top of page
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
LEARN MORE
GENE TECH TIMES
The Future Has Arrived
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
One CRISPR Dose, 87% Fewer Attacks: Inside Intellia's Phase 3 Win for Hereditary Angioedema
Intellia's one-time CRISPR therapy lonvo-z cut hereditary angioedema attacks by 87% in the Phase