more for GMP vector slots, rely on site-specific QC assays, and repeat pre-clinical toxicology from scratch; miles away from Liu’s sub-90-day aspiration. The United States currently lacks:
Geographic reach. Production remains concentrated in a handful of coastal hubs; patients elsewhere often cross state lines for dosing.
Shared digital infrastructure. Batch-release data live in siloed LIMS, making cross-site comparability almost impossible.
Economic resilience. Large plants built for blockbuster demand now sit idle, while hospital vector cores cannot scale beyond a dozen lots per year.
Lessons from health systems that are already doing it
The U.K.’s Advanced Therapy Treatment Centre (ATTC) Network embeds GMP suites inside NHS hospitals and links them through a national digital backbone. In February 2025 the UK government injected another £17.9 million to extend the model, explicitly to accelerate early-phase ATMP trials across the country. Every centre runs 2–4 multipurpose rooms and shares a harmonised set of SOPs - exactly the federated approach Liu argues the U.S. needs.
Singapore’s ACTRIS hub takes the same idea to city-state scale: 14 GMP-compatible suites, four translational labs and a single QC core feed hospitals, academics and start-ups under one roof, coordinated by a government logistics task-force. Both systems show that small, interoperable nodes can beat single mega-sites on utilisation, workforce agility and patient proximity.
Investing in nimble, connected manufacturing infrastructure today paves an Interventional-Genetics Interstate that will shorten timelines, cut costs and most importantly get transformative treatments to rare-disease patients faster.
How a U.S. Interventional-Genetics Interstate could take shape
Repurpose idle space. One wing of a downsized Resilience plant could house several 500 ft² closed-system pods tailored to micro-batch gene-editing runs, while the rest converts to warehousing and analytics. That keeps sunk capital alive without overshooting local demand.
Create a national QC cloud. Require BGTC and URGenT awardees to deposit release data into a shared LIMS opening the door to “plug-in” manufacturing sites that regulators can audit remotely.
Adopt a “grants-not-guarantees” funding model. NIH C06 facility grants and state matching schemes already release construction dollars in tranches tied to occupancy or throughput milestones; the same approach could seed regional micro-factories without repeating past oversupply.
Spin a workforce fly-wheel. Short, NIIMBL-style bootcamps that rotate technicians between hospital cores, CDMOs and start-ups would create a steady talent circuit instead of one-off hiring sprees.
Institutionalise digital twins. Inline PAT sensors plus cloud process models now routine in UK Catapult pilots, can cut tech-transfer time and give the FDA live visibility, shaving weeks off each bespoke batch.
What it means for technology suppliers
For analytics and PAT vendors, interoperability will clinch procurement decisions. Equipment makers should design skids that fit tight footprints and swap quickly between AAV, LNP and mRNA workflows. Digital-platform providers can own the value chain by stitching together batch genealogy from lab bench to hospital bedside. In short: if your tools make micro-batch production faster, cheaper and easier to regulate, the Interstate will need you.
The road ahead
The United States already has all the raw materials: pioneering science, ambitious CDMOs, and a small but growing federal appetite for public-private gene-therapy ventures. What it lacks is connective tissue. The next five years will decide whether the U.S. can deliver a federated, nimble network that can crank out micro-batches for 7,000+ ultra-rare conditions. Borrowing the ATTC formula of regional hubs plus national standards, and the ACTRIS emphasis on co-located clinical-manufacturing ecosystems, could close today’s fissures faster than any single plant investment. The prize is tangible: a future in which a child diagnosed this morning could receive a customised in-vivo therapy before the end of the school term.
Reflections from ASGCT 2025
He Wishes for the Cloths of Heaven
W. B. Yeats (first published 1899)
Had I the heavens’ embroidered cloths,
Enwrought with golden and silver light,
The blue and the dim and the dark cloths
Of night and light and the half-light,
I would spread the cloths under your feet:
But I, being poor, have only my dreams;
I have spread my dreams under your feet;
Tread softly because you tread on my dreams.
A breakthrough worth noting: an n-of-1 gene-editing therapy. Let’s celebrate the science, but keep patients and their families front-of-mind and proceed with care as we work to reform the business model.
On the flight home from New Orleans, the week’s jazz still echoing in my earbuds, a line from Yeats surfaced -“Tread softly because you tread on my dreams.” It summed up my reflection on the hopes and uncertainties that patients and families carry as they wait for the promise of cell and gene therapies.
