August 2025
Providing timely updates for the patient advocate community!
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In This Issue:
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Check out U.S. federal government/non-profit registration rates at the Policy Summit
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Catch up on the field with ASGCT’s Q2 2025 Gene, Cell, & RNA Therapy Landscape Report
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Latest Community Quick Take explores the need-to-knows of CGT manufacturing
Events + Engagements
📌 Join Us In Person at the Policy Summit Sept. 25-26
Join us in person at the 2025 Policy Summit in Washington, D.C. at the Washington Marriott Georgetown Sept. 25-26, 2025. Hear from speakers across government, academia, and industry who will share insights on emerging issues in the cell and gene therapy field and explore innovative solutions.
NEW: This year the Policy Summit offers a registration discount for U.S. federal government employees and nonprofits. Early-bird rates end August 29. Don’t miss the chance for face-to-face networking with CGT leaders shaping the future of the field.
⭐ Attend the NORD Breakthrough Summit
Register for the 2025 NORD Breakthrough Summit, October 19-21, in Washington D.C. Following the theme From Voices to Breakthroughs, it is a must-attend event for companies and individuals passionate about rare disease research and orphan product development. ASGCT staff will have a booth in the exhibition hall.
💻 Register for Free Workshops from FDA + Duke Margolis Institute for Health Policy
🎤 Participate in Public Listening Session from FDA’s Office of Therapeutic Products
Register for a public listening session on September 18 to hear input from CGT sponsors on leveraging internal and public knowledge, including CMC, nonclinical, and clinical data, across therapeutic contexts. Interested parties, including patients and advocacy groups are encouraged to submit written comments by October 17, and share their perspectives on how FDA approaches data sharing.
📖 Congressional Briefing Addresses Barriers to Treatment for SCD Patients
Read a summary of ASGCT’s recent Congressional briefing, "Addressing Equitable Access to Gene Therapies for Children with Sickle Cell Disease." The briefing was sponsored by Sens. Tim Scott (R-SC), Raphael Warnock (D-GA), and Cory Booker (D-NJ), and hosted in partnership with the Children’s Hospital Association. It featured clinicians and other stakeholders exploring the access limits faced by SDC warriors and how Congressional action can help to alleviate those barriers.
Resource Hub
📣 New Resource! Community Quick Take on Why Manufacturing Matters
Watch Drs. Steven Gray and Angelica Meyer in the latest ASGCT Community Quick Take. Moderated by Dr. P.J. Brooks, they explain the vital role of plasmids, the importance of quality checks, and the typical timelines it takes to make a batch of AAV vectors for use in clinical trials. Content was developed in partnership with the Bespoke Gene Therapy Consortium (BGTC), a public-private partnership devoted to removing manufacturing and regulatory bottlenecks for AAV gene therapies.
Coming soon: on August 27 the next Community Quick Take will be available featuring Dr. Amy Waldman with insights on Preparing for the Unknowns of CGT clinical trial participation. You can find previous CQTs on our Patient Education site.
🧬 Listen: Developing Therapeutic Platforms with Dr. P.J. Brooks
Tune in to a conversation with Dr. P.J. Brooks Deputy Director at NIH’s NCATS Division of Rare Diseases Research Innovation, as he shares insights into how developing cell and gene therapies as platforms could accelerate treatments for multiple rare diseases, the projects advancing this approach, and what’s giving him hope about the future of the field.
♥️ August is SMA Awareness Month
Share our educational resources to explore how cell and gene therapy works for Spinal Muscular Atrophy (SMA). We recognize the strength of the SMA community, and invaluable contributions that have been made to advance treatments like gene therapy. SMA is caused by a single faulty gene, making it a good candidate for gene therapy which delivers a working copy of the SMN1 gene directly into motor neuron cells. Newborn screening and approved gene therapy treatments are available, showing significant advancements towards improved outcomes in the SMA community.
🗣️ Thank You, HNRNP Family Foundation, Rare Village, and ZMYND11 Treatment Foundation
The ASGCT Speaker Bureau collaborates with Patient Advocacy Groups to advance the understanding of cell and gene therapies.
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ASGCT Speaker Bureau, and Board of Directors member, Dr. Allison Bradbury, gave a live presentation during the HNRNP Gene Family Meeting in July on “Clinical Trial Readiness.” She aimed to demystify the science of ASOs and other gene therapy approaches, along with sharing the realities of participating in research.
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Thank you to ASGCT Board Member Dr. Kim Goodspeed, Chair of the Patient Outreach Committee and Speaker Bureau member, for leading Rare Village Foundation through an explanation on gene therapy basics, clinical trials, and the power of patient data.
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ASGCT also teamed up with the ZMYND11 Treatment Foundation, to provide an insightful background on genetics from Dr. Nicolas Abreu.
🔬 Download the Q2 Gene, Cell, + RNA Therapy Landscape Report
Check out the recently released 2025 quarterly landscape report which highlighted FDA’s approval of Zevaskyn for recessive dystrophic epidermolysis bullosa (RDEB) and mNexspike for COVID-19. Notably, 64% of the 80 new gene therapy trials initiated were for oncology indications, the highest proportion in the past year.
‼️New Glossary Helps Clarify How Clinical Trials Work
FDA and NIH released the MoRE Glossary, a new set of clear, shared definitions for how clinical trials are designed and discussed. It explains terms like real-world data, umbrella trials, and synthetic data - especially important for CGT treatments, as they often use innovative trial designs.
For additional resources, check out our Patient Education website, which includes a glossary of key CGT terms.
Breakthrough Moments
✔️ Recent CGT Approvals and Designations
Precigen announced FDA approval of Papzimeos (zopapogene imadenovec-drba), an investigational immunotherapy for the treatment of individuals with recurrent respiratory papillomatosis (RRP), an HPV-related disorder with no cure. Read our statement from ASGCT President Terry Flotte, MD on the approval.
