October 2024
Providing timely updates for the patient advocate community!
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Welcome to the debut issue of our NEW patient-focused newsletter.
The American Society of Gene and Cell Therapy (ASGCT) is excited to introduce The Patient Press, a new resource for patients, advocates, and caregivers. We look forward to sharing the latest news, events, and resources on cell and gene therapies with you, six times a year.
In This Issue:
Events
🧬 Empowering Patients 2025: A Cell and Gene Therapies Summit
Registration is open for this inaugural, free virtual event hosted by ASGCT March 12-13, 2025 from 9 a.m. – 12:30 p.m. ET. These two half-days of content are geared toward foundation leaders, patients, and caregivers. Here's an overview of each day's sessions:
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Day 1: Programming will include an overview of gene therapy approaches and clinical trials for rare diseases as well as discussion on the complexity of clinical trial design, and pathways forward for discontinued trials
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Day 2: On the second day, enjoy a morning coffee chat with the FDA, discussions about platform technologies, a panel of patient advocacy groups offering advice in CGT drug development, and an exploration of goals of care when considering a CGT trial.
🥼 FDA-Partnered Workshop: AAV Class Considerations
ASGCT and FDA will host a joint virtual scientific workshop on Jan. 22 and 23, 2025. This free, public event will take place on two half days and explore class-wide considerations for AAV gene therapies, focusing on immunogenicity and immune suppression challenges.
💻 FDA Webinars
👋🏾 Saying Hi to Our New Friends
ASGCT staff were happy to make connections with many familiar and new faces at the Global Genes Week in Rare in Kansas City and at the NORD Rare Summit in Washington, D.C. We hope to stay in touch and be a resource along your CGT journey, so please explore our Patient Education site and feel free to use and share our materials!
Resource Hub
💭 View Our Latest Community Quick Takes
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Genetic Testing and Clinical Trial Eligibility explores how genetic variants may impact trial eligibility, risk profiles, and immune responses for Duchenne muscular dystrophy (DMD), giant axonal neuropathy (GAN), and Angelman Syndrome.
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Informed Consent provides five tips on being prepared for the important process of ensuring a patient or caregiver understands possible risks and benefits of a clinical trial before giving their permission to participate.
🧬 Defining CGT Terms in Plain Language
A glossary of cell and gene therapy terms is now available. Whether you’re sharing findings or seeking to better understand key concepts, this tool aims to offer some clarity and consistency for patients and the public.
❗ Gaucher Disease Awareness Month
Check out our resources on Gaucher disease and how gene therapy is being researched in clinical trials to deliver a working GBA gene into cells to slow or stop disease progression. This month we recognize the dedicated community working tirelessly to investigate these treatments to improve the lives of those impacted by this disease.
🔬 Need Insights on Being Research Ready?
Our Research Readiness for Rare Diseases symposium from the 27th Annual Meeting is now available to view on-demand. This session was organized by an investigator and clinician as a two-hour jumpstart guide for patient groups preparing their disease for research in gene therapy.
🧫 Latest Research from Advancing CGTs in Cancer Event
ASGCT hosted a scientific conference this month in Philadelphia and you can now view 11 plain language summaries of the scientific abstracts presented during the conference.
Breakthrough Moments
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📢 Upcoming FDA Decision Dates
November 13: PTC Therapeutics' Upstaza will be reviewed for the treatment of patients aged 18 months and older with a confirmed diagnosis of aromatic L–amino acid decarboxylase (AADC) deficiency.
November 16: Autolous Therapeutics' Obe-cel will be reviewed for the treatment of patients with adult acute lymphoblastic leukemia.
📰 Rare in the News
Rare Disease Innovation Hub – FDA launched the hub to better collaborate and communicate across the two centers (CBER and CDER) to provide more uniform responses to sponsors. It focuses on different rare diseases, the vast majority of which do not have any treatment.
Duchenne* – FDA-approved gene therapy Elevidys was granted an expanded label to make it available to all Duchenne muscular dystrophy patients at least four years of age who have specific genetic mutations. To learn more about the latest research on gene therapy for Duchenne and other forms of muscular dystrophy, register for our upcoming Breakthroughs in Muscular Dystrophy hybrid event next month.
Dup15q syndrome – News of a canceled trial stunned parents and children enrolled in the phase 2 study, along with the academic researchers running it.
Huntington’s disease* – New data is available from two small gene therapy trials testing low and high doses over two years. In patients with early Huntington’s disease, the larger dose of AMT-130 slowed disease progression by 80% compared to a “natural history cohort” of patients. Patients receiving that dose had about the same motor and cognitive function as when the study began.
KIF1A mutation – STAT News highlighted the story of Susannah Rosen and the inspiring journey of a family working to treat their daughter’s ultra-rare condition. The article shares Susannah’s story and dives into whether personalized antisense oligonucleotide (ASO) therapies can be made at scale for other rare diseases.
LCA1* – Data from the first-ever gene therapy clinical trial for Leber congenital amaurosis 1 (LCA1), a rare inherited blindness, showed significantly improved vision at 12 months post-treatment.
Sanfilippo syndrome* – Ultragenyx announced that FDA will recognize the biomarker heparan suflate (HS) as a surrogate endpoint in gene therapy trials. With that agreement, Ultragenyx is preparing to file for approval of its AAV gene therapy around the end of the year.
Sickle cell disease* – Nearly a year on from the first approvals of gene therapies for sickle cell disease, The New York Times profiled Kendric Cromer as one of the first children to be treated. While “the first day of his new life” has dawned, many other patients face obstacles to receiving these innovative therapies.
Synovial sarcoma – FDA's approval of Tecelra is the first gene therapy approved for adults with metastatic synovial sarcoma and the first FDA-approved T cell receptor (TCR) gene therapy.
*Learn more
Visit ASGCT's patient-friendly resources on gene therapy for these diseases:
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