Navigating Funding and Development Challenges for Transformative Therapies

At ASGCT's 28th Annual Meeting in May, leaders from across the ecosystem gathered to explore innovative solutions to advance these transformative and potentially curative treatments from concept to patient access.

Fireside Chat: Navigating the Evolving Funding Landscape

A fireside chat moderated by ASGCT's Immediate Past President, Dr. Paula Cannon (USC) brought together Dr. P.J. Brooks (NCATS Division of Rare Diseases Research Innovation), Dr. Mimi C Lee (ARPA-H), Dr. Rosa Canet-Aviles (CIRM), and Pat Furlong (PPMD). This diverse panel of experts provided crucial insights into securing funding from preclinical research through commercialization, offering perspectives from federal agencies, state-level initiatives, and the patient advocacy community.

A central theme emerged regarding the need to reimagine metrics for the value, and return on investment, in CGT development. The panelists explored how traditional valuation models often fail to capture the transformative nature of these therapies, particularly for rare diseases where patient populations are small but the impact on quality of life can be profound. They also discussed navigating a fragmented and relatively undeveloped funding landscape for CGTs.

The discussion further delved into critical considerations unique to CGT funding, including:

• Infrastructure requirements for research and clinical trials

• The necessity of centralized manufacturing capabilities and platform approaches

• The challenges of supporting long-term follow-up studies required for CGT safety monitoring and limited durability data

The audience Q&A addressed pressing concerns from early-career investigators and emerging biotech leaders navigating the complex funding ecosystem, with panelists offering practical advice for securing support at various development stages and for different modalities.

Fireside Chat: Reviving Hope in Deprioritized CGT Programs

ASGCT President Dr. Terence Flotte (UMass Chan Medical School), chaired a fireside chat addressing the critical challenges of discontinued CGT programs that show scientific promise but face commercial viability hurdles. The session explored pathways to revive these treatments from multiple perspectives - patient advocates, academic researchers, industry leaders, and clinical innovators. ASGCT explores these issues and themes further in a forthcoming white paper to be published in Molecular Therapy.

Oralea Marquardt (National Tay-Sachs & Allied Diseases Association) represented the patient community, highlighting the devastating impact when promising therapies for rare diseases are shelved due to commercial considerations. Dr. Donald B. Kohn (UCLA) provided insights from the academic research perspective, discussing how institutional researchers approach the scientific and regulatory challenges to “pick up” programs which have been deprioritized. From industry, Dr. Rachel McMinn (Neurogene) addressed the difficult decisions companies face when balancing scientific promise against financial realities. Finally, ASGCT Board Member Dr. Claire Booth (UCL Great Ormond Street Institute of Child Health) shared successful approaches for advancing deprioritized therapies through innovative academic-industry partnerships and alternative development pathways.

The panel explored several innovative solutions for reviving promising therapies, including:

• Collaborative funding models between patient advocacy groups, academia, and industry

• Flexible regulatory pathways that acknowledge the unique challenges of rare disease therapies; Alternative clinical trial designs appropriate for ultra-rare conditions

• Creative intellectual property agreements that enable continued development when primary sponsors step back

Supporting CGTs: Thinking Outside the Box

Combined, these fireside chats highlighted how the CGT field is rethinking fundamental aspects of therapeutic development to address the unique nature of genetic medicines. And discussions throughout ASGCT's 28th Annual Meeting emphasized the need for continued dialogue and collaboration to ensure that innovative funding approaches and development models can support the next generation of cell and gene therapies. While traditional pharmaceutical development paths often prove inadequate for CGTs, creative solutions are emerging through cross-sector collaboration.

Join us at the 2025 Policy Summit in September to continue exploring strategies that can accelerate CGT development and ensure promising treatments reach the patients who need them!

Caitlin is ASGCT's Manager of Public Policy.