A milestone worth celebrating
Professor Kiran Musunuru’s late-breaking CPS1 “N-of-1” base-editing success in baby KJ created much excitement and celebration. Six months from molecular diagnosis to first infusion; ammonia crises averted; a liver-transplant listing cancelled. As blueprints for bespoke in-vivo editing go, this one is pure architectural poetry.
Yet one swallow does not make a summer
Our modelling estimates, visible here pulling on published commercial GT COGs and the three-dose LNP regimen puts the price tag for this breakthrough therapy well north of US $1 million. Even if payers were willing, the bespoke CMC, analytics and logistics cannot be amortised across a wider patient pool today. KJ’s story is a beacon, not yet a bridge.
Voices that kept us grounded
In a powerful fireside panel, patient-advocate Oralea Marquardt reminded us what happens when hopes are shattered after investors pivot and trials are shelved. Prof. Don Kohn recounted how an approved ADA-SCID gene therapy survived only by shifting into a Public Benefit Corporation (Rarity Inc.) when Orchard exited. Dr Claire Booth urged proportionate regulation and innovative reimbursement, warning that repeated withdrawals erode family trust.
Their refrain? Language matters. So does honesty about timelines, affordability, and the probability that your child may not make the inclusion cut.
The industry’s unrequited love story
Capital may flow to platforms that promise scale, not to ultra-rare programmes that demonstrably save lives but defy spreadsheet logic. Until we align payment models and regulatory pathways with clinical value delivered, scientific breakthroughs will continue to outrun commercial reality. Musunuru himself flagged the need for shared reagent banks and harmonised analytics to lower entry costs for single-patient INDs.
Where Lonrú sees light
Platform-ready modular CMC dossiers - codify repeatable elements so every next bespoke therapy starts at 60 % completion.
Outcome-based annuity payments - spread the >$1 M sticker over durability, with claw-backs for under-performance.
Public-interest manufacturing co-ops - echoing Rarity’s public benefit corporation, pool idle academic clean-room capacity for ultra-rare runs.
Digital evidence commons - capture long-term real-world data to sharpen cost-effectiveness arguments and feed adaptive pricing.
Treading softly, but moving forward
Yeats’ plea is our directive: each dataset, each funding decision, each splashy headline lands on someone’s dream. Let’s celebrate KJ’s reprieve without overselling affordability and timelines for every rare disease. Let’s channel the excitement into structural fixes that let breakthrough science and sustainable business models grow in tandem.
At Lonrú, illuminating that path, from innovation to equitable impact is our raison d’être. We invite partners who share this conviction to walk with us, softly but determinedly.
- written by Lonrú Consulting’s Principal and Founder, Caoimhe Nic An tSaoir, PhD MBA
Illuminate your next step. Reach out to explore Lonrú’s VantagePoint™ frameworks.
How patient-led business models are accelerating the next wave of cell & gene therapies
This week’s ASGCT kicked off with a half-day workshop that put patient advocacy organisations (PAOs) centre-stage: “The Business of Advocates Advancing CGTs.” The line-up showcased six distinct financing and operating models that rare-disease groups are already using to push novel therapies from idea-to-IND and, in a few cases, to market.
Why does Lonrú care? Because every one of these blueprints re-writes the traditional value chain, creating new touch-points where enabling-technology providers can plug in and add velocity. Below is our take on what’s working, where the white-space lies, and how the field can move faster—together.
1. Venture Philanthropy: recycling capital, de-risking pipelines
PAOs such as EB Research Partnership have proven you can pair philanthropy with an equity stake and then redeploy the upside into the next programme. Capital recycling has already generated multi-billion-dollar returns in cystic fibrosis and is now fuelling gene-therapy approvals.
Lonrú lens: This model needs sharp diligence on equity, royalty waterfalls and post-marketing obligations—areas where our VantagePoint™ Scale framework routinely stress-tests cash-flow projections and pricing scenarios.
2. “Virtual Biotech” & Parent-Run INDs: lean, data-rich, execution-focused
FOXG1 and Cure Rare Disease keep IP and the IND inside the foundation, while outsourcing CMC and clinical operations. With <$10 million they can marshal CROs, CDMOs and AI-assisted registries to reach first-in-human trials.
Lonrú lens: An AI-enabled, gap-analysis tool can shave months off regulatory prep and keep lean teams focused on go/no-go inflection points.
