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LATEST NEWS
Beacon's Gene Therapy for X-Linked Retinitis Pigmentosa Succeeds in a Pivotal Trial
Laru-zova met its primary goal in the pivotal VISTA trial: 31% of high-dose patients gained three or more lines of dim-light vision, versus none in the control group. It is the first XLRP gene therapy to win a late-stage trial.
8 hours ago4 min read
Four Years In, uniQure's Huntington's Gene Therapy Still Slows Decline as It Heads to the FDA
Four-year data for AMT-130 show high-dose patients losing everyday function 61% more slowly than matched untreated patients, but a key composite score weakened. Here's what the results mean as the FDA reviews uniQure's application.
1 day ago4 min read
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 173 min read
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The Future Has Arrived
Beacon's Gene Therapy for X-Linked Retinitis Pigmentosa Succeeds in a Pivotal Trial
Laru-zova met its primary goal in the pivotal VISTA trial: 31% of high-dose patients gained three or more lines of dim-light vision, versus none in the control group. It is the first XLRP gene therapy to win a late-stage trial.
8 hours ago
Four Years In, uniQure's Huntington's Gene Therapy Still Slows Decline as It Heads to the FDA
Four-year data for AMT-130 show high-dose patients losing everyday function 61% more slowly than matched untreated patients, but a key composite score weakened. Here's what the results mean as the FDA reviews uniQure's application.
1 day 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 17
FDA Grants Priority Review to What Could Be the World's First In Vivo CRISPR Therapy
The FDA accepted Intellia's application for lonvo-z in hereditary angioedema with priority review, setting a March 10, 2027 decision date. It could be the first CRISPR therapy that edits genes inside the body.
Sep 8
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 24
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