The Advocate

November 2024: Volume 4, Issue 11

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In This Issue:

Attend a New FDA-Partnered Workshop on AAV Gene Therapies
Read Our Response to FDA’s Draft Strategic Plan on AMT
Serve on a Policy or Outreach Committee in 2025
Submit Feedback on New Cell Therapy Standards 
Patient Outreach Committee Members Recap FDA Listening Session
NCATS Final Strategic Plan Supports CGT Development 
New ASGCT Newsletter Highlights Patients, Caregivers, and Advocates
Events
Policy News

ASGCT Advocacy

Attend a New FDA-Partnered Workshop on AAV Gene Therapies

ASGCT and FDA have joined forces to present a critical scientific workshop addressing one of gene therapy's most pressing challenges: immunogenicity in AAV-based treatments. Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data will convene virtually on Jan. 22 and 23, 2025, from 9 a.m.- 1 p.m. ET. The event will bring together experts from industry, academia, and FDA to tackle complex questions surrounding immune responses to AAV vectors.

The comprehensive program will cover key topics including immunogenicity, strategies for antibody testing, immunosuppression regimens, and approaches to redosing. Through presentations and interactive panel discussions, attendees will gain valuable insights into managing immunogenicity challenges while hearing directly from FDA representatives about regulatory perspectives and expectations. This workshop is a unique opportunity for researchers, clinicians, and regulatory affairs professionals to engage in collaborative discussions on scientific innovation and regulatory frameworks. Register today!

Read Our Response to FDA’s Draft Strategic Plan on AMT

FDA recently released a draft strategy document outlining its plans to facilitate the adoption of innovative manufacturing technologies, such as advanced manufacturing technologies (AMTs), for drugs and biologics including CGTs. This effort is part of the FDA's commitments under the Prescription Drug User Fee Act (PDUFA) VII and the 2022 Food and Drug Omnibus Reform Act (FDORA) to support the use and implementation of innovative manufacturing approaches. 

AMTs such as continuous manufacturing, distributed manufacturing, and novel analytical methods have significant potential to improve product development speed, strengthen supply chains, and prevent drug shortages - key considerations for the rapidly growing CGT field. However, regulatory and technical barriers have slowed broader adoption of these technologies. 

To address these challenges, FDA's draft strategy outlines initiatives such as enhancing the Emerging Technology Program (ETP), collaborating with international regulators on harmonization, and implementing the new Advanced Manufacturing Technologies Designation Program (AMTDP) established by Congress. In addition to previous comments on the AMTDP draft guidance, ASGCT recently submitted a detailed response to FDA’s draft strategy document:  

• AMTDP Independence: ASGCT supports FDA’s AMTDP as a distinct pathway and recommends that AMT eligibility remain separate from the Advanced Technologies Team (CATT) criteria. That would prevent restrictive overlaps and support broader access for innovative CGT manufacturing approaches.  

• Biologics License Applications (BLAs), AMT data, and Drug Master Files (DMFs): ASGCT recommends FDA permit BLAs to cite AMT data in DMFs, which would streamline regulatory submissions and support investment in new manufacturing approaches while addressing intellectual property concerns. 

• Clarification on designation criteria and benefits: ASGCT advocates for clear guidance on eligibility criteria, especially around “substantial improvement” metrics, and calls for a “Graduated AMT” status to maintain regulatory benefits as FDA familiarity with these technologies expands. 

ASGCT emphasized that realizing the full benefits of AMTs will be crucial as the CGT field continues to grow and diversify. The Society looks forward to working with FDA to find solutions to these modern-day challenges.  

Serve on a Policy or Outreach Committee in 2025

ASGCT is now accepting applications for its standing and scientific committees. This is a valuable opportunity for members to become actively involved in shaping the Society's initiatives and to support the advancement of CGT.