3. Social-Purpose Corporations (SPCs): mission lock with investment flexibility
Elpida Therapeutics channels philanthropic and venture dollars into a public-benefit corp, ring-fencing mission while still courting mainstream capital.
Lonrú lens: Governance complexity is real, but SPCs open a route for ESG-oriented investors looking for measurable patient impact alongside returns—a sweet spot for differentiated analytics and storytelling.
4. Hospital-Held Licences: academic GMP meets cost-recovery access
Great Ormond Street is preparing to license and manufacture an ADA-SCID therapy abandoned by industry, aiming for cost-price delivery within the NHS.
Lonrú lens: When sponsors exit, institution-led rescue pathways preserve clinical know-how and datasets. They also create demand for modular QC, batch-release and real-world-evidence solutions that technology vendors can supply at scale.
5. Capacity-Building Grants (CZI Rare-as-One, PCORI): seeding data infrastructure
Five-year grants of up to $2 million are giving fledgling groups governance training, registry tooling and DEI mandates that de-risk later venture rounds.
Lonrú lens: Data is the currency. Offering in-kind AI analytics or registry architecture can be a strategic “give-to-get” for service providers aiming to become embedded long before commercial inflection.
6. Event- & Media-Driven Revenues: community engagement that pays for R&D
From $10 million reality-show prizes to cross-country endurance events, creative fundraising is underwriting natural-history studies and AAV vector work.
Lonrú lens: Volatile by nature, but high-visibility campaigns drive brand equity and patient-reported outcomes—alongside rich datasets that feed development models.
Where patient advocacy becomes the catalyst for next-gen cell & gene therapies.
Three strategic signals for the road ahead
Capital efficiency matters more than capital volume. Each archetype hinges on agile, data-guided decision gates—exactly where AI-powered market and regulatory intelligence can compress timelines and costs.
Ownership of IND-critical data is becoming the bargaining chip. Whether a PAO holds equity, an SPC licence, or a hospital marketing authorisation, control of natural-history and CMC datasets dictates partnering leverage.
Ecosystem players that illuminate complexity will win. The most successful advocates are not just funding experiments; they’re curating information—registries, biobanks, outcome measures—now ripe for advanced analytics and digital-first collaboration.
Where Lonrú Fits
Our mission is to accelerate growth for the technologies that enable CGT breakthroughs. The alternative models highlighted at ASGCT open multiple entry points for our clients:
VantagePoint™ Insights to benchmark emerging PAO pipelines against partner demand.
VantagePoint™ Validation to design capital-efficient tech-transfer and CMC roadmaps that suit lean, virtual structures.
VantagePoint™ Connect to matchmake between mission-driven funds, hospital GMP suites and enabling-tech innovators.
Illuminating the route from patient-driven discovery to scalable market success.
Ready to illuminate your next move? Let’s talk at ASGCT in New Orleans—or book a virtual debrief post-meeting.
Through the Lonrú Lens – What to Watch at ASGCT 2025
The American Society of Gene & Cell Therapy’s annual meeting is always a bell-wether for where the field is heading, and this year’s New Orleans programme is the biggest yet. Our data-driven sweep of the abstracts surface five story-lines worth tracking – plus a free interactive dashboard to help you cut through the noise once you arrive in NOLA.
1. 2,196 reasons to plan your diary
2,196 presentations spread over five days – workshops, orals, symposia, posters and fireside chats.
1,925 individual speakers from 1,094 organisations.
Poster-heavy: ~67 % of all slots.
Take-away → the sheer scale demands a smart filtering strategy.
2. AAV still wears the crown – but the court is changing
Below is a quick-glance infographic of the five strongest themes surging through the agenda:
General gene therapy & AAV delivery remains the heavyweight, anchoring ≈ 640 talks that probe tropism, PK/PD and clinical translation.
CAR-T and other engineered T-cells follow with just over 320 sessions, reflecting the field’s pivot toward solid-tumour applications and off-the-shelf platforms.
AAV production & manufacturing clocks in at ≈ 250 presentations, proof that CMC and scale-up have moved from backstage to centre stage.
LNP–mRNA delivery—once a niche—now commands nearly 90 slots, signalling non-viral vectors’ mainstream arrival.
And AAV capsid engineering for CNS rounds out the leaderboard with ≈ 80 talks, underscoring the race to breach the blood-brain barrier.
