Regenxbio’s Duchenne Gene Therapy Shows Success in Trial, FDA Submission Next
Regenxbio announced positive results from its Duchenne muscular dystrophy gene therapy trial, achieving high levels of a critical muscle protein. The...
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Gene Therapy Viruses Linked to Rare Tumor in Young Patient: FDA Commissioner Resigns Amid Safety Concerns
FDA Commissioner Marty Makary resigns after reports link gene therapy viruses to a boy's tumor. Acting Commissioner Kyle Diamantas to temporarily lead...
New Study Warns: Common Drug May Reduce Gene Therapy Effectiveness in Dravet Syndrome Patients
A new trial by Encoded Therapeutics reveals that sirolimus, a drug used to prevent immune rejection in gene therapy, may also diminish treatment effec...
Rare Gene Therapy Side Effect: Toddler Develops Tumor Linked to Treatment
A 13-month-old boy, Adam, developed a golf-ball sized brain tumor after undergoing experimental gene therapy for a rare disease. His parents chose the...
Capsida Biotherapeutics Unable to Determine Cause of Child’s Death in Gene Therapy Trial
Capsida Biotherapeutics remains uncertain about the cause of a child’s death in a gene therapy trial conducted last September. The investigation has s...
Astellas' Breakthrough Treatment Revives Hope for Rare Disease Patients and Families
Astellas Pharma's experimental treatment for a rare, devastating disease has reignited hope among patients and advocates. The breakthrough comes amid...
FDA Leadership Shake-Up Sparks Hope in Gene Therapy Sector Amid Rome Conference
Gene therapy leaders gathered in Rome last week, where optimism stemmed from recent rare disease treatment approvals and the impending departure of FD...
Latus Bio Secures $42M to Launch Huntington’s and Batten Disease Gene Therapy Trials
Latus Bio, a gene therapy startup, has raised $42 million to advance two clinical trials in 2024, including treatments for Batten disease and Huntingt...
FDA Approves Groundbreaking Gene Therapy Restoring Hearing in Deaf Children
The FDA has approved Otarmeni, a gene therapy for severe-to-profound hearing loss caused by OTOF gene mutations. Clinical trials showed 80% of treated...
Mother of Girl Who Received Bespoke Gene Therapy Launches New Biotech to Scale Treatments
Julia Vitarello, whose daughter Mila received a custom gene therapy eight years ago, is launching a new biotech to scale bespoke treatments. Her previ...
Breakthrough Five-Mechanism Obesity Drug Shows Promise in Mice, NIH Funding Hits Record Low
A next-generation obesity drug combining five mechanisms has demonstrated superlative effectiveness in mice, potentially surpassing GLP-1 drugs. Meanw...
CRISPR Breakthroughs Offer Hope for Down Syndrome and Genetic Disorders Treatment
A groundbreaking gene therapy from Regeneron Pharmaceuticals restored hearing in 80% of deaf children, including a 18-month-old patient who can now he...
KFF Health News Minute: Key Health Policy Updates and Trends in 2024
The KFF Health News Minute delivers weekly insights on critical health policy shifts, from Title X changes to Medicare coverage expansions. Recent epi...
Eli Lilly Partners with AI Biotech Profluent to Advance Next-Gen Gene Editing Technology
Eli Lilly has partnered with AI-focused biotech Profluent to develop advanced gene editors capable of inserting entire genes into patients. The deal i...
Astellas Revives XLMTM Gene Therapy Trial After Patient Deaths: Key Biotech Updates
Astellas is reentering clinical trials for its next-generation XLMTM gene therapy following patient deaths. Eli Lilly acquires Ajax Therapeutics for u...
Intellia's CRISPR Gene Therapy Lonvo-z Slashes Swelling Attacks in Rare Disease Trial, Paving Way for Approval
Intellia Therapeutics announced Monday that a single dose of its CRISPR-based gene editing treatment, lonvo-z, dramatically reduced swelling attacks i...
Sanofi's Research Priorities Under Scrutiny as New CEO Belén Garijo Takes Charge
Sanofi's research priorities are under review as new CEO Belén Garijo begins her tenure. The company's recent focus on immunology has yielded lacklust...
Groundbreaking Gene Therapy Trial Shows 90% Hearing Improvement in Deaf Children
A groundbreaking gene therapy trial in China has demonstrated remarkable success, with 90% of deaf children showing significant hearing improvement. T...
KFF Health News Minute: Key Health Policy Updates and Trends Explained
The KFF Health News Minute delivers weekly updates on critical health policy developments. Recent episodes cover rising healthcare costs, Medicaid acc...