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

1. 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.

2. 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.

3. 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.

4. 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.

5. 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

1. Platform-ready modular CMC dossiers – codify repeatable elements so every next bespoke therapy starts at 60 % completion.

2. Outcome-based annuity payments – spread the >$1 M sticker over durability, with claw-backs for under-performance.

3. Public-interest manufacturing co-ops – echoing Rarity’s public benefit corporation, pool idle academic clean-room capacity for ultra-rare runs.

4. 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

1. 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.
   

2. 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.

3. 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.

⤢ Full Screen

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.

1. 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:

1. Close & automate the core workflow to drive consistency.

2. Iterate and optimise based on fresh, high-fidelity data.

3. 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.

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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.

Catalysing the COGs Revolution: The Role of Enabling Technologies in CGT

Lonrú is at Advanced Therapies UK this week and day one discussions highlighted emerging trends, groundbreaking innovations, and pressing challenges within the cell and gene therapy (CGT) landscape, placing a strong emphasis on enabling technologies and their critical role in addressing one of the industry's biggest hurdles—the Cost of Goods (COGs).

Enabling technology providers are emerging as key catalysts capable of significantly reducing COGs, addressing one of the industry's primary barriers to patient access. The keynote panel underscored how high manufacturing costs often prevent eligible patients from receiving approved therapies due to regional payer reluctance, emphasizing the urgent need for cost-effective solutions—precisely the type of innovation Lonrú champions.

A particularly compelling panel discussion, "Academic Translation: Advancing Cutting Edge Technologies," chaired by Lara Campana, from Resolution Therapeutics (a University of Edinburgh Spinout) delved deeper into the challenges faced by technology founders in establishing meaningful collaborations. When Lonrú posed the critical question, "Looking back, where do you see the biggest missed opportunities for enabling technologies or partnerships to accelerate development—and what would you do differently?" panelists candidly discussed the difficulties their teams encountered in demonstrating proof of concept of their technologies and managing complex industry collaborations, IP ownership, and partnership structuring.

These insights resonate strongly with Lonrú's expertise. We specialize in identifying, cultivating, and establishing strategic collaborations, effectively bridging the gap between academic innovation and commercial execution. Our tailored approach and advanced analytics help enabling technology providers navigate complexities, optimize commercialization pathways, and significantly lower COGs.

As the industry progresses, the imperative is clear: innovation must be paired with strategic partnerships to achieve tangible cost reductions and improve patient accessibility worldwide.

If you're ready to leverage your enabling technology for maximum impact, Lonrú is here to illuminate your path forward.

Illuminating CRISPR Clinical Trial Progress: A Lonrú Lens for Enabling Tools Providers

A recent Keystone meeting showcased a multitude of CRISPR-based clinical trials, underscoring the tremendous growth and development in the field. As excitement around CRISPR therapies intensifies, enabling tools providers—ranging from assay developers to manufacturing technology companies—are uniquely positioned to support these therapeutic programs in critical ways. In this post, we take a close look at some of the latest CRISPR clinical trial updates (therapeutic applications only) and introduce Lonrú Consulting’s new public dashboard designed to help enabling technology companies recognise emerging needs and opportunities.

CRISPR Therapeutics: What’s New from Keystone?

This year’s Precision Genome Engineering Keystone meeting confirmed that CRISPR-based therapies have made considerable strides, with an approved therapy now commercially launched and an expanded pool of proof-of-concept data and early clinical safety readouts for new indications. Key highlights include:

Emerging Pipeline Diversity

• While oncology and monogenic hematological diseases continue to account for the majority of CRISPR clinical trials, diversity of clinical trial categories is expanding to include neurological disorders and metabolic diseases.

• This broadening pipeline hints at a growing need for specialized vectors, delivery systems, and lab instrumentation that can facilitate gene editing across a wider range of cell types and patient populations.

Focus on Delivery and Efficiency

• Many panels spotlighted the importance of refining both ex vivo and in vivo delivery modalities. This shift underscores the demand for robust viral and non-viral delivery tools, high-fidelity nucleases, and scalable manufacturing platforms—all areas where enabling technology providers can shine.

Quality and Regulatory Considerations

• As CRISPR programs move through clinical phases, compliance with evolving global regulatory standards has become paramount. Tool providers offering GMP-ready reagents, automated QC processes, and validated assay kits stand to gain traction as sponsors seek to streamline their clinical workflows from pre-clinical development to clinical manufacture.

Why It Matters for Enabling Tools Providers

CRISPR sponsors rely on high-performance instrumentation, reagents, software, and manufacturing technologies to maintain clinical momentum. From single-cell analysis platforms that verify gene edits to advanced vector packaging systems, every stage of CRISPR therapy development demands specialized support.

• Assay & Analytics
  Tool developers can build or adapt products that precisely predict or measure gene-editing efficiency, off-target effects, and cell viability. Demand continues to grow for analytical solutions that can scale from early R&D to late-phase GMP settings.

• Automation & Scale-Up
  As more candidates progress into mid- and late-stage trials, sponsors will need integrated, automated workflows—particularly for ex vivo engineering of immune cells and beyond. Vendors who provide modular, automated solutions can help sponsors reduce costs and improve reproducibility.

• Regulatory-Grade Toolkits
  Enhanced guidelines around gene editing are emerging. GMP-certified reagents, standardized protocols, and robust documentation platforms offer sponsors peace of mind and help expedite regulatory approvals.

Introducing Lonrú’s CRISPR Clinical Trial Dashboard

In line with our mission to help technology providers identify strategic opportunities, Lonrú Consulting is releasing our new, publicly accessible CRISPR Clinical Trial Dashboard below and on our interactive dashboards page. This interactive database lets you:

• Browse Active Sponsors and Investigational Targets
  Instantly see which companies are leading in oncology, rare diseases, or other emerging applications—to map those areas back to their specialized tool needs.

• Filter by Phase, Status, and Indication
  Determine where your tools and technologies can fit best based on trial phase (e.g., early-phase for novel assays vs. near-market for scalable manufacturing solutions).

• Pinpoint Potential Partnerships
  Identify top CRISPR sponsors that may be seeking next-gen delivery methods, automation solutions, or advanced analytics to complement their therapeutic pipelines.

By consolidating and visualizing information on CRISPR therapeutic trials, we aim to give enabling tools providers an easy way to track potential leads, time new product launches, and tailor their solutions to fast-evolving sponsor needs.

How Enabling Tools Providers Can Use Lonrú’s Dashboards

1. Market Positioning

   • Understand which clinical areas (e.g., B-cell malignancies, sickle cell disease) are seeing the greatest CRISPR activity, and tailor your product roadmap or marketing accordingly.

2. Targeted Business Development

   • Pinpoint sponsors that match your capabilities—whether you offer reagents optimized for allogeneic cell therapies or automation equipment ideal for large-scale gene editing.

