Regulatory Innovation Paves the Way for Faster Access to Life-Saving Cell and Gene Therapies

At ASGCT's 28th Annual Meeting, a slate of sessions highlighted how regulatory innovation is enabling the cell and gene therapy (CGT) field to deliver faster access to life-saving treatments for patients with rare diseases. From FDA's START Pilot Program to global harmonization efforts, these innovations collectively address barriers to development and accelerate patient access to transformative therapies. 

The START Pilot Program: Accelerating Rare Disease Treatments 

The symposium "FDA's START Pilot Program in Action: Insights from Year One" featured outcomes from participating companies: Neurogene, Moderna, and Myrtelle. Representatives from these organizations demonstrated how the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot has advanced their development timelines through scheduled meeting cadences, timely responses, and collaborative problem-solving. In some cases, development timelines have shortened by several years, highlighting the critical importance of continued FDA support for timely access to rare disease care. 

Neurogene’s Founder and CEO Rachel McMinn, PhD, described how START enabled early alignment with FDA on registrational trial design for its NGN-401 gene therapy for Rett syndrome. Interim data showed clinically meaningful gains across motor, communication, and autonomic domains, helping the company move toward a planned BLA update in the near future. Susan Telliard, representing Moderna, shared how START improved the pace and quality of regulatory dialogue for its mRNA-3705 therapy for methylmalonic acidemia (MMA). According to Susan, rapid feedback helped FDA and Moderna agree on the pivotal study design, and she shared the company expects to start a registrational study in 2025. Adrian Stecyk, CEO of Myrtelle, reported that START support for their MYR-101 of Canavan disease led to key regulatory agreements, including use of a natural history comparator and a single-trial approval path. Adrian shared encouraging clinical results and manufacturing readiness position the program for potential commercial launch by 2026. 

Global Regulatory Perspectives: Breaking Down International Barriers 

 The "Fireside Chat: Global Regulatory Perspectives" brought together both current and former senior leaders from regulatory authorities across the US, Australasia, and Japan for a discussion on opportunities and challenges in aligning global approaches to CGT oversight. 

Panelists Ian Alexander, PhD, MD, of Syndey Children’s Hospitals Network, Yoshiaki Maruyama, Japan’s Pharmaceutical and Medical Devices Agency (PMDA), and former FDA Adnan Jaigirdar, MD, FACS shared how their respective regions are adapting regulatory frameworks to support CGT development, highlighting recent updates, collaborative initiatives, and region-specific priorities. The discussion emphasized the importance of reducing unnecessary duplication in development and review with Dr. Alexander noting, “There’s what we call clinical trial notification, where the Australian regulators do not re-review something that’s already been reviewed by what they regard as a comparable overseas regulator. And I think that’s a strength - because if you had a clinical trial that had been approved by the FDA in the US and you had a trial site here, there’s a high probability our regulator would be comfortable.” 

While recognizing that regulatory diversity reflects national needs and contexts, speakers underscored the value of continued dialogue and convergence where possible - particularly around shared scientific principles. Rather than aiming for uniformity, the conversation highlighted how mutual understanding, and strategic alignment can support efficient global development without compromising regulatory independence. 

This dialogue segued to the "Global Convergence: CMC Harmonization for Advanced Therapies" symposia, where industry leaders tackled the challenge of navigating disparate regulatory requirements. Yoko Momonoi from Takeda provided an overview of key differences across global jurisdictions, including divergent donor eligibility testing standards, region-specific GMO regulations, and inconsistent GMP expectations for raw materials. Mitchell Tai of Bristol Myers Squibb shared case studies from Breyanzi’s global lifecycle management, and how synchronized and sequential filing strategies must adapt to jurisdictional timelines and testing protocols. Sara Mills from Artiva emphasized how developing modular CMC documentation and implementing regional appendices can maintain global alignment while allowing for local flexibility. 

Together, these sessions highlighted how greater alignment across regulatory systems can unlock more efficient global development pathways and help extend patient access to advanced therapies, including in smaller markets. 

Implementing New Guidance for CGT Success 

The comprehensive Annual Meeting workshop "Regulation Ready: Key Compliance Updates and What They Mean for CGT Development" complemented these global discussions by focusing on practical implementation of recent FDA guidance. The workshop featured the following speakers: Kimberly Benton, PhD, of Dark Horse Consulting Group; Christopher Murphy of Akron Biotech; George Buchman, PhD, of Catalent; John Tomtishen of Cellares; Houman Deghani, PhD, of Cabaletta Bio; and Ann Lee, PhD, of Prime Medicine. Speakers provided actionable insights on meeting evolving expectations for product safety, potency, manufacturing changes, and platform technologies. 

Speakers emphasized early qualification of human- and animal-derived materials, clear potency strategies tied to mechanism of action, and robust comparability planning using totality-of-data approaches. Case studies shared by Dr. Ann Lee and Dr. Houman Deghani illustrated how modular platforms and early assay development can streamline clinical development. Dr. George Buchman and John Tomtishen discussed how FDA’s new designation programs for platform technologies and advanced manufacturing are creating regulatory efficiencies - highlighting how Cellares’ automated Cell Shuttle system earned AMT designation and how Catalent’s UpTempo AAV platform supports accelerated timelines through standardization. 

Together, the speakers reinforced that early alignment with FDA and cross-functional risk assessment are key to navigating regulatory complexity and advancing cell and gene therapies more efficiently. 

Unifying Vision: From Innovation to Access 

These interconnected sessions revealed a cohesive vision for regulatory innovation in service of patient access. Key themes emerged across all discussions: 

• Enhanced collaboration and harmonization between regulators and industry worldwide, including practical approaches to navigating divergent global CMC requirements 

• Flexible, fit-for-purpose regulatory engagement models that accelerate rare disease development through programs like START 

• Practical implementation strategies for new regulatory frameworks 

• Expanded regulatory pathways that recognize platform technologies and advanced manufacturing as tools for greater efficiency and scalability 

The insights shared at ASGCT's 28th Annual Meeting show how regulatory innovation, from specialized programs to international harmonization, creates new pathways for bringing transformative therapies to patients faster while maintaining rigorous safety standards. Recorded sessions are available on-demand through July 18, 2025. This momentum will continue at the 2025 Policy Summit, September 25-26, where stakeholders will further explore evolving regulatory frameworks to support the next generation of cell and gene therapies. Registration is now open for this highly informative event! 

Andrew is ASGCT's Regulatory Affairs Manager.

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