Policy and outreach committees offer a unique chance to engage with ASGCT's advocacy efforts. In addition to contributing program proposals for the Annual Meeting, those committees are responsible for:

• The Government Relations Committee develops relationships with government entities, including Congress, NIH, and CMS. They also recommend policy positions that support CGT research, development, and access.
• The Regulatory Affairs Committee educates members on the worldwide regulatory environment and interacts directly with FDA, including through FDA guidance comments, liaison meetings, and other engagement opportunities. They also provide Society representation to entities developing field standards.
• The CMC Committee focuses on the Chemistry, Manufacturing, and Controls (CMC) space, providing focused expertise for guidance comments and other FDA interactions. Along with Government Relations and Regulatory Affairs, the CMC Committee helps plan the annual Policy Summit.
• The Patient Outreach Committee develops a wide range of accessible resources and events that educate on CGT topics, with a focus on patient audiences and other members of the public.
• The Global Outreach Committee develops programs for research collaborations, award opportunities, and outreach efforts with the goal of fostering equitable access to gene therapies throughout the world. The committee focuses on low- and middle-income countries.

Share with your colleagues and apply today! You can find a full list of ASGCT committees and more information on the application process and timeline here.

​Submit Feedback on New Cell Therapy Standards 

The Foundation for the Accreditation of Cellular Therapy (FACT) and the Joint Accreditation Committee ISCT-Europe & EBMT (JACIE) are seeking public comments on two important draft standards documents: the Ninth Edition of Hematopoietic Cellular Therapy Standards and the Third Edition of Immune Effector Cell Standards. Given the critical need to maintain high-quality standards in cellular therapy, we invite stakeholders to review and provide feedback on these drafts by Dec. 15. Key updates include consolidated collection standards, REMS practices, and new audit requirements. Please complete the HCT survey for comments on HCT/combined practices, and/or the IEC survey for IEC-specific feedback. Your input is essential for ensuring these standards effectively serve our field. 

Patient Outreach Committee Members Recap FDA Listening Session

FDA conducted listening sessions with patients and caregivers to gather insights on their experiences and concerns regarding the short- and long-term risks of approved gene therapies. These sessions, organized by the Center for Biologics Evaluation and Research (CBER), aim to develop patient-centered protocols for long-term post-market studies and to understand what information patients find valuable in making treatment decisions. Participants discussed the types of information they would need when choosing gene therapy, their perspectives on potential risks, and their experiences with long-term follow-up studies.

For a more detailed summary on this first listening session, read this piece from Allison Bradbury, PhD, Lauren Beretich, PhD, MS, CGC, and Yulia Grishchuk, PhD, members of ASGCT’s Patient Outreach Committee.

NCATS Final Strategic Plan Supports CGT Development 

ASGCT commends the National Center for Advancing Translational Sciences (NCATS) on its final 2025-2030 Strategic Plan, which reflects meaningful input from the gene and cell therapy community. The strategic plan demonstrates a strengthened commitment to advancing treatments for rare diseases, improving clinical trial diversity, and accelerating translational science. 

ASGCT is particularly encouraged by the plan's enhanced focus on platform technologies, collaboration with regulatory agencies, and strategies to address health disparities. The final plan incorporates several key recommendations from ASGCT, including more robust language around decentralized clinical trials, patient engagement, and the unique challenges of developing therapies for rare diseases. 

ASGCT remains committed to partnering with NCATS and other federal agencies to advance cutting-edge cell and gene therapies that offer hope to patients with currently untreatable conditions. The Society applauds NCATS' forward-looking vision and looks forward to continued collaboration in transforming scientific discoveries into life-changing medical treatments. 

New Newsletter Highlights Patients, Caregivers, Advocates

ASGCT is pleased to announce the launch of The Patient Press, our new bimonthly newsletter dedicated to keeping the patient community informed and engaged in the rapidly evolving CGT landscape. The inaugural edition, launched in October, marks an important expansion of ASGCT's patient-focused initiatives. The Patient Press delivers accessible coverage of CGT fundamentals, upcoming events, regulatory approvals, research breakthroughs, and more.  

The first issue featured the Empowering Patients 2025 Summit, our CGT glossary release, and highlights on research developments in genetic conditions. ASGCT believes that patient perspectives are vital to advancing drug development; this newsletter represents our commitment to providing reliable, actionable information to our patient community. Stay connected with the latest in CGTs by signing up for future editions of The Patient Press.