3. Competitive Benchmarking

   • Track your peers (other platform and tool vendors) to see how they are positioning their products across varying therapeutic trials. Use these insights to refine your messaging or to guide strategic investments in R&D.

4. Regulatory & Compliance Insights

   • Monitor how trials across different geographies progress through regulatory milestones. Align your quality management systems and documentation practices to meet sponsor expectations in each region.

Closing Reflections

Keystone illuminated just how quickly CRISPR therapeutics are evolving. For enabling tools providers, each new clinical milestone signals a rising need for more specialized, reliable, and scalable products. With sponsors juggling multiple priorities—speed, safety, cost efficiency, and regulatory compliance—there’s never been a better time to cement your role as a crucial partner in the CRISPR value chain.

Lonrú Consulting invites you to explore our dashboard and harness these data-driven insights. If you’d like to learn more about how we can help you position your technologies or amplify your market impact, we’re here to illuminate the path forward.

Ready to dive in?

Customize our CRISPR Clinical Trial Dashboard to uncover key trends and potential partnerships. Reach out at info@lonruconsulting.com or visit Lonrú Consulting to learn how our data analytics and strategic advisory can illuminate your next big opportunity.

At Lonrú Consulting, we believe in empowering the technologies behind the therapies. Together, let’s unlock the full potential of CRISPR and continue shaping the future of genomic medicine.

Bridging the Gap—Enabling Technology Providers & the Path to the Clinic

Lonrú is attending the Precision Genome Engineering: Translating the Human Genome to the Clinic meeting in Killarney this week. The meeting brings together the leading minds in gene editing, delivery systems, and therapeutic development to discuss the latest advancements shaping the future of precision medicine. From new CRISPR-based tools and enabling technologies to the challenges of translating innovation into the clinic, the conference highlights the critical steps needed to move from discovery to impact. Our poster presentation yesterday on streamlining preclinical-to-IND pathways generated interest, not just from therapeutic developers, but also from enabling technology providers eager to gain a clearer view of the challenges their partners and customers face in clinical translation.

Why Are Enabling Technology Providers Paying Attention?

While much of the conversation around gene editing focuses on therapeutic breakthroughs, every successful CRISPR-based therapy depends on the enabling technologies that support it—from delivery systems and genome engineering platforms to advanced analytical tools and scalable manufacturing solutions. Many enabling technology providers recognize that to best serve their customers, they need a deeper understanding of:

✔ Regulatory bottlenecks that delay clinical progression and impact product adoption.
✔ Common reasons for IND holds, including safety concerns, CMC issues, and assay validation gaps.
✔ Preclinical validation requirements that define what data therapeutic developers need to move forward.

By better aligning their solutions with these pain points, enabling technology providers can position themselves as indispensable partners in the journey from discovery to the clinic.

Beyond the Poster: Full Report & Decision Trees

Attendees we met with appreciated having access to our full report to dive deeper into the topics covered in the poster. In particular, the decision tree framework resonated with both therapeutic and technology developers, offering a structured approach to mitigating risks early in the development process.

For those who couldn’t attend the poster session—or who want to explore the findings in more detail—you can view and download the full report, poster, and decision trees here:

🔗 Click here to access the materials

Lonrú’s full report, poster and decision tree framework are available for download.

The conversation doesn’t end here—if you’re an enabling technology provider looking to bridge the gap between research and clinical success, let’s connect. Lonrú Consulting specializes in strategic insights that help align enabling technologies with the needs of therapeutic developers, ensuring that innovation translates into impact.

Lonrú at Keystone: Precision Genome Engineering and the Future of CRISPR Clinical Translation

The upcoming Keystone Symposium on Precision Genome Engineering: Translating the Human Genome to the Clinic (March 3-6, 2025, in Killarney, Ireland) presents an exciting opportunity for the field of gene editing to converge on the critical scientific and regulatory advancements necessary for therapeutic translation. As a firm dedicated to empowering technology providers in the Cell and Gene Therapy (CGT) sector, Lonrú Consulting is eager to engage in discussions that address key industry challenges and pathways for accelerating CRISPR-based therapeutic approvals.

What We’re Looking Forward To

The Keystone agenda is packed with high-impact sessions, and several key themes stand out as particularly relevant for tool providers and regulatory strategists:

Regulatory Innovation and Harmonization to Expand Access to Precision Gene Therapies

The evolution of global regulatory frameworks remains one of the largest hurdles in transitioning CRISPR therapies from preclinical research to clinical implementation. We are particularly interested in sessions that discuss strategies to streamline Investigational New Drug (IND) and Clinical Trial Application (CTA) submissions, as we consider how tools providers may help therapeutic developers in this space.

From Target to IND: Preclinical Studies for ex vivo and in vivo Therapies

With the rise of in vivo genome-editing therapies alongside more established ex vivo approaches, ensuring safety and efficacy through robust preclinical models is paramount. Lonrú is keen to explore discussions on off-target risk mitigation, potency assays, and long-term safety assessments.

Genome Engineering in the Clinic: Advances in Therapeutic Applications

As CRISPR-based therapies move into human trials, the interplay between genome engineering technologies and clinical strategy becomes increasingly complex. We anticipate valuable insights into how industry leaders are addressing patient risk management, staggered dosing strategies, and immune response monitoring.

Lonrú’s Contribution: Bridging the Gap Between Preclinical and Clinical Translation

Lonrú’s poster evaluates global regulatory trends and identifies common pitfalls in IND/CTA submissions that lead to clinical holds and delays, we also introduce our decision tree framework, a tool to help developers navigate common pitfalls.

At Keystone, Lonrú will present our poster, “Bridging the Gap: Streamlining Preclinical to Clinical Trial Applications (IND/CTA) for CRISPR Therapies.” This work evaluates global regulatory trends and identifies common pitfalls in IND/CTA submissions that lead to clinical holds and delays.

Our analysis emphasizes four strategic imperatives:

• Early engagement with regulators to preemptively address potential concerns.

• Enhanced off-target validation to minimize safety risks.

• Optimized Chemistry, Manufacturing, and Controls (CMC) readiness to ensure scalable production.

• Parallel global regulatory filings to accelerate approvals across different jurisdictions.

To support these strategies, we are unveiling our Decision-Tree Framework, a structured tool designed to help developers de-risk their preclinical-to-clinical transitions. This framework is segmented into three core areas:

• Preclinical & Safety Considerations (off-target assessment, long-term safety studies, in vivo vs. ex vivo risk management)

• CMC & Manufacturing Readiness (potency assay validation, lot-to-lot consistency, delivery system characterization)

• Regulatory & Trial Design Strategy (harmonization of submission dossiers, risk mitigation planning, patient safety protocols)

Post-Conference Insights & Resources

Following the Keystone meeting, Lonrú will provide a comprehensive conference report, including key takeaways from the most impactful sessions and commentary on emerging industry trends.

Additionally, our Decision-Tree Framework and poster will be made available for download, offering a valuable resource for CRISPR developers seeking to refine their IND/CTA strategy.