Talk Hotspots: Top 5 Topics at ASGCT 2025
3. Gene editing graduates from “emerging” to essential
Editing-related abstracts almost double year-on-year (122 talks). While Cas9 platform work still leads, prime/base editing, compact nucleases and first-in-human in-vivo data are pushing the field from tool-building towards translation.
4. Who’s punching above their weight?
Academia still owns the podium – UMass Chan, Duke, Minnesota, Fred Hutch – yet tool suppliers such as Thermo Fisher and Millipore Sigma climb the rankings with manufacturing and analytics content. Corporate voices are broad but not yet dominant: the top-20 companies represent <15 % of the agenda.
5. What narratives will shape corridor talk?
Below is a visual snapshot of the four “big-picture” narratives we believe will dominate the hallway chatter in New Orleans. Use it as a quick mental map, then scroll on for the deeper dive in our interactive tool.
Manufacturing Moves Mainstream – nearly 250 abstracts tackle capacity, process intensification and analytics, reflecting the sector’s new clinical bottleneck.
Delivery Diversification – with ~90 LNP-mRNA talks and 80+ capsid-CNS sessions, non-viral and next-gen viral vectors are hedging against single-platform limits.
Precision Editing Surges – the fastest-growing cluster at 120+ presentations, spanning prime/base editors, compact nucleases and first-in-human data.
Oncology Evolves – more than 320 engineered-cell abstracts showcase armoured/allo CAR-Ts, NK and macrophage approaches pushing beyond haematologic malignancies.
Together, they frame the strategic questions investors and BD teams will be asking long after the plenaries end.
Four Trends Shaping ASGCT 2025
Meet us on-site & try our free scheduling tool
Our founder, Dr Caoimhe Nic An tSaoir, will be on the ground all week – connect with her to discuss how Lonrú’s data-first approach can illuminate your business decisions.
To make those decisions easier, we’re sharing a complimentary interactive dashboard:
Search the entire programme by topic cluster, speaker or organisation.
Visualise busy time-blocks to avoid clashes.
Zero in on your niche – whether it’s capsid engineering, LNP formulation or clinical trial design.
It’s the fastest way to find your “must-see” talks amid 2k+ abstracts – and it’s yours, free, courtesy of Lonrú.
👉 Try the dashboard below and bookmark it before you pack your bags.
Why talk to Lonrú?
If you’re an enabling-technology or tools company, our VantagePoint™ suite turns noise into strategic clarity: market intelligence, validation road-maps, go-to-market acceleration and investor engagement – all backed by AI/ML analytics. Let’s discuss how we can help you stand out long after the convention centre lights dim.
Meeting on the Med: a pulse check on CGT
The Alliance for Regenerative Medicine’s Meeting on the Med brought therapeutic developers, regulators, investors, manufacturers and technology vendors together for three intense days of debate and deal‑making in Rome. From main‑stage keynotes by EMA Executive Director Emer Cooke to packed break‑outs on automation and payment models, the conference offered a clear signal: 2025 is the year CGT moves from pilot phase to industrial scale.
Below are five high‑level themes that dominated the agenda, paired with practical ways Lonrú Consulting can help stakeholders translate talk into traction.
Dr Caoimhe Nic An tSaoir, PhD MBA representing Lonrú Consulting at its first ARM Meeting on the Med—illuminating innovation in Rome.
Industrialisation & Cost‑of‑Goods: from artisan to assembly line
Conference focus
Speakers from Cellares, ScaleReady and SmartCella touted up to 10× productivity gains with closed, automated systems, while plenary panels urged “robotics, AI and digitalisation” to improve labour hours.
Why timing matters – Conference veterans warned: “Don’t bolt robots onto a sub-optimal process.” The smart sequence is:
Close & automate the core workflow to drive consistency.
Iterate and optimise based on fresh, high-fidelity data.
Roboticise only when the process is lean enough to justify the CapEx.
Where Lonrú helps
VantagePoint™ Scale models the true economics of automated vs. manual workflows—integrating batch size, labour inputs and capital depreciation—so equipment vendors and therapy developers can make evidence‑based CapEx decisions.
