April 2025
Providing timely updates for the patient advocate community!
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In This Issue:
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A patient advocate guide to ASGCT’s upcoming Annual Meeting
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Latest Community Quick Take covers AAV immune responses to gene therapy
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Did you miss the Empowering Patients Summit? Watch all sessions on YouTube NOW
Events
📢 Patient Advocate Guide to ASGCT’s 28th Annual Meeting
Get ready for an exciting lineup of sessions designed specifically for patient advocates at ASGCT’s Annual Meeting in New Orleans! Don’t miss these key events + sessions:
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Workshop: The Business of Advocates Advancing CGTs – May 13, 8 a.m. – 12 p.m.
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To Biomarker or Not to Biomarker? – May 14, 8 – 9:45 a.m.
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Fireside Chat: Reviving Hope in Deprioritized CGT Programs – May 15, 3:45 – 5:30 p.m.
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BGTC Annual Meeting – May 15, 1:30 – 5:30 p.m.
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Patient Advocate Meet-Up – May 15, 3:15 – 4:15 p.m.
🦓 Show your stripes! Grab a zebra sticker at ASGCT Central (booth 837) to show your support for the rare disease community. While you're there, visit the ASGCT Central impact wall to share what progress for patients means to you, and join our Advocacy team at the Welcome Reception on May 13, 5:30 - 7:30 p.m.
👂 Hear from the Siblings: Sharing Their Journey with Medical Complexity
Join the Courageous Parents Network to watch a panel of individuals who have siblings with medical complexities discuss how this has shaped their lives. They dive into their fears and worries, what has helped, and what has not while supporting their siblings along the journey.
🫁 Attend a Patient-Focused Drug Development Meeting
Join NORD and the PAP Foundation for a powerful virtual externally-led patient focused drug development (EL-PFDD) meeting on Autoimmune Pulmonary Alveolar Proteinosis (aPAP) a rare lung disorder. This is your chance to hear patient and caregiver stories, engage in live Q&A and polling, and provide valuable insights to researchers and drug developers.
Resource Hub
✨ Thank You for Making the Empowering Patients Summit a Success!
All sessions from the Empowering Patients 2025: A Cell and Gene Therapy Summit, are now available to access on YouTube. With 1,000 patient advocates registered, attendees and speakers virtually networked and took part in lively and insightful Q&A sessions. Thank you to the patient advocates, researchers, industry leaders, and regulators who came together to drive meaningful conversations about the future of cell and gene therapy.
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Read a full recap of the Summit, by All My Health, to get a glimpse into the patient driven partnerships and progress discussed throughout.
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Share these sessions with your community! Follow the steps below to share specific portions of Day 1 and 2 of the Summit:
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Pause the video at the exact time you want to share.
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Click the "Share" button below the video.
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Check the "Start at" box (it will show the current time in the video).
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Copy the link provided and send it to your network!
🔎 Unpack AAV Immune Responses
Watch the latest Community Quick Take on the immune system’s role in adeno-associated virus (AAAV) gene therapy! Explore why managing immune responses is especially important for high-dose therapies targeting the brain, and how pre-existing antibodies to AAV vectors affect patient eligibility and treatment outcomes.
👏 ASGCT Ethics Committee Discusses the Evolving CGT Ecosystem
Check out the latest fireside chat, The Big Picture, that addresses data capture for long-term monitoring of safety, standardization to enable comparability to help identify next-generation therapies, and tensions that exist in the evolving landscape.
🎤 Listen: Trust, Innovation, and the Power of Patient Advocacy Podcast
Listen to Brendan Hayes of the National Bleeding Disorders Foundation dive into how patient communities shape the development of new therapies, the historical context that influences decision-making in clinical trials, and the unique considerations facing those who might choose to pursue cell and gene therapies.
📼 FDA’s CDER Releases New Videos for Rare Disease
Learn how to tackle key challenges in data collection and endpoint development with two new videos on Considerations for Collecting/Using Natural History Data Fit for Use in the Regulatory Setting and Understanding Endpoints in Rare Disease Drug Development, published by LEADER 3D’s Accelerating Rare disease Cures (ARC) program, aiming to explore challenges in rare disease drug development.
♥️ April 11 Was World Parkinson’s Disease Day
On International Parkinson's Day, we reflect on the challenges faced by those living with Parkinson's and the promising advances in gene and cell therapy (CGT). Parkinson’s is a progressive neurodegenerative disease that affects movement and quality of life. However, research in CGT is opening doors to potential treatments that could target the underlying causes.
🤝 Thank You, SMA-PME Research!
ASGCT Speaker Bureau member Dr. Rachel Bailey presented at the SMA-PME Research Board Meeting on April 16, covering the preclinical study process for ultra-rare diseases, gene therapy approaches, and actionable steps to accelerate research and raise awareness.
If you’re interested in having a member of the Bureau speak to your patient community on gene therapy research and clinical trial topics, please contact ASGCT's Senior Patient Outreach Manager Ali Kujawski at akujawski@asgct.org.