Bridging the Gap: Streamlining Preclinical to Clinical Trial Applications (IND/CTA) for CRISPR Therapies

We are thrilled to present our poster at Keystone, showcasing key insights into regulatory readiness, risk mitigation, and translational strategies. Proudly featuring insights from Lonrú Consulting and Johns Hopkins Carey Business School, our work highlights data-driven approaches to accelerating CRISPR-based therapeutic development.

For those attending Keystone, we welcome discussions on how Lonrú can support technology providers in navigating regulatory complexities and accelerating the path to clinic.

If you are interested in receiving any of the resources outlined here, please complete the form below and we’ll be in touch after the conference.

How the Focus of Cell and Gene Clinical Trials in the UK is Shifting

The UK remains a global hub for cell and gene therapy (CGT) innovation, but where is the field heading? What indications are seeing the most investment? How are sponsors adapting their pipelines?

At Lonrú Consulting, we apply data-driven insights to illuminate these questions, helping technology providers and innovators navigate the evolving CGT landscape. Our latest analysis of UK CGT clinical trials (2010–2024) highlights shifting therapeutic priorities, changing sponsor dynamics, and emerging areas of opportunity.

A Recent Decline in CGT Clinical Trials

While the UK has maintained strong CGT activity over the past decade, 2024 saw a decline in new trial initiations. A total of 26 new trials were initiated in 2024, compared to 31 in 2023, 32 in 2022, and 28 in 2021. This decline reflects several industry-wide challenges, including regulatory complexities that extend trial approval timelines, funding constraints that particularly affect early-stage biotech companies, and a strategic shift among sponsors toward refining pipelines post-pandemic.

Although this slowdown is worth monitoring, it may also indicate a shift in the field, with a move toward high-value, well-validated programs rather than a focus on sheer trial volume. Understanding how companies are adapting to this changing landscape is essential for stakeholders in the CGT ecosystem.

Explore Lonrú’s interactive dashboard examples to view UK CGT trial trends for further insights.

A Shifting Sponsor Landscape

The mix of sponsors in UK CGT trials is evolving. Large pharmaceutical companies, such as AbbVie and Novartis, are increasing their presence, signaling long-term investment in CGT. UK-based biotech firms remain active in driving first-in-human trials, though some, like Achilles Therapeutics, have recently pivoted away from clinical-stage programs. Academic institutions such as University College London (UCL) continue to play a critical role in early-stage innovation, particularly in rare diseases and regenerative medicine.

This diverse sponsor base reflects both the increasing commercial interest in CGT and the persistent role of academic centers in developing new approaches. The dynamic between large pharma, biotech startups, and research institutions will be key to shaping the next phase of clinical development.

The Evolution of Indications in CGT Trials

By categorizing CGT clinical trials into broader therapeutic areas, we can see shifts in industry focus.

Oncology remains the dominant area for CGT trials, but the landscape is evolving. Hematologic cancers, including multiple myeloma, leukemia, and lymphoma, continue to drive significant trial activity, largely fueled by CAR-T and other engineered cell therapies. Solid tumors, such as lung cancer, liver cancer, and glioblastoma, remain a major focus, though the pace of new trial initiation has been more gradual. Adoptive cell therapies, including tumor-infiltrating lymphocytes (TILs) and engineered T-cell receptors (TCRs), are emerging as alternatives to traditional CAR-T approaches, offering new avenues for immunotherapy.

Rare and genetic disorders have gained significant momentum in CGT research. Duchenne muscular dystrophy, cystic fibrosis, and spinal muscular atrophy have all seen increased trial activity in recent years. Many of these trials leverage gene therapy and gene editing approaches, reflecting industry confidence in in vivo genetic correction as a viable treatment strategy.

Neurological disorders are an emerging frontier for CGT. While historically underrepresented in CGT trials, conditions such as ALS, Parkinson’s disease, and Huntington’s disease are now receiving increased investment. Challenges remain in delivering therapies effectively across the blood-brain barrier, but clinical interest in gene therapies for neurodegenerative and neuromuscular conditions continues to rise.

Ophthalmology has consistently been a growth area in CGT. Retinal diseases, including retinitis pigmentosa and Leber’s congenital amaurosis, are among the top indications for AAV-based gene therapies. These conditions are well-suited for local gene delivery, reducing systemic risks while improving treatment precision.

Hematological conditions outside of cancer, such as sickle cell disease and hemophilia, continue to attract CGT investment. However, with recent regulatory approvals such as Casgevy for ex vivo gene editing in sickle cell disease, the number of new clinical trials in these areas may moderate over time as companies transition toward commercialization.

Strategic Takeaways for CGT Stakeholders

Understanding these trends is essential for stakeholders across the CGT ecosystem. Enabling technology providers, Biotech and pharmaceutical companies should align their pipeline strategies with the shift beyond oncology into neurology, ophthalmology, and rare diseases, where CGT applications are growing. Technology providers and contract development and manufacturing organizations (CDMOs) must adapt to an evolving landscape where scalable allogeneic cell therapies, in vivo gene editing, and AAV-based delivery are gaining prominence. Investors should look beyond oncology, as emerging CGT applications in neurological and metabolic disorders present new opportunities for value creation.

As the industry moves forward, the ability to navigate these macro trends will be critical for companies developing platforms and enabling technologies. Lonrú Consulting provides real-time data analytics, market intelligence, and commercialization strategies to help companies position themselves effectively in a competitive and evolving field.

The Critical Role of Academic Translational Centers in Advancing Cell and Gene Therapies

As the field of cell and gene therapy (CGT) matures, academic translational centers remain indispensable in driving innovation and bridging the gap between early research and clinical application. Recent efforts in the UK, Japan, Australia, Spain, the USA, Germany, and Ireland illustrate how dedicated translational frameworks, such as the RISE model, are shaping sustainable CGT ecosystems (Naresh et al., 2025). However, these efforts are now facing significant financial headwinds, in the United States, where drastic cuts to NIH indirect funding threaten to undercut decades of translational research momentum.

The RISE Framework: A Global Lens on CGT Translation

The RISE model (Resource sharing, Interdisciplinary collaboration, Sustainable financial models, and Education) underscores the importance of structuring academic environments to support the full translational pipeline of CGTs (Naresh et al., 2025). This approach is evident in:

• United Kingdom: Institutions like UCL lead CGT innovation, leveraging partnerships with the Cell & Gene Therapy Catapult to scale new therapies. Lonrú’s interactive dashboard highlights how UK-based academic centers are leading in CGT trial sponsorship and development phases (Lonrú Consulting, 2025).

• Japan: The PMDA's progressive regulatory framework, coupled with translational research centers, has propelled Japan to the forefront of regenerative medicine.

• Germany and Spain: Public funding initiatives have strengthened hospital-integrated manufacturing capabilities, increasing trial accessibility.

• Australia: Investments in non-viral vector platforms are accelerating local innovation.

