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LATEST NEWS
FDA Proposes One Approval for Many Mutations in Personalized Gene Editing
A new FDA draft guidance would let one license cover a personalized gene editor and its patient-specific versions. New mutations could be added with lab data instead of new clinical trials, a path built for cases like KJ Muldoon's.
Oct 294 min read
Estuary Launches With $103 Million to Bring Gene Therapy to Polycystic Kidney Disease
Estuary Biotherapeutics launched with $103 million to develop one-time, kidney-directed AAV gene therapies for ADPKD, the most common inherited kidney disease. Here is how restoring polycystin proteins could stop, or even reverse, kidney cysts.
Oct 224 min read
Should Gene Therapy Be Used for Common Diseases? Two Cardiologists Propose Five Questions
One-time genetic treatments are moving from rare diseases to common ones like high cholesterol. In JAMA Internal Medicine, two University of Michigan cardiologists explain why permanence, rare side effects and cost call for a new framework.
Oct 224 min read
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GENE TECH TIMES
The Future Has Arrived
FDA Investigates Cancer Risk With Zolgensma After Vector DNA Found Next to a Tumor Gene
The FDA confirmed that Zolgensma's AAV vector inserted next to the GLI1 cancer gene in two children who developed tumors. Here's what was found and what it means for SMA families and AAV gene therapy.
31 minutes ago
FDA Approves Fayuvi, the First Treatment for Sanfilippo Syndrome Type A
The FDA approved Ultragenyx's Fayuvi (UX111), an AAV9 gene therapy and the first treatment of any kind for Sanfilippo syndrome type A, often called childhood Alzheimer's. Here's how it works and what the data showed.
Sep 16
FDA Places New Clinical Hold on Regenxbio's Hunter Syndrome Gene Therapy
The FDA placed a clinical hold on Regenxbio's RGX-121 for Hunter syndrome after MRIs found spinal nodules in 5 of 48 patients, derailing plans to resubmit. Here's the science and the timeline.
Aug 23
Casgevy, the First CRISPR Medicine, Expands to Children as Young as 2
The FDA expanded Vertex's CRISPR therapy Casgevy to children aged 2 and older with sickle cell disease or transfusion-dependent beta-thalassemia. Here's how it works and why the approval for toddlers is notable.
Jul 29
6-Year Update on Gene Therapy for CALD: Positive Results with Continued Caution
A recent 6-year checkup on boys treated with gene therapy for cerebral adrenoleukodystrophy (CALD) reveals lasting benefits for most patients. CALD is a rare, severe brain disease that primarily affects young boys, often leading to a rapid decline in neurological function. In this follow-up, most patients showed stable brain function and minimal disabilities, showcasing the potential of gene therapy to bring hope to families facing this devastating diagnosis. A Closer Look at
Oct 19, 2024
AAV-Based Gene Therapy Clinical Trial Offers Hope for Children with Congenital Deafness
The AK-OTOF-101 clinical trial by Akouos, Inc., a subsidiary of Lilly & Co., brings renewed hope to children suffering from congenital deafness through innovative AAV-based gene therapy. This pioneering research is taking place at The Children's Hospital of Philadelphia, which is leading the charge in developing effective treatments for this challenging condition. The clinical trial utilizes adeno-associated virus (AAV) vectors to deliver the OTOF gene, which encodes the otof
Mar 30, 2024
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