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LATEST NEWS
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
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 83 min read
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 244 min read
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GENE TECH TIMES
The Future Has Arrived
One Infusion, Lower Cholesterol: Early Data Back Lilly's Bet on Verve
Phase 1 data for VERVE-102, a one-time base-editing treatment that switches off PCSK9, showed LDL cholesterol cuts of up to 62% lasting up to 18 months. Here's what it means for heart disease.
May 25
Eli Lilly Acquires Verve Therapeutics for $1.3 Billion to Advance Gene Editing for Heart Disease
Eli Lilly has announced its $1.3 billion acquisition of Verve Therapeutics, a leading biotech company specializing in gene editing for cardiovascular diseases. This strategic move aims to accelerate the development of one-time gene therapies using base editing technology, targeting high-risk heart disease patients. The deal marks a significant step forward in Eli Lilly’s expansion into precision genetic medicine.
Jul 20, 2025
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