2. Regulatory Convergence: alignment gives way to harmonisation
Conference focus
EMA, MHRA, AIFA and other agencies previewed a joint dossier framework for ATMPs, signalling a shift from bilateral “alignment” to multilateral convergence in 2025‑26. Under this model, one core CMC/clinical package could be reviewed simultaneously by several regulators, trimming duplicate studies and bringing therapies to multiple markets months sooner.
Where Lonrú helps
VantagePoint™ Insights offers a policy tracker that maps emerging common modules, review timelines and regional inflection dates —allowing CGT technology suppliers to support submissions and market launches with confidence.
3. Financing Climate: capital comes with a data‑driven thesis
Conference focus
Investors are showing signs of optimism that deal flow will rebound in H2 2025, but before cheques are written they want proof of differentiated tech, clear ROI and capital‑efficient scale‑up paths. Capital will flow first to platforms able to quantify near-term revenue and manufacturing economics.
Where Lonrú helps
VantagePoint™ Accelerate packages market sizing, NPV/DCF modelling and competitive white‑space analysis into investor‑ready narratives—helping technology providers translate ground-breaking science into a thoroughly de‑risked business case.
4. Ethics, Access & Novel Payment Models: transparency is non‑negotiable
Conference focus
Panels on ethics urged more openness around cost of goods, while payers showcased staged‑payment and subscription pilots to manage six‑figure therapy costs. A shared takeaway emerged: sustained access depends on communicating with transparency and demonstrating tangible value—tailored to regulators, payers, clinicians, and patient communities alike.
Where Lonrú helps
VantagePoint™ Illuminate distils complex value propositions and pricing strategies into clear, stakeholder‑specific messaging—supporting technology firms as they negotiate value‑based contracts and build public trust.
5. Emerging Geographies: the Gulf states step onto the CGT stage
Conference focus
A dedicated session spotlighted UAE and KSA initiatives to build GMP capacity and local innovation hubs, with regional investors actively scouting technology partners. Speakers emphasised the opportunity; whoever locks in early reference sites now will shape standards for a market that is forecast to treat tens of thousands of regional patients by 2030.
Where Lonrú helps
VantagePoint™ Connect maps innovation clusters, stakeholder networks and funding programmes in any target geography, then brokers introductions for enabling-technology providers to regulators, hospital networks, strategic investors and channel partners—giving first movers the inside track on commercial pilots and policy dialogues.
The takeaway
The ARM meeting in Rome confirmed that scale, regulatory convergence, and credible economics are now the gating factors for progress in CGT—and it is the enabling-technology companies who can unlock every one of those gates. Lonrú’s mission to Accelerate Growth of Cell and Gene Therapy Technology Companies — exists precisely to drive momentum at this inflection point.
Whether you’re rolling out an automated manufacturing platform, launching next-gen analytical tooling, or scaling a digital batch-record system, Lonrú turns fresh industry insights into practical roadmaps—combining market intelligence, stakeholder alignment, and financial modelling so breakthrough technologies reach industrial impact sooner. Ready to view your CGT strategy from a higher vantage point? Let’s talk.
Unlocking Cell and Gene Therapies: Introducing VantagePoint™ – Lonrú Consulting’s Suite of Solutions For Growth
As cell and gene therapies (CGT) continue to revolutionize medicine, enabling technology companies are increasingly becoming a key pillar of progress. These companies develop critical platforms, tools, and technologies that facilitate the scalable, cost-effective, and precise production and delivery of groundbreaking treatments. At Lonrú Consulting, we understand that these enabling technologies are more than just support systems—they form part of the vital infrastructure required to democratize access to advanced therapies globally.
That’s why we’ve developed VantagePoint™, a comprehensive suite of solutions carefully crafted to propel enabling technology companies at every stage of their growth journey. VantagePoint™ encapsulates our deep industry expertise and advanced AI-driven analytics into a unified approach, uniquely positioning our clients to overcome challenges, identify opportunities, and accelerate commercialization within the dynamic CGT landscape.
Lonrú Consulting’s VantagePoint™: A Unified Suite of AI-powered Solutions Propelling Enabling Technology Companies at Every Stage of Growth.
The VantagePoint™ Advantage
VantagePoint™ addresses the fundamental areas critical to the growth and scalability of enabling technology providers:
VantagePoint™ Insights – Strategic market intelligence that provides real-time, actionable clarity, empowering your company to anticipate market trends, understand competitive landscapes, and make confident, informed decisions.