• Ireland: The recently proposed all-island CGT strategy signals the country’s commitment to fostering a unified research, clinical, and commercial environment (Lonrú Consulting, 2025).

Without vital support, translational momentum is at risk—academic labs remain the backbone of cell and gene therapy innovation, but funding challenges threaten to slow progress from discovery to patient impact.

Threats to Academic Research: The US Funding Crisis

While academic medical centers have historically been the foundation of CGT innovation, recent proposals in the US to cap indirect cost reimbursements represent a direct threat to translational research (NIH, 2025). These funds support vital infrastructure, including laboratories, safety measures, IT systems, and core personnel.

Johns Hopkins University, among others, has sounded the alarm (JHU, 2025):

“These abrupt and sweeping cuts in NIH funding pose an extraordinary challenge to the important and lifesaving work of our faculty, staff, and students. They jeopardize the longstanding and remarkable research partnership that was forged between the federal government and higher education at the conclusion of WWII and put at risk the future of the American research enterprise as a whole.”

The repercussions extend beyond academia—clinical trials reliant on NIH support, particularly in oncology, rare diseases, and regenerative medicine, now face an uncertain future. The loss of indirect funding could delay or halt up to 600 ongoing clinical trials at institutions like Johns Hopkins alone (JHU, 2025).

How Lonrú Supports Academic Translational Efforts

In an era where public funding faces unprecedented change, technology transfer offices (TTOs) must adapt. Lonrú Consulting provides strategic tools to enhance translational readiness in CGT, ensuring academic programs can efficiently navigate regulatory, funding, and commercialization hurdles.

• Tools for Translational Readiness: AI-driven tools to de-risk preclinical to clinical translation for academic labs.

• Market Positioning for Translational Centers: Interactive tools to identify funding opportunities and investor alignment, optimizing commercialization potential.

• Sustainable Financial Models: Advisory support for securing diversified funding, including philanthropic partnerships, venture investments, and alternative grant strategies.

As the impact of policy changes over funding are realised, Lonrú remains committed to supporting the academic innovation ecosystem—helping institutions turn research into real-world therapies and ensuring that promising CGT advances do not languish in the 'Valley of Death.'

Final Thoughts: Translational Research as a Pillar of CGT Sustainability

As global governments and research institutions refine their CGT strategies, translational research must remain a priority. Whether through RISE-inspired frameworks or adaptive funding models, securing the future of CGT requires sustained investment and infrastructure support. Institutions, industry partners, and policymakers must work together to uphold the critical role of academic translational centers.

References

JHU. (2025). Our essential research partnership with the NIH. Office of the University President and Office of Johns Hopkins Medicine. Email communication

Lonrú Consulting. (2025, January 29). UK Cell and Gene Therapy Clinical Trials To Date. Interactive Dashboards. https://www.lonruconsulting.com/interactive-dashboards

Lonrú Consulting. (2025, February 5). Building an All-Island CGT Strategy: Ireland’s Opportunity to Deliver Cell and Gene Therapy. Lonrú's Lens. https://www.lonruconsulting.com/blog-1-1/building-an-all-island-cgt-strategy-irelands-opportunity-to-deliver-cell-and-gene-therapy

Naresh, N. U., Hammill, D., Kessel, M., Hajjar, R. J., & Yozwiak, N. (2025). As new cell and gene therapies emerge from academia, we must RISE to the opportunity. Nature Biotech, 43(January 2025), 143-146. https://www.nature.com/articles/s41587-024-02520-9

NIH. (2025, February 7). Supplemental Guidance to the 2024 NIH Grants Policy Statement: Indirect Cost Rates. Office of The Director, National Institutes of Health. https://grants.nih.gov/grants/guide/notice-files/NOT-OD-25-068.html

Building an All-Island CGT Strategy: Ireland’s Opportunity to Deliver Cell and Gene Therapy

Last week’s Cell and Gene Therapy in Ireland conference underscored a pivotal moment for the Irish life sciences sector. The key takeaway? Ireland is in a strong starting position to deliver an ambitious all-island CGT strategy, leveraging its existing infrastructure, research expertise, and growing investment landscape to build a sustainable, patient-centric ecosystem.

Why Now? Ireland’s Readiness to Deliver in CGT

With capital continuing to flow into advanced therapies, Ireland has a unique opportunity to integrate its North-South partnerships, its biopharma expertise, and its strategic geographic positioning to drive a cohesive CGT strategy that delivers for patients across the island and beyond.

Currently, Ireland’s cell and gene therapy (CGT) landscape is still in its early stages compared to established hubs like the UK, Japan, Spain, and the US. However, this presents an advantage: a modern, efficient system can be designed from the ground up that avoids legacy inefficiencies seen in other markets.

The IMPACT initiative, introduced by Professor Sakis Mantalaris during the conference and highlighted in the BPCI budget recommendation, proposes an integrated ecosystem that spans research, clinical application, manufacturing, and distribution​. A key lesson from successful CGT hubs —such as Spain and Japan’s focus on non-viral engineering, and the UK’s predominant use of Lentivirus and AAV vectors—is the critical role of platform technologies in creating a sustainable, scalable ecosystem. As Ireland builds its CGT infrastructure, how essential will it be for stakeholders—including government bodies, academic institutions, biopharma companies, and clinical networks—to align around specific platform technologies to drive efficiencies, de-risk investment, and accelerate progress across the ecosystem?

What is the Strategy?

Ireland’s CGT strategy is outlined under IMPACT (Irish Medicines Centre for Personalised Advanced Cellular Therapeutics), which aims to:

• Develop a coordinated research and clinical ecosystem spanning both Northern Ireland and the Republic​.

• Leverage Ireland’s existing strengths in biopharma manufacturing to accelerate CGT production at scale.

• Expand clinical trial infrastructure to position Ireland as a leader in early-stage CGT research.

• Enhance regulatory and market access frameworks to ensure patient-centric delivery.

These steps align with ensuring Ireland remains competitive in the global CGT space and delivering for patients across the island.

The Benefits of an Early-Stage, All-Island Strategy

While Ireland is entering the CGT ecosystem later than the UK and US, this early-stage positioning offers several advantages:

• Strategic Design from the Start – Unlike legacy markets that must retrofit infrastructure, Ireland can build a modern, integrated CGT system from the ground up.

• North-South Collaboration – A joint approach ensures access to talent, resources, and patient populations across the entire island, creating a single innovation network.

• Geographic and Demographic Advantages – With a relatively small, yet diverse population, Ireland is well-suited for early-phase trials and real-world evidence generation.

• Existing Biopharma Expertise – Ireland’s strong pharma sector can be leveraged to accelerate CGT manufacturing and attract further investment​.

Learning from Global Success Stories

To understand how Ireland can scale CGT efficiently, it’s worth reflecting on how other nations have built their infrastructure:

The UK Model: Public-Private Alignment
The UK’s success in CGT was driven by:

• Government-backed initiatives (e.g., the Cell & Gene Therapy Catapult).

