The Advocate

January 2025: Volume 5, Issue 1

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In This Issue:

Next Week: FDA-partnered Workshop on Immunogenicity for AAV Gene Therapies
Policy Fellowship Applications Open Through Feb. 24 
Shape ASGCT’s Policy and Advocacy by Volunteering for a Committee  
Continuing Resolution Funds Key CGT Agencies Through Mid-March 
Cures 2.1 White Paper Charts the Future of Biomedical Innovation 
Looking Back at a Year of ASGCT Advocacy  
Read Our Summary of CBER Patient-Focused Listening Meeting
Call for Participation: Share Your Insights on Gene Therapy Regulation 
Events
Policy News

ASGCT Advocacy

Next Week: FDA-partnered Workshop on Immunogenicity for AAV Gene Therapies 

Don't miss this unique opportunity to engage with leading experts in AAV gene therapy development. ASGCT and FDA are co-hosting a free virtual workshop Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data on Jan. 22-23, from 9 a.m. - 1 p.m. ET. 

This dynamic workshop assembles prominent voices from FDA, academia, and industry to tackle pressing challenges in AAV gene therapy development. Expert speakers including Dr. Terence Flotte (UMass Chan Medical School), Dr. Barry Byrne (University of Florida), Dr. Andrew Byrnes (FDA), and Dr. Louise Rodino-Klapac (Sarepta Therapeutics) will lead discussions on critical topics from immunogenicity and preexisting immunity to redosing strategies and evidence-based immunosuppression approaches. 

Each session features interactive panel discussions where you can engage directly with experts, gain insights from FDA reviewers, and learn about cutting-edge research and development strategies. This collaborative event offers a unique chance to help shape the future of AAV gene therapy alongside leaders from across the field – register now! 

Policy Fellowship Applications Open Through Feb. 24 

Apply for ASGCT’s Congressional Policy Fellowship by Feb. 24! In partnership with AAAS, we'll selected one applicant for a year-long policy fellowship in a legislative office on Capitol Hill beginning Sept. 2025. Fellows will provide high-quality, science-based, independent guidance to federal policy makers and elevate awareness of the Society and the field. ASGCT’s first Fellow, Dr. Adrian Bankston, has shared her initial experiences and how science policy has shaped her career. A summary of application materials and FAQ are available, so take a look and apply now. 

Shape ASGCT’s Policy and Advocacy by Volunteering for a Committee  

ASGCT is accepting applications for standing  and scientific committees through Jan. 17. This is a valuable opportunity for members to become actively involved in shaping the Society's initiatives and to support the advancement of CGT. Share with colleagues interested in making meaningful contributions to advance ASGCT’s mission! 

Continuing Resolution Funds Key CGT Agencies Through Mid-March 

Just before the December funding deadline, Congress passed a Continuing Resolution (CR) to extend federal funding through March 14. This temporary measure ensures the government can continue operating while maintaining funding at prior-year levels for key agencies like the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). The CR preserves the existing budget with minimal adjustments, providing stability but limiting opportunities for expansion or new initiatives. 

Outgoing NIH Director Monica Bertagnolli has cautioned that flat funding could hinder biomedical innovation and discourage emerging researchers. Similarly, the FDA’s ability to launch new programs to accelerate CGT approvals remains constrained under the current funding levels. Adding to these challenges, the CR did not reauthorize the Rare Pediatric Disease Priority Review Voucher Program, a critical tool for incentivizing treatments for rare diseases. 

This CR sets the stage for critical advocacy opportunities as Congress faces a mid-March deadline to pass a comprehensive appropriations package, or another stopgap measure. ASGCT will continue to emphasize the importance of sustained, predictable funding and renewed incentives for innovation, ensuring that the biomedical research community can deliver CGTs for patients worldwide. 

Cures 2.1 White Paper Charts the Future of Biomedical Innovation 

U.S. Representatives Diana DeGette and Larry Bucshon, MD released a new white paper outlining a vision for "Cures 2.1," the next iteration of landmark biomedical research legislation building on the 21st Century Cures Act and Cures 2.0 Act. The white paper, developed following hundreds of stakeholder responses to their summer 2024 Request for Information (RFI), proposes several key priorities including modernizing Medicare coverage policies, expanding access to novel technologies, enhancing real-world evidence research, bringing clinical trials to nontraditional settings, and ensuring equitable access to cutting-edge therapies. 

ASGCT submitted a comprehensive response the original RFI, highlighting critical issues facing the CGT field such as Medicaid coverage barriers, manufacturing challenges, and the need for enhanced FDA-CMS coordination. The Society emphasized the importance of consistent coverage policies across state Medicaid programs, flexible manufacturing guidelines that consider the unique aspects of CGTs and streamlined processes for implementing novel payment arrangements. 

The Society will continue to engage with new and returning members of Congress as Cures 2.1 develops, ensuring that the perspectives of the CGT research community help shape policies that foster innovation, reduce barriers to patient access, and support the clinical application of transformative therapies. 

Looking Back at a Year of ASGCT Advocacy  

In 2024, ASGCT made significant strides in advancing CGT policy and advocacy. Key highlights include: 

• Successfully influencing final FDA guidance on genome editing and CAR T-cell products.  

• Establishing the first Congressional Policy Fellow to integrate scientific expertise into legislative processes. 

• Providing expert testimony during Rare Disease Week and hosting educational congressional briefings on critical CGT topics.  

• Publishing policy-focused articles in the Molecular Therapy journal family. 

• Engaging directly with legislative offices and key policy stakeholders to educate decision-makers about gene editing, HIV, and hemophilia, and more.  

• Submitting comprehensive feedback on multiple draft guidance documents, requests for information, and payment policy proposals. 

For a full review of ASGCT's 2024 achievements, we encourage members to read the policy blog post on our website. To learn more about our upcoming policy priorities for 2025 and ways to get involved, please visit our Advocacy homepage or consider joining a standing committee. 

Read Our Summary of CBER Patient-Focused Listening Meeting  

During the recent FDA CBER Patient Listening Session, patients and caregivers shared powerful stories about the urgency of early, and adult, enrollment into gene therapy clinical trials. Their insights emphasized the critical importance of acting swiftly to preserve function rather than focusing on repair of irreversible damage. It also explored the challenges and hopes these families face, from the emotional toll of navigating life-changing decisions to the need for increased transparency and ongoing communication about consent throughout the clinical trial journey. Read more in the recap of the session on our blog and watch the full recording.  

Call for Participation: Share Your Insights on Gene Therapy Regulation 

Gene therapies hold immense promise for revolutionizing healthcare by addressing genetic disorders. However, the field’s complexity and evolving regulatory landscape pose significant challenges, particularly when navigating diverse global requirements. To tackle these hurdles and shape harmonized regulatory strategies, your input is crucial. 

The Hertfordshire University Pharmacy School is conducting a research survey to explore global cell and gene therapy regulatory concerns. ASGCT members are invited to share their valuable insights. The survey is anonymous and takes no more than 5 minutes to complete. 

Take the survey 

Your feedback will help identify key areas of concern and drive meaningful progress in the field. Thank you for your participation!