VantagePoint™ Validation – Strategic technology validation and partnering solutions, helping emerging technology providers navigate complexity with confidence, secure robust partnerships, and validate innovations strategically within the competitive CGT market.
VantagePoint™ Accelerate – Precision-driven go-to-market and business development strategies designed to fast-track commercialization, optimize financial planning, and accelerate market adoption, ensuring that innovative technologies reach the patients who need them, faster.
VantagePoint™ Illuminate – Tailored strategic positioning and messaging that clearly articulates your technology’s unique value, enhancing brand visibility, and effectively engaging stakeholders, from investors to industry partners.
VantagePoint™ Connect – Strategic stakeholder engagement services, leveraging deep market connections and relationship-building frameworks to align enabling technology companies with key translational networks, biotech firms, investors, patient advocates and industry leaders for impactful collaboration and accelerated growth.
VantagePoint™ Scale – Comprehensive commercial feasibility and strategic scaling support, delivering robust pricing strategies, detailed market entry roadmaps, and scalable growth frameworks that ensure sustainable, long-term commercial success.
Enabling Technologies: The Key to Democratizing Advanced Therapies
By optimizing the reach and placement of innovative enabling technologies, Lonrú Consulting helps to democratize the transformative potential of cell and gene therapies. These technologies significantly reduce costs, improve accessibility, and enhance treatment precision, ultimately allowing more patients across diverse geographic and economic contexts to benefit from cutting-edge treatments.
Lonrú Consulting: Your Partner in Transforming CGT
Lonrú Consulting was founded on the vision of illuminating opportunities within the CGT industry. With over 15 years of global experience in the life sciences sector, Lonrú Consulting, led by Dr. Caoimhe Nic An tSaoir, integrates scientific rigor with strategic acumen, uniquely positioning us to support enabling technology companies effectively.
We have specifically tailored our expertise to empower technology providers, understanding that their success directly translates into broader patient access to life-changing therapies. Through VantagePoint™, we provide clarity, strategic insight, and precise execution, driving your company's success while catalyzing the industry’s evolution.
At Lonrú, we believe the future of cell and gene therapy depends heavily on innovation in enabling technologies. Together, let’s illuminate the route to viable transformative therapies.
Let us illuminate your business—contact us to discover how the VantagePoint™ suite can accelerate your journey in cell and gene therapy.
Navigating Strategic Partnerships: How Platform Technology Providers Can Overcome Validation Challenges
At last week's Advanced Therapies UK meeting, one theme resonated clearly among platform technology companies supporting cell and gene therapy (CGT) production: the complex yet essential role of partnerships in validating their technologies. These strategic collaborations are critical, yet they often present significant hurdles, from scoping and initial engagement to execution.
Companies face multiple challenges when entering third-party partnerships. Firstly, the precise scoping of collaborations often proves difficult, leading to misaligned expectations and inefficient use of resources. Secondly, engaging suitable partners involves navigating complex negotiations and managing expectations around technology validation and intellectual property. Finally, executing these partnerships demands rigorous project management and clear, constant communication—areas where resource constraints frequently limit effectiveness.
Why are these partnerships vital? Successful validation through third-party collaborations provides crucial credibility, enhancing market trust and significantly boosting investor confidence. Moreover, strategic partnerships open doors to larger networks and new commercial opportunities, accelerating technology adoption and growth.
However, for many enabling technology companies, internal teams are deeply entrenched in R&D, fundraising efforts, or focused on sales execution and revenue generation, often leaving strategic partnership initiatives underserved. This is precisely where Lonrú Consulting steps in.
Launching Lonrú's VantagePoint™ Validation Platform: Illuminating strategic clarity for enabling technology companies in Cell & Gene Therapy.
At Lonrú, we specialize in illuminating critical strategic partnership needs. Our structured approach offers technology providers unparalleled visibility, strategic insight, and clarity when validating their enabling technologies. From clearly defined validation objectives and partner alignment to meticulous execution and impactful outcomes, our VantagePoint™ Validation platform ensures strategic clarity and precision in every partnership.
Explore more about our VantagePoint™ Validation platform and strategic partnership services here.
If you're an enabling technology company underpinning cell and gene therapy production or delivery, it's time to explore how strategic outsourcing can accelerate your journey. Let's illuminate your pathway to validation together.
Ready to discuss your strategic partnership needs? Connect with Lonrú Consulting today and let us help you turn your validation challenges into powerful opportunities.