• Strong academic and hospital manufacturing networks.

• Regulatory adaptability, positioning the UK as a leader in AAV/Lentivirus vector production​.

Spain’s Early Investment in Public Health Infrastructure

• Spain built regional CGT hubs integrated with public healthcare.

• Strong clinical trial networks positioned it as a European leader in CAR-T therapies.

The US Approach: Venture-Backed Innovation

• A high-risk, high-reward model enabled rapid CGT development.

• Strong links between academia, startups, and biopharma companies fostered an ecosystem of continuous innovation​.

Key Challenges for Ireland

Despite its strong starting position, Ireland must address critical barriers to ensure its CGT strategy delivers long-term impact:

• Limited Clinical Trial Infrastructure – Investment in trial sites and patient access programs is needed​.

• High Cost of CGT Treatments – Reimbursement frameworks must be carefully designed to ensure equitable access.

• Skills and Talent Gaps – More funding is needed for CGT-specific education and workforce training programs.

• Regulatory Complexity – Ireland must establish a streamlined, innovation-friendly regulatory framework to attract CGT developers​.

The Path Forward: Positioning Ireland as a CGT Leader

If Ireland successfully aligns its CGT strategy across research, clinical development, and manufacturing, it can position itself as a global leader in next-generation therapies.

What’s Next?
The momentum from the CGT in Ireland conference highlights a critical window for action. As Ireland advances its all-island CGT strategy, Lonrú Consulting will continue to support industry leaders, startups, and policymakers in navigating the complexity of CGT innovation.

 Want to be part of the conversation? Get in touch to explore how we can collaborate in shaping Ireland’s future in CGT.

CGT Ecosystems: Ireland Has The Opportunity To Build From The Ground Up.

De-risking Cell and Gene Therapy Development: Global Regulatory Strategies for Streamlined Development

Dr. Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER), recently shared insights at the 2025 JP Morgan Healthcare Conference, outlining the FDA’s ongoing efforts to accelerate advances in cell and gene therapy (CGT). With significant breakthroughs in 2024—including approvals for tissue-engineered products, mesenchymal stem cell therapies, and solid tumor-targeting T-cells—the CGT sector continues to make significant progress. However, despite these advances, challenges in regulatory harmonization, market access, and cost-effectiveness persist, requiring strategic policy measures and industry collaboration to de-risk CGT development in the US and beyond.

The Current CGT Landscape in the US

The US remains a leader in CGT innovation, with the FDA approving 24 cell and gene therapies to date, and is predicted to review or receive applications for a record 15 new therapies in 2025. 

Figure 1. Showing FDA-approved and pipeline cell and gene therapies. 

Recognising the need for new approaches to regulating these innovative therapies, Dr. Peter Marks discussed several new initiatives underway at the FDA. Key initiatives include:

• Risk-based regulatory frameworks: The FDA is exploring a new approach to human cells, tissues, and cellular-based products (HCTPs), addressing over- and under-regulation issues with a planned scientific workshop in February 2025.

• Platform technologies like CRISPR: Marks emphasized the need for a regulatory shift, treating CRISPR as a platform tool rather than a product, which could streamline development and approval processes, potentially under the draft platform technology guidance. 

• Rare disease initiatives: Programs such as the Rare Disease Hub and collaborative efforts with the EMA are expected to accelerate treatment pathways for underserved conditions.

While these regulatory-focused initiatives are promising, ongoing efforts should also focus on reducing manufacturing bottlenecks, ensuring sustained funding for innovation, and fostering public-private partnerships to sustain momentum in the sector.

The UK Approach: A Strategic Roadmap for Growth

The UK’s CGT ecosystem is supported by institutions such as the Cell and Gene Therapy Catapult and the Advanced Therapy Treatment Centres (ATTC), which focus on scaling up manufacturing and integrating therapies into the National Health Service (NHS). Recent milestones include:

• NHS adoption of CRISPR-based therapies: The approval of Casgevy for transfusion-dependent beta-thalassemia demonstrates the UK’s leadership in pioneering CGT adoption within public healthcare.

• Regulatory flexibility: The Medicines and Healthcare products Regulatory Agency (MHRA) is actively harmonizing with global frameworks to enable smoother market entry.

By aligning with the UK’s emphasis on infrastructure and workforce development, other markets can strengthen their own pathways to commercialization.

Germany’s National Strategy: Balancing Innovation with Compliance

Germany is positioning itself as a European leader in CGT with the introduction of a National Strategy for Gene and Cell Therapies, launched in June 2024. This strategy focuses on:

• Regulatory streamlining: Efforts to simplify legal pathways and align with the European Medicines Agency (EMA).

• Investment in infrastructure: Enhancements to Germany’s biomanufacturing capabilities to ensure scalable production of CGT products.

• Industry collaboration: Strong ties between government bodies and industry stakeholders to foster a competitive environment.

Other countries can learn from Germany’s comprehensive approach by expanding infrastructure investment and creating strategic alliances to enhance manufacturing scalability.

Japan’s Regulatory Flexibility: A Model for Rapid Market Access

Japan’s regulatory framework for regenerative medicine, governed by the Pharmaceuticals and Medical Devices Agency (PMDA), may represent one of the most progressive models globally. With a conditional and time-limited approval pathway, Japan has enabled rapid commercialization of therapies such as CAR-T cell treatments. Key elements of Japan's success include:

• Accelerated approval pathways: Conditional approvals based on preliminary efficacy data, allowing early patient access.

• Post-market surveillance: Rigorous follow-up requirements ensure long-term safety and efficacy.

• Public-private collaboration: Strategic partnerships between government, academia, and industry have accelerated innovation.

To remain competitive, regulatory bodies in other regions could consider adopting Japan’s adaptive regulatory approach for certain high-priority therapies, thereby accelerating patient access without compromising safety.

Opportunities for Global Regulatory Synergy

Despite regional differences, there are clear opportunities to align regulatory processes across key markets:

1. Harmonized Regulatory Frameworks: The FDA, EMA, MHRA, and PMDA could collaborate on common standards for CGT approvals, reducing redundancies and facilitating global market entry.

2. Data-Sharing Initiatives: Establishing global databases, leveraging federated learning models to share real-world evidence can enhance regulatory decision-making and improve patient safety outcomes.

3. Streamlined Manufacturing Guidelines: Aligning quality and manufacturing standards globally can reduce costs and accelerate production scalability.

Stakeholders across the CGT ecosystem should work toward strengthening partnerships with international regulatory bodies to create a more unified approach to CGT approvals and commercialization.

A Call for Strategic Action

As CGT continues to revolutionize medicine, proactive steps are needed to de-risk development by:

• Modernizing regulatory frameworks to accommodate platform technologies.

• Increasing funding for infrastructure and workforce development.

• Strengthening international regulatory cooperation to facilitate global market access.

