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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
Gene Therapy Successfully Reverses Heart Failure in Minipig Study: A Big Step in Heart Failure Treatment
Heart failure, which affects over 64 million people worldwide, is currently considered irreversible without a heart transplant. Researchers at the University of Utah have developed a gene therapy that could change this. Using minipigs as a model, scientists successfully reversed heart failure by delivering a therapeutic gene called cBIN1 directly into heart cells. The therapy, which targets a key protein often deficient in heart failure patients, resulted in significant recov
Dec 11, 2024
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