The Business of Reaching Patients: ESGCT 2025 Preview

2025’s most important cell and gene therapy (CGT) conversations focus on how we pay, where we manufacture, and who gets access to therapies. ESGCT’s programme quietly concentrates several talks that sit right at that junction. Below are the sessions we’re watching for signals on real-world access, sustainable economics, and the operating models that will define CGT’s next chapter.

1. Clinical Trials & Commercialisation Workshop

(Tuesday, a.m.)

Theme: pricing, access, and launch realism.
Expect a candid sweep from regulatory mechanics to payer expectations and patient‑group perspectives. Two standouts:

• Reimbursement models for ATMPs: a practical tour of contracts (outcome‑based, annuity, hybrid) and the data demands they impose on sponsors.

• Advanced therapies in rare disease: research to commercial developer‑side lessons on first approvals, launch phasing, manufacturing readiness and payer evidence.

What to listen for: Where multi‑year outcomes contracts are actually landing; how to “design for reimbursement” early (endpoints, real-world-evidence capture, and post‑market commitments built into the plan).

2. In Vivo Gene Therapy

(Wednesday, 15:30–17:30)

This block is a litmus test for one‑and‑done value narratives. The way durability, safety, and quality of life are framed here will influence payer appetite and trial design across the board, especially for programs signaling pivotal readiness. Watch for language around post‑approval data, registry infrastructure, and the end of lifelong chronic therapy in specific conditions.

3. Manufacturing I: Delivery Technologies

(Wednesday, 18:00–19:30)

Session highlight: INV30 A Strategic Platform Roadmap for Commercializing Gene Therapy - Cesar Trigueros (Viralgen).
This is likely to read as an operating model talk with a view to CMC: platform efficiencies, indication sequencing, COGS sensitivity, QC and comparability, and what “commercial‑ready” actually looks like for vector manufacturing in 2025.

Also worth catching in the same session: practical levers that move the unit economics viral vector cell‑line engineering, downstream simplification, and analytical automation, for sponsors who need cost curves that stand up to HTA scrutiny.

4. Accessibility of Gene Therapy

(Thursday, 15:30–17:30)

A rare, concentrated discussion of how to reach more patients: logistics, site qualification, patient access, and payer readiness. Expect pragmatic perspectives from hospital systems and regulators on topics like:

• Shipment, chain‑of‑identity, and turn‑around constraints that still block scale.

• What “fair access” and “practical affordability” look like for ultra‑rare populations.

• How to structure evidence generation so that pricing debates become data‑driven rather than aspirational.

5. Manufacturing II: Cell Therapies

(Thursday, 18:00–19:30)

Session highlight: INV60 Decentralized, innovative point‑of‑care CAR‑T manufacturing platform - Ruiz Astigarraga (Galapagos BV).
This is the business model experiment many have been waiting to see at scale: push manufacturing closer to the bedside to compress vein‑to‑vein, increase throughput, and reduce logistics risks.

Listen for:

• What the real time gains are (collection → release).

• How digital release, comparability, and QA work across a network of centers.

• The site‑level economics (capex/opex, staffing, training) and who pays for what.

Related talks in this block - TIL manufacturing, cryo dynamics, and LVV platform learnings - fill in the operational detail that determines whether “point‑of‑care” is a headline or a workable system.

6. National Initiatives in Gene & Cell Therapy

(Thursday, 18:00–19:30)

This session maps country‑level frameworks for scaling academic manufacturing, coordinated trials, and patient access and how infrastructure and philanthropy‑backed models may intersect with national health systems to broaden availability.

What to listen for:

• How Germany, Spain, Italy, France, the UK and Sweden are organizing capacity and standards.

• Whether cross‑border data and release protocols are converging enough to reduce duplication and cost.

• The role of hospital exemption and academia‑industry hybrids in earlier patient access.

7) Regulatory Requirements for CTAs

(Friday, 11:00–13:00)

With senior voices from agencies, expect clarity on:

• First‑in‑human expectations for manufacturing and control.

• How the evolving EU legislative landscape interacts with CGT development timelines.

• What regulators want to see in post‑approval evidence to support sustained access and reimbursement.

8) EuroGCT Lunchtime Symposium

(Friday, 13:30–14:30)

Policy and regulatory strategy focused on translation and equitable delivery. Watch for signals on registries, real-world-evidence, and center‑of‑excellence concepts that could become the “plumbing” for outcome‑based payment and cross‑site learning.

Lonrú Clients: What this means for CGT enablers

• Design for access, not just efficacy. If decentralized and rapid‑release models gain ground, demand rises for modular cleanrooms, integrated analytics, networked QA, and software that makes batch review and chain‑of‑identity auditable across sites.

• Plan the contract before the trial. Build outcomes capture, follow‑up cadence, and real‑world endpoints into protocol design to support annuity or outcomes‑based payments later.

• Academia is a serious buyer. National initiatives and philanthropic ecosystems (Italy is a prime example) are creating structured channels for tools, analytics, and training - especially where public infrastructure underwrites early access.

The sessions to circle in your diary

• Clinical Trials & Commercialisation Workshop (Tuesday, a.m.) pricing, contracting, and the patient perspective.

• In Vivo Gene Therapy Plenary (Wednesday, 15:30–17:30) framing “near‑market” for payers.

• Manufacturing I (Wednesday, 18:00–19:30) INV30 Cesar Trigueros (Viralgen) on a commercialization roadmap.

• Accessibility of Gene Therapy (Thursday, 15:30–17:30) practical access and logistics.

• Manufacturing II (Thursday, 18:00–19:30) INV60 Ruiz Astigarraga (Galapagos BV) on decentralized CAR‑T.

• National Initiatives (Thursday, 18:00–19:30) include Italy alongside Germany, Spain, France, the UK, Sweden.

• Regulatory Requirements for CTAs (Friday, 11:00–13:00) policy reality check.

How Lonrú can help

Lonrú’s VantagePoint™ services support companies turning these signals into action:

• Insights: market and policy intelligence to anticipate the access path.

• Validation & Accelerate: go‑to‑market design, including networked manufacturing and payer‑ready evidence plans.

• Connect & Scale: partnerships with national initiatives, academic centers and investors; pricing and scale frameworks aligned to real‑world delivery.