• Addressing cost barriers to ensure broad patient access.

By drawing from strategies across the US, the UK, Germany, and Japan, the global CGT sector can create a more resilient and competitive landscape, ensuring that transformative therapies reach patients faster and more efficiently.

Want to stay ahead in cell and gene therapy development? Explore Lonrú’s strategic solutions.

From Semiconductors to Genes: Engineering Precision in Advanced Therapies

Exploring insights from ARM's JPM 2025 panel discussion and the evolving quest for molecular-level understanding in cell and gene therapy.

Background

At this year’s JPMorgan Healthcare Conference, the ARM panel discussion on "Capital Markets and Commercial Insights: Navigating Opportunities and Challenges in CGTs" brought an unexpected but interesting comparison to light. Keith Crandall of Arch Ventures, drawing from his semiconductor and photonics background, made a compelling case for how the biotech industry—specifically cell and gene therapy (CGT)—can learn from the rigor and precision that revolutionized semiconductor manufacturing.

Crandall noted that in semiconductor production, every molecule is quality-checked, and processes are controlled down to the quantum level. In contrast, CGT manufacturing still grapples with variability and a limited characterization of biological components at a molecular level. According to Crandall, the key to unlocking CGT’s full potential lies in adopting semiconductor-level precision and embedding quality control (QC) from the outset—not as an afterthought.

The Semiconductor Blueprint: A Roadmap for CGT

The semiconductor industry underwent significant transformation from its early, experimental days to become one of the most exacting fields in modern manufacturing. The drivers of this shift included:

• Standardisation and Process Control: Introduction of Statistical Process Control (SPC), Six Sigma, and rigorous certification processes (Anonyuo et al., 2024).

• Deep Foundational Understanding: Mastery over materials and physics that enabled predictable, repeatable outcomes.

• Leadership Evolution: Semiconductor firms appointed leaders with strong manufacturing backgrounds, shifting from leaders with academic experience. 

• Collaborative Ecosystems: Industry-wide knowledge-sharing through consortia like SEMATECH helped accelerate improvements across the board (Irwin & Klenow, 1996).

Precision at the Molecular Level: Lessons from Semiconductors for the Future of Cell and Gene Therapy

CGT: A Field in Search of Precision

Despite the substantial advancements in CGT, significant hurdles remain, particularly in manufacturing scalability and consistency. The inherent complexity of biological systems introduces unpredictable variances—an issue that semiconductor methodologies could help address.

Key Areas for CGT Innovation Inspired by Semiconductors

1. Molecular-Level Understanding
   Advances in single-cell analysis and multi-omics technologies are helping bridge the knowledge gap in CGT. Spain, for example, has a network of research groups conducting single-cell RNA sequencing pre- and post-CAR-T cell therapy to better understand therapeutic persistence and variability (Rodriguez-Madoz et al., 2023). Similar efforts are emerging globally, including:

• MIT’s Center for Biomedical Innovation (USA): Exploring process control and predictive analytics in cell therapy manufacturing.

• Fraunhofer Institute (Germany): Investigating automation and in-line analytics for bio-manufacturing.

• Cell and Gene Therapy Catapult (UK): Driving standardisation and scalable process development across the industry.

2. Standardised QC Protocols
   The standardisation of QC protocols in ATMP manufacturing is evolving through the adoption of real-time process controls, automation, and the promise of harmonised regulatory frameworks from bodies like the FDA, EMA, and PMDA. 

3. Automation and Closed Systems
   Leveraging automation and robotics to minimise human intervention in CGT manufacturing aligns closely with semiconductor principles. Companies such as Ori Biotech and Autolomous are pioneering efforts in digital manufacturing platforms tailored for advanced therapies.

4. Leadership Evolution
   As CGT companies advance, there is a growing recognition of the need for leadership with expertise in industrial-scale manufacturing. Bridging the gap between clinical and operational expertise could unlock significant efficiencies and scalability.

The Future Outlook: Unlocking Precision in CGT Manufacturing

Crandall’s perspective underscores that precision in biological manufacturing will do more than just improve yields and consistency—it could fundamentally change how we approach cell engineering and advanced therapeutic manufacturing. By minimising variability and embedding precision from the outset, CGT manufacturers could realise therapies that are safer, more predictable, and ultimately more accessible to patients worldwide.

Conclusion

The intersection of semiconductor precision and CGT innovation presents an exciting frontier. While biology’s inherent complexity presents challenges, adopting rigorous QC frameworks and fostering cross-industry collaboration can accelerate CGT’s trajectory from bespoke treatments to widely available, cost-effective therapies.

Lonrú Consulting continues to explore and support these transformational shifts—illuminating the opportunities for CGT companies looking to navigate complexity with precision and clarity.

References

Anonyuo, S., Kwakye, J., & Ozowe, W. (2024, November 22). A review of quality control and process optimization in high-volume semiconductor manufacturing. World Journal of Engineering and Technology Research. https://zealjournals.com/wjetr/content/review-quality-control-and-process-optimization-high-volume-semiconductor-manufacturing

Irwin, D., & Klenow, P. (1996, November 12). Sematech: Purpose and Performance. Proceedings of the National Academy of Sciences. https://www.pnas.org/doi/full/10.1073/pnas.93.23.12739

Rodriguez-Madoz, J. R., Prosper, F., & et al. (2023, November 2). Sequential Scmultiomics of In Vivo CAR-T Cells Allows Characterization of Transcriptional Differences between Patients, and Identifies IL10 As a Potential Mechanism of Resistance to CAR-T Cells in MM. Blood, 142(Supplement 1), 3433. https://ashpublications.org/blood/article/142/Supplement%201/3433/499052/Sequential-Scmultiomics-of-In-Vivo-CAR-T-Cells

Illuminating the Future of Cell and Gene Therapies

Key Insights and Lonrú’s Role in Driving Ecosystem Collaboration

Bridging the Gaps in the Evolving CGT Landscape

The cell and gene therapy (CGT) industry is experiencing transformative growth, but with this progress comes a host of challenges. Insights shared during ARM’s State of the Industry Capital Markets and Commercial Insights panel revealed that success in CGT hinges on addressing key obstacles such as regulatory complexity, stakeholder engagement, and ecosystem collaboration. These challenges, if unaddressed, could hinder the sector’s ability to deliver groundbreaking therapies to patients worldwide.

ARM’s State of the Industry Briefing: Panelists discussing capital markets and commercial insights.

Key Themes and Challenges in CGT

1. Regulatory Complexity Across Regions
   The CGT sector operates within highly regulated environments, with frameworks varying between regions like the U.S., Europe, and Asia. Panelists stressed the need for tools that help companies adapt their regulatory strategies to meet jurisdiction-specific requirements efficiently. In an earlier session, the FDA’s Dr. Peter Marks underscored early efforts to align regulatory requirements across geographies, aiming to reduce the burden on sponsors. We’ll explore this topic in detail in an upcoming blog.

