Key Takeaways
- Infrastructure Gap: Scientific progress in CGT is currently moving faster than the regulatory and reimbursement systems required to support it.
- Platform Standardization: Shifting toward modular "platform thinking" can streamline CMC processes and regulatory approvals for bespoke therapies.
- Manufacturing Bottlenecks: Scaling remains difficult due to manual batch records, labor-intensive processing, and cleanroom constraints.
- AI Integration: Machine learning is actively improving AAV vector design and manufacturing consistency, leading to safer and more effective delivery.
- Access Models: Long-term growth depends on hub-and-spoke delivery systems and early engagement with payers to establish value-based reimbursement.
1. Innovation is outpacing infrastructure
Several speakers described how current clinical, regulatory, and manufacturing systems are not keeping pace with the speed of scientific progress in CGT. Vanessa Almendro-Navarro, Vice President and Head of Science and Technology Innovation at Danaher, said that the field now faces an “ecosystem” problem rather than a scientific one.
The tools for gene editing, AAV delivery, and CAR-T production have been repeatedly validated across programs, but the supporting infrastructure remains limited. This includes a lack of well-established referral pathways and reimbursement models.
In turn, this mismatch between next-generation therapies and the current ecosystem leads to concerns around commercial viability and regulatory roadblocks. In the U.S., most CGT products are regulated by the FDA’s Center for Biologics Evaluation and Research (CBER) (FDA CGT product page). As the number of CGT programs in clinical development grows, regulatory frameworks built for conventional biologics do not always accommodate individualized or genetically targeted therapies. Investors are now assessing not only the strength of the underlying science but also the scalability of operations, reimbursement readiness, and clarity of regulatory pathways. Programs that demonstrate integration across these areas are more likely to attract sustained investment. Speakers noted that the next growth cycle in CGT will depend on translating technical capability into predictable business models.
Adapting the current landscape to better accommodate CGTs requires:
- Shared data frameworks between clinical and manufacturing programs
- Early patient identification and referral systems that connect diagnosis to genetic qualification and trial eligibility — not just top-of-funnel awareness
- Regulatory and reimbursement models designed for individualized therapies
Building that framework is what will determine whether these therapies move from isolated clinical successes to routine patient care.
2. Platform thinking for repeatable development
Vanessa Almendro-Navarro also outlined the importance of applying platform thinking to CGT development. Rather than building each therapy from scratch, companies can create modular platforms that standardize design, chemistry, manufacturing, and control (CMC) processes, along with regulatory documentation.
The case of baby KJ marked the first use of bespoke in vivo CRISPR therapy, developed in just six months to treat a rare genetic condition. The team behind the case is now seeking a platform designation that would validate the backbone of the therapy under a single regulatory framework. Once that backbone is approved, new therapies could be advanced by changing only the guide RNA while maintaining the same chemistry, manufacturing, and control processes. This model was highlighted as an example of how future individualized therapies could be developed and scaled under a shared regulatory structure, reducing the regulatory and development burden for each subsequent therapy in the program.
3. Manufacturing remains the main constraint
Barbara Ressler, Vice President of Process Sciences at Roslin CT, presented an overview of the operational limits that still define CGT manufacturing. Allogeneic therapies use donor-derived cells produced in standardized batches, while autologous therapies use a patient’s own cells and require a one-batch-per-patient model (Novartis’s CGT explainer). This patient-specific manufacturing approach creates challenges for cost, quality, and throughput.
Key bottlenecks include:
- Manual batch records and paper-based quality reviews
- Cleanroom space constraints
- High labor requirements for processing
- Long testing and release cycles
Although automation and robotics are beginning to help, Ressler noted that scaling production will require redesigned workflows and integrated data systems, not just incremental process improvements.
4. AI and data-driven design are improving performance
Advances in AI in cell and gene therapy are starting to show measurable impact. April Sena, Vice President of Technical Operations at Life Edit Therapeutics, shared how the company uses genome mining and machine learning to identify new nucleases and optimize AAV vector design. This approach increases the percentage of full capsids in production runs, aligning with FDA quality expectations and improving manufacturing consistency.
Adrian Veres, Co-founder and Chief Scientific Officer at Dyno Therapeutics, discussed how AI-guided AAV design is helping create safer and more effective delivery systems. Dyno’s new capsids achieved improved central nervous system (CNS) delivery, reduced liver tropism compared to AAV9, and higher neuronal transduction efficiency at lower doses.
These results show that AI-guided design can improve capsid performance and manufacturing consistency in ways that reduce the iterative burden typically associated with vector optimization. More consistent vector production and improved tissue targeting can simplify dosing protocols, broaden patient eligibility, and reduce enrollment complexity for genetically stratified trials.
5. Patients and advocates are driving awareness and access
A panel on patient advocacy emphasized how patients are increasingly leading efforts in funding, education, and outreach. Many rare disease foundations now fund bespoke gene therapy programs directly.
This pattern has been documented in detail on The Genetics Podcast, with guests including Terry Pirovolakis (CEO of Elpida Therapeutics) and Justin Porcano (Co-founder and Executive Director of Save Sight Now), both of whom transitioned from outside industry to lead patient-driven drug development programs.
Speakers highlighted that while this engagement accelerates progress, it also exposes system gaps. Patients should not have to become organizers or fundraisers to access treatment. ASGCT's patient education platform (ASGCT patient education) is one example of existing infrastructure covering conditions treated, clinical trial participation, and genetic diagnosis pathways. Expanding clinician education, establishing referral networks, and formalizing partnerships with advocacy groups is necessary to distribute responsibility more evenly.
6. Economics and access determine long-term growth
Joe DePinto, Head of Cell, Gene, and Advanced Therapies at McKesson, presented data showing that while the CGT market is expected to grow more than 30% annually through 2030, therapy approvals have slowed. The FDA’s “Flexible Requirements for Cell and Gene Therapies to Advance Innovation” initiative is one concrete example of an evolving regulatory environment (FDA CGT product page). Shifts in regulatory standpoints and approval timelines have created uncertainty for sponsors.
In a parallel discussion, Erica Cischke, Vice President of Government Affairs at ARM, focused on reimbursement and care delivery. Key recommendations included:
- Early engagement with payers to develop value-based reimbursement models
- Expanding treatment access through hub-and-spoke delivery systems, supported by coordinated patient identification, genetic qualification, and engagement infrastructure that can operate across distributed care settings
- Increasing awareness that approved CGTs are commercially available, not experimental
One applied example of the hub-and-spoke model was discussed on The Genetics Podcast: BridgeBio uses it to de-risk rare disease drug development and spread risk across programs. These structural changes will help align financial incentives and clinical operations with the realities of gene and cell therapy.
Building the infrastructure that CGT progress now requires
The workshop made it clear that the next phase of CGT progress depends on system design. Scientific innovation has outpaced infrastructure, and the sector now needs coordinated frameworks that connect research, manufacturing, and clinical delivery. That includes infrastructure for patient identification, referral pathways, and genetic testing access. Without these connected systems, even approved therapies may not reach their intended patient populations. Strengthening this layer is essential to translate clinical advances into routine care. Standardization, collaboration, and data integration will determine how quickly and effectively CGTs can move from individual success stories to routine care.
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