🏛️ Policy Updates
Round Up of Medicaid Changes Affecting Patients – Recent passage of the One Big Beautiful Bill Act (HR1), also known as reconciliation, will bring significant changes to Medicaid, expected to impact over 10 million recipients. For rare disease patients and families, the key elements to be aware of include:
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Work Requirements: Starting December 2026, most adults under 65 will need to work, volunteer, or go to school at least 80 hours per month to keep their Medicaid coverage. This doesn't apply to seniors, people with disabilities, or parents with young children under 14.
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More Frequent Eligibility Checks: Beginning in 2027, states will check if participants qualify for Medicaid every six months instead of once a year. This could mean more paperwork and the potential of losing coverage due to missed deadlines or documentation issues.
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New Co-pays: People with incomes above the federal poverty line will now have to pay out-of-pocket costs for most medical services like doctor visits and lab tests. Participants could pay up to 5% of their yearly income. Notably, primary care and mental health services are exempt.
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Reduced State Funding: States will have less flexibility to use certain funding mechanisms (like provider taxes) to get additional federal support for their Medicaid programs. That could lead to reduced services or eligibility in some areas.
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Impact on Rural Areas: The bill includes a $50 billion Rural Health Fund to partially offset Medicaid cuts for rural hospitals and healthcare services. Individual states must apply for the funding, which is set to expire in 2032.
Updates on National Newborn Screening System - The federal Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) was disbanded earlier this year; the committee had guided states on which conditions to screen for, which is important for early diagnosis and treatment. In recent weeks there have been several updates on the path forward. First, the Health Resources and Services Administration (HRSA) has issued a request for comment on adding Duchenne muscular dystrophy (DMD) to the Recommended Uniform Screening Panel (RUSP) without the ACHDNC. Second, EveryLife Foundation for Rare Diseases is hosting a virtual Newborn Screening Evidence Review Workshop on September 12. The workshop gives clinical and patient communities a chance to discuss completed DMD and Metachromatic Leukodystrophy (MLD) nomination packages with newborn screening experts. Finally, a recent House Energy & Commerce subcommittee held a hearing on the Newborn Screening Saves Lives Reauthorization Act, with bipartisan support for early screening access.
FDA Developing Plausible Mechanism Pathway for Earlier Rare Disease Approvals - FDA Commissioner Marty Makary confirmed that work is underway on a proposed “plausible mechanism” approval pathway, aimed at allowing certain products, particularly for rare diseases, to receive earlier approval if they demonstrate a scientifically plausible mechanism of action. Details on eligibility and implementation have not yet been disclosed.
CGT Access Model Participating States Announced - CMS announced that the Cell and Gene Therapy (CGT) Access Model will include 33 states, the District of Columbia, and Puerto Rico, all offering outcomes-based Medicaid coverage for sickle cell gene therapies. Those states include roughly 84% of Medicaid beneficiaries with sickle cell disease.
Apply for a New FDA Funding Opportunities for Rare Disease Research - The FDA’s Office of Orphan Products Development (OOPD) has announced new funding opportunities to accelerate rare disease research. One supports clinical trials evaluating safety, efficacy, and potential new indications, while the other funds innovative natural history studies to address unmet needs. Through the Orphan Products Grants Program, FDA aims to advance collaborative research, expand treatment options, and improve outcomes for the rare disease community.
Changes to Orphan Drug Medicare Price Negotiations - Recent legislation broadened the Inflation Reduction Act's orphan drug exemption to now exclude drugs that treat multiple rare diseases from Medicare drug price negotiations. This change removes earlier rules that only excepted drugs treating a single rare disease.
🗞️ Rare in the News
Autoimmune Diseases* - Orbital Therapeutics released preclinical data showing its in vivo CAR-T therapy successfully eliminated B cells in the blood, spleen, and lymph nodes of monkeys without the need for chemotherapy preconditioning typically required for commercial CAR-T treatments.
Blood Cancers* - The FDA announced it has eliminated Risk Evaluation and Mitigation Strategies (REMS) requirements for currently approved BCMA- and CD19-directed CAR-T cell immunotherapies used to treat blood cancers. Patients now only need to stay near a healthcare facility for two weeks instead of four, and follow-up monitoring no longer has to happen exclusively at certified centers. This change means more flexibility, less strain on patients and families, and quicker delivery of potentially curative options
Cerebral Adrenoleukodystrophy* - FDA has restricted the use of bluebird bio’s gene therapy Skysona to boys with cerebral adrenoleukodystrophy (CALD) who lack a stem cell donor or other treatment options.
Duchenne Muscular Dystrophy (DMD)* - Sarepta Therapeutics announced the FDA lifted its voluntary pause on shipments of ELEVIDYS, the only approved gene therapy for Duchenne muscular dystrophy, for ambulatory patients. The pause, initiated to allow a safety review, was resolved after the FDA determined the therapy was not linked to a reported patient death in Brazil. Discussions continue on risk mitigation for non-ambulatory patients.
Metabolic and Immune Disorders - The Chan Zuckerberg Initiative is funding a new Center for Pediatric CRISPR Cures with UC Berkeley to develop personalized gene-editing therapies for children with ultra-rare immune and metabolic disorders. Inspired by “Baby KJ’s” success, the center aims to create custom CRISPR treatments for eight children over the next three years.
Sanfilippo / MPS III* - FDA issued Ultragenyx a complete response letter (CRL) for its AAV gene therapy UX111, which targets Sanfilippo syndrome type A, a rare pediatric neurodegenerative disease. FDA cited manufacturing issues following a recent inspection. The company announced plans to address facility-related concerns and resubmit for potential approval in 2026.
*Learn more
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