Efficacy, safety and precision are pushing innovation across viral vectors, non-viral delivery, genome editing and next-generation platforms.

AAV and LVV remain the established workhorses, but they're evolving fast toward more scalable, cost-effective systems, while novel delivery technologies open up diseases and patient populations that weren't previously addressable.

Platform innovation is becoming the thing that actually separates developers from each other.

Key discussion points:

• In vivo delivery platforms stacked up: how AAVs, LVVs and LNPs compare on safety, immunogenicity, durability, targeting and redosing
• Non-viral/LNPs — manufacturing and CMC: formulation, stability, analytics and scale-up
• Next-gen vectors and emerging payloads: hybrid systems, VLPs, mRNA, saRNA, circRNA and gene editing platforms

The gap between clinical approval and real-world reach is still cell and gene therapy's defining problem.

Community oncologists often hold back on referrals, the economics of losing a patient to a specialist center, combined with genuine unfamiliarity with CAR-T, keep patients from ever reaching the therapies approved for them.

Closing that gap needs developers, payers, providers and patient communities working the problem together, not separately.

Key discussion points:

• Referral and awareness: why community oncologists don't refer, and what would change that
• Eligibility and payer authorization: the second-opinion problem and the prior-authorization wall
• CAR-T Vision 2030: what the payer/authorization workflow needs to look like to double the treated population
• Global access: what it will actually take to move the needle for patients everywhere

Advanced therapies need supply chains unlike anything else in healthcare: precise chain of custody, cryopreservation logistics, and a "final mile" that can't afford to fail.

As more commercial therapies reach the market, the pressure on connected, globally integrated supply chains is only going to grow.

Key discussion points:

• Scaling beyond 20% of eligible patients: the manufacturing and supply chain advances needed to get there
• COGs control: how far there is still to go, and where the real targets should be
• Supply chain as commercial strategy: starting materials, shelf life, cryopreservation and the final mile
• Site readiness: the role of automation in making point-of-care delivery reliable

Regulatory frameworks are evolving alongside the science, not always at the same pace.

Developers are navigating diverging regulatory expectations, shifting policy, and reimbursement landscapes that look completely different market to market, and closer collaboration between regulators is the only way to make approvals faster and more consistent globally.

Key discussion points:

• Regulatory navigation in 2027: IND & BLA feedback, CMC guidance, and global regulatory divergence
• Ex-US IIT-derived clinical data: acceptability and what it means for global trial strategy
• Accelerated approval pathways: do the benefits actually outweigh the costs?
• Running IITs outside the US or Europe: lessons from recent examples in China and beyond

Success in advanced therapies is often decided long before pivotal trials begin.

Getting early scientific decisions right, and generating robust proof of concept, is what lays the foundation for regulatory success, investment, and everything that follows.

Key discussion points:

• What "phase-appropriate" actually looks like: balancing IND needs against later-stage development
• Potency and comparability assays: how to approach them from an early stage
• Streamlining clinical proof of concept: innovation in early trial design and execution, and the pitfalls to avoid