2. Ecosystem Collaboration: A Critical Imperative
   The panel emphasised that the CGT industry’s growth depends on strong collaboration among biotechs, academic institutions, investors, and regulators. However, many companies struggle to create these connections due to siloed approaches or timing of communication strategy, many leaving it late in the development process.

3. Stakeholder Engagement and Communication
   Effective stakeholder engagement remains a top challenge, with companies needing to tailor their messages to resonate with specific audiences—investors, regulators, patients, or clinicians. A one-size-fits-all approach simply does not work in such a diverse ecosystem.

4. Data Integration for Better Decision-Making
   Companies face the challenge of integrating data, ranging from clinical trial results to real-world patient outcomes. Transforming this data into actionable insights that drive strategic decisions is crucial but often underdeveloped.

5. Speed-to-Market and Competitive Pressure
   The CGT sector needs streamlined operations and fast time-to-market. Key challenges include identifying eligible patients, fostering trust through early communication, establishing accessible treatment centers, and aligning stakeholders. Addressing these challenges is critical for successful product launches.

The panel also surfaced intriguing perspectives that warrant deeper exploration. Keith Crandell of ARCH Venture Partners drew parallels between the CGT industry and the semiconductor sector, suggesting that lessons from semiconductors’ QC’ing and standardisation could shape CGT’s future success. Click here for a blog post diving into this concept.

Lonrú Consulting: Enabling Collaboration and Tailored Engagement

At Lonrú Consulting, we understand that thriving in the CGT sector requires more than cutting-edge therapies—it demands a collaborative, data-driven approach to overcoming industry challenges. Here’s how we help address these key themes:

• Driving Ecosystem Collaboration with Tailored Solutions
  Lonrú’s AI/ML-powered solutions facilitate collaboration by providing frameworks that align stakeholders, streamline workflows, and foster co-creation. Our interactive dashboards offer a centralized view of shared objectives, enabling seamless partnerships between biotechs, academic institutions, and investors.

• Enhancing Stakeholder Engagement
  Lonrú specializes in crafting stakeholder-specific messaging, ensuring that communication resonates with diverse audiences. Whether it’s presenting data to regulators, showcasing ROI to investors, or educating clinicians, our solutions enable companies to deliver personalized, impactful narratives.

• Turning Data into Actionable Insights
  Our advanced data analytics solutions can be deployed to integrate clinical, market, and patient datasets to provide clear, actionable insights. This supports informed decision-making, helping clients navigate regulatory landscapes, anticipate market trends, and optimize therapeutic strategies.

• Accelerating Speed-to-Market
  By owning resource-intensive tasks such as market development research, product development strategy, and marketing collateral creation, Lonrú reduces bottlenecks in the development process. This allows companies to move faster without sacrificing quality or compliance.

• Empowering Precision Communication
  With Lonrú’s tailored AI-driven content generation, companies can create high-impact materials—from investor decks to engaging podcasts—quickly, effectively and without compromising on quality. This ensures that stakeholders receive the right message, at the right time, through the right channels.

Lonrú’s Vision: Illuminating Opportunities For Innovation

Lonrú Consulting is poised to empower CGT organisations by simplifying complexity, fostering collaboration, and enhancing communication. By leveraging advanced AI/ML solutions and industry expertise, we help clients build strong ecosystems, engage stakeholders meaningfully, and bring transformative therapies to market faster.

Ready to Illuminate Your Path?
Let Lonrú Consulting help you navigate the intricacies of CGT with tailored, innovative solutions. Contact us today to learn more about how we can accelerate your journey to success.

Charting the Trajectory of Cell and Gene Therapy from 2024 to 2025

What to Expect from the 2025 State of the Industry Briefing

This morning at Lonrú we’re looking back on 2024, and sharing our take on what to expect from today’s discussions at the Alliance for Regenerative Medicine’s (ARM) 2025 State of the Industry briefing.

The past year was marked by significant progress, including advancements in in vivo therapies, global regulatory milestones, and an ongoing push to address affordability and accessibility in cell and gene therapy (CGT). As the industry continues to mature, we anticipate that today’s briefing will highlight key inflection points and lay out the challenges and opportunities for the year ahead.

From in vivo innovations and manufacturing breakthroughs to expanded therapeutic targets and global market shifts, the CGT sector is poised for another transformative year. Here’s our take on what shaped 2024 and what may define the future.

2024: A Year of Momentum and Milestones

Breakthroughs Becoming the Norm

2024 began with optimism following a stormy 2023 in capital markets. Despite economic volatility throughout 2024, the CGT sector achieved major milestones: nine new therapies were approved in the U.S., and two in Europe. The FDA’s approval of two sickle cell gene therapies in late 2023 further underscored the field’s transformative potential. These developments heralded a “new normal” where groundbreaking therapies routinely became available to patients (ARM, 2024).

Advancing Cell Therapy

By February, the industry spotlight shifted toward engineered cell therapies. The approval of tumour-infiltrating lymphocyte (TIL) therapy for solid tumours represented a watershed moment, while investment in autoimmune applications highlighted the expanding scope of cell therapies beyond oncology (ARM, 2024).

Reframing the Value Narrative

August saw renewed focus on the affordability and accessibility of CGT. Frameworks like “Gene Therapy for Patients and Society” (GPS) and “CAR-T for Patients and Society” (CAR-PS) were developed to demonstrate the long-term value and cost-effectiveness of these therapies. Such efforts aimed to reshape public perception and healthcare decision-making (CMS, 2024).

New Gene Editing Frontiers

December capped the year with approvals for two adoptive cell therapies targeting solid tumours and the introduction of next-generation gene editing techniques like prime and epigenetic editing in clinical trials. These developments opened new possibilities for tackling previously untreatable conditions (ARM, 2024).

In Vivo Growth: A Rising Star

While much of 2024’s focus was on ex vivo therapies, in vivo approaches gained significant traction. These methods, which deliver genetic modifications directly within the patient’s body, promise to enhance precision and scalability. Key developments included:

• CRISPR-Based In Vivo Therapies: Clinical trials demonstrated promising results in diseases like Duchenne muscular dystrophy and retinal disorders. These breakthroughs highlight the growing maturity of in vivo technologies (ARM, 2024).

• Next-Generation Gene Editing: Techniques such as prime* and epigenetic editing, which offer higher precision and reduced off-target effects, entered early clinical stages (Tune Therapeutics, 2024) (O'Hanlon Cohrt, 2024).

• Innovative Delivery Mechanisms: Advances in nanoparticle and viral vector technologies further improved the efficiency and safety of in vivo therapy delivery (ARM, 2024).

Looking ahead, in vivo growth will likely feature prominently in discussions about the next wave of CGT innovations, particularly in areas like systemic genetic disorders and regenerative medicine.

