The evolution of cell and gene therapy: 6 themes shaping the next phase of growth

At the recent Evolution of the Cell and Gene Therapy (CGT) Sector Workshop by the Alliance for Regenerative Medicine (ARM), speakers from across industry, academia, and advocacy explored what needs to happen next for cell and gene therapies to reach full maturity. The consensus was clear that innovation in CGT is progressing faster than the systems that support it. The next challenge is to make these therapies scalable, reliable, and accessible. In this blog, we cover six key themes that emerged from the discussions.

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 validated again and again, 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. 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 could entail:

• Shared data frameworks between clinical and manufacturing programs
• Early patient identification and referral systems
• Regulatory and reimbursement models designed for individualized therapies

As the CGT field continues to accelerate, it will be crucial to build a framework that can allow it to reach patients who need these life-saving therapies. 

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 from earlier this year 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, saving time and money in the development process.

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. Autologous therapies require individual batches for each patient, which 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 developments show that AI and data analytics can shorten development cycles and reduce uncertainty across both R&D and manufacturing.

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.

There have been several powerful examples of this on The Genetics Podcast, with guests like Terry Pirovolakis (CEO of Elpida Therapeutics) and Justin Porcano (Co-founder and Executive Director of Save Sight Now) pivoting away from their careers to become immersed in the world of drug development to save their children with rare genetic diseases. 

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. Expanding clinician education, establishing referral networks, and formalizing partnerships with advocacy groups could help 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. This is largely linked to fluctuations in regulatory standpoints and decisions over the past year.

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
• Increasing awareness that approved CGTs are commercially available, not experimental

An excellent 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 systems for next-generation therapies

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. Standardization, collaboration, and data integration will determine how quickly and effectively CGTs can move from individual success stories to routine care.

 To explore more trends shaping the gene therapy field in 2025, read our whitepaper.