Breakthrough Moments
☑️ CGT Approvals
Encelto was approved for adults with neurodegenerative retinal disease, macular telangiectasia type 2 (MacTel). It is a first-of-its-kind implantable cell therapy, as well as the first and only approved treatment for this disorder.
🔜 Upcoming PDUFA Dates
Abeona Therapeutics COL7A1 is an investigational autologous gene therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). FDA accepted Abeona’s Biologics Licensing Application (BLA) resubmission and is expected to make an approval decision by April 29.
🏛️ Policy Updates
ASGCT on Capitol Hill - ASGCT leaders and staff have been active in Washington, D.C., meeting with House and Senate offices to discuss the value of gene therapies and the importance of NIH funding for biomedical research. The Society also held an educational briefing in early April for congressional staff on gene editing technologies. ASGCT looks forward to continuing these conversations with congressional leaders, stressing the negative impact of policy disruption for patients.
HHS Announces Staff Cuts at Key Health Agencies - The U.S. Department of Health and Human Services (HHS) will reduce its workforce by 10,000 more employees in addition to those who had already left in recent months, bringing total cuts to about 25% of staff. There have been conflicting reports on whether HHS plans to rehire some employees. The impacts on gene and cell therapy-related agencies includes:
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FDA: 3,500 jobs have been cut, including a large number of policy and communications roles. Reviewers and inspectors were exempt, but the staff who support their jobs were not.
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NIH: The National Institutes of Health (NIH) will decrease its workforce by approximately 1,200 employees, including the directors of five institutes and other senior leaders.
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CMS: Around 300 roles will be eliminated.
NIH Funding Update - At this time, the NIH budget is set through Sept. 2025. The Institutes are working at funding levels originally set in 2023; the effects of inflation make that an effective cut. As Congress turns to funding for the 2026 Fiscal Year, ASGCT is engaging with appropriations leaders to encourage robust NIH funding. As part of that conversation, ASGCT is closely monitoring NIH facilities & administration funding (F&A, also known as indirect costs). For more information about the F&A system, you can read ASGCT's March statement on the proposed cuts. ASGCT is pleased that the F&A rate system has been reaffirmed for now via congressional action and a federal judge’s ruling, but there may be future efforts to reintroduce the cuts.
HHS Dissolves Federal Newborn Screening Panel - HHS also dissolved the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), which previously guided state-level newborn screening (NBS) recommendations. This federal panel's closure raises concerns as it may delay identification of eligible patients for early treatment. Decisions were anticipated soon on screening for metachromatic leukodystrophy and Duchenne muscular dystrophy, both conditions with recent gene therapy approvals. ASGCT has previously engaged with the ACHDNC to support NBS through the Recommended Uniform Screening Panel, and will continue to monitor how this closure may impact patient identification and access.
📰 Rare in the News
Blood Cancer - Orca Bio announced that their experimental T-cell therapy significantly reduced the risk of chronic graft-versus-host disease (GVHD) by 74% in a Phase 3 study, offering a potential breakthrough for blood cancer patients. They plan to submit for FDA approval later this year.
Duchenne Muscular Dystrophy (DMD)* - A 16-year-old patient died from liver failure after receiving Sarepta's Elevidys gene therapy for Duchenne muscular dystrophy, the first known fatality linked to the treatment. Clinical trials in Europe have been paused, but American trials are continuing.
Duchenne Muscular Dystrophy (DMD)* - REGENXBIO reported positive biomarker data from the first patient under 4 years old treated with its DMD gene therapy had higher micro dystrophin expression than in older patients. The company plans to seek accelerated approval for RGX-202 next year, aiming to expand treatment to children as young as 1.
Genetic Hearing Loss (GHL)* - Regeneron reported hearing recovery in key speech frequencies of 12 children, and improved speech perception in 1 during the Phase 1/2 CHORD trial for genetic hearing loss due to otoferlin (OTOF) gene variants. The therapy was well tolerated, with no serious adverse events linked to the drug.
Heart Disease - Verve Therapeutics reported results from a new gene editing therapy called VERVE-102, designed to lower “bad” cholesterol (LDL) in people with inherited high cholesterol or early heart disease. The therapy also showed no serious side effects in the patients studied, marking an important milestone in the safety of gene editing treatments.
Inherited Retinal Disease (IRD)* - MeiraGTx reported significant vision improvements for children born legally blind due to AIPL1-associated severe retinal dystrophy after treatment with their experimental gene therapy. Beyond vision, the therapy has shown benefits in communication, schooling, and social integration.
Lung Disease - Beam Therapeutics reported promising Phase 1/2 results in their gene-editing treatment for alpha-1 antitrypsin deficiency (AATD) lung disease, showing a reduction in mutant proteins and an increase in corrected proteins in nine patients with the condition.
Spinal Muscular Atrophy (SMA) - Investigators reported older children with SMA aged 2–17 showed significant improvement in muscle function after receiving Novartis’ Zolgensma gene therapy, compared to those given a sham treatment. These results suggest that Zolgensma, previously approved only for infants, may benefit a broader age range of patients.
*Learn more
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