Predictions for 2025

Based on 2024’s trajectory, today’s State of the Industry briefing may shine a light on several critical themes:

1. Regulatory and Manufacturing Innovations
With the FDA’s expanded Office of Therapeutic Products, efforts to harmonise global regulatory frameworks and streamline manufacturing processes will take centre stage.

2. Expanding Therapeutic Horizons
Expect updates on the broadening range of disease targets, including autoimmune conditions and rare disorders, as clinical breakthroughs continue to multiply.

3. Addressing Affordability and Access
ARM’s ongoing advocacy for outcomes-based agreements and cost-efficiency frameworks will remain a focal point as the industry works to balance innovation with equitable patient access.

4. Continued In Vivo Innovation
Advancements in delivery systems and new data from clinical trials of in vivo therapies will likely feature heavily, positioning this approach as a cornerstone of future CGT strategies.

5. Ecosystem Collaboration
Fostering partnerships and collaboration across academia, biopharma, and regulatory bodies will underpin sustainable CGT growth.

Illuminating the Path Forward

2024 demonstrated that the CGT sector is no longer in its infancy. Breakthroughs are expected, and the focus has shifted toward refining processes, improving access, and expanding therapeutic potential. As 2025 unfolds, Lonrú Consulting will continue to illuminate the opportunities for our clients, providing data-driven insights and tailored strategies to help them navigate this complex and dynamic landscape.

*Prime Medicine’s clinical program PM359 is an ex-vivo engineered HSC product (O'Hanlon Cohrt, 2024). Prime editing technology holds promise as an in-vivo engineering platform (Newby & Liu, 2021). 

References

ARM. (2024, January 8). Introduction and Industry Update - Cell & Gene State of the Industry Briefing 2024. YouTube. Retrieved January 13, 2025, from https://www.youtube.com/watch?v=8uyekVKDo8Y

ARM. (2024, April 30). SECTOR SNAPSHOT ADVANCES IN ENGINEERED CELL THERAPY. ARM - Industry updates. https://alliancerm.org/wp-content/uploads/2024/05/Sector-Snapshot-4.30.2024.pdf

ARM. (2024, December 30). Alliance for Regenerative Medicine - December 2024 Sector Snapshot. ARM Industry Updates. https://alliancerm.org/wp-content/uploads/2025/01/20250107-2024-Sector-Snapshot.pdf

CMS. (2024, March 7). Cell and Gene Therapy (CGT) Access Model. enters for Medicare & Medicaid Services. Retrieved January 13, 2025, from https://www.cms.gov/priorities/innovation/innovation-models/cgt

Newby, G., & Liu, D. (2021, November 1). In vivo somatic cell base editing and prime editing. Molecular Therapy, 29(11), 3107-3124.

O'Hanlon Cohrt, K. (2024, May 1). The FDA Has Cleared the First Clinical Trial Application for a Prime Editor. CRISPR Medicine News. https://crisprmedicinenews.com/news/the-fda-has-cleared-the-first-clinical-trial-application-for-a-prime-editor/

Tune Therapeutics. (2024, November 14). Tune Therapeutics Moves into Clinical Spotlight with TUNE-401: A First-in-Class Epigenetic Silencer for Hepatitis B. Investors and Media. https://tunetx.com/tune-therapeutics-moves-into-clinical-spotlight-with-tune-401-a-first-in-class-epigenetic-silencer-for-hepatitis-b/

Introducing Lonrú Consulting

Navigating Complexity

Illuminating Innovation

Empowering the Future of Cell and Gene Therapy

The cell and gene therapy (CGT) sector is experiencing unprecedented growth, diversification, and challenges (McKinsey & Co., 2022). At Lonrú Consulting, we’re here to help organisations navigate this complex landscape by providing tailored solutions that empower innovation, optimise efficiency, and drive impactful results.

Our Mission

Lonrú Consulting is dedicated to delivering data-driven insights and actionable strategies that address the unique challenges of the CGT industry. Through collaboration, advanced tools, and domain expertise, we guide clients toward sustainable growth and innovation in a highly competitive market.

Why Now for AI/ML?

The rapid evolution of AI/ML tools over the last 18 months has transformed how organisations address critical challenges across many industries. The wider adoption of AI/ML in the life sciences field has triggered both excitement and caution (Nature, 2023). On one hand, these tools promise to revolutionise processes such as drug discovery, regulatory navigation, and patient engagement (Hutson 2024). On the other hand, perceptions of these tools as “black boxes” with potential accuracy and reliability issues have made some organisations hesitant to fully commit (McKinsey & Co., 2023)​. Despite these concerns, the tide is shifting as the technology continues to improve and demonstrate measurable impact.

Perceptions in Life Sciences

Early adopters of AI/ML in life sciences report a 30% improvement in engagement and satisfaction scores when using AI/ML powered tools for content personalisation and insight generation. Many life sciences leaders emphasise the importance of clear AI strategies, with companies adopting customised solutions seeing twice the impact compared to those using off-the-shelf models​ (McKinsey & Co., 2023). However, challenges such as data privacy, algorithmic transparency, and the potential for misinformation (e.g., hallucinations in outputs) continue to shape perceptions (Nature Editorial, 2024)​.

Building Confidence in AI for CGT

Transparency and customisation are critical to unlocking the full potential of AI in life sciences. Companies with defined AI strategies report twice the impact compared to those without (McKinsey & Co. 2023). At Lonrú, we ensure that every AI-powered solution is tailored to meet our clients’ specific goals, fostering trust and maximizing outcomes.

How Lonrú Can Help

Our AI/ML-driven solutions evolve with client needs, ensuring relevance in the fast-paced CGT landscape. By blending cutting-edge technology with domain expertise, we address industry-specific challenges and enable our clients to:

• Analyze Data Effectively: Transform complex datasets into actionable insights.

• Optimize Workflows: Eliminate inefficiencies and boost productivity.

• Facilitate Collaboration: Connect biotechs, academia, and investors to foster innovation and growth.

In an industry where speed, precision, and trust are paramount, Lonrú equips clients to navigate complexity confidently, seize emerging opportunities, and drive impactful innovation.

Stay updated on our capabilities and projects by connecting with Lonrú Consulting on LinkedIn using the link below.

References

Hutson, M. (2024, March 14th). Cutting to the chase. Health sciences. https://media.nature.com/original/magazine-assets/d41586-024-00753-x/d41586-024-00753-x.pdf

McKinsey & Co. (2022, November 16th). How AI can accelerate R&D for cell and gene therapies. Life Sciences. https://www.mckinsey.com/industries/life-sciences/our-insights/how-ai-can-accelerate-r-and-d-for-cell-and-gene-therapies

McKinsey & Co. (2023, May 6th). Early adoption of generative AI in commercial life sciences. McKinsey Direct. https://www.mckinsey.com/industries/life-sciences/our-insights/early-adoption-of-generative-AI-in-commercial-life-sciences#/

Nature. (2023, December 6). Science and the new age of AI. Immersive. https://www.nature.com/immersive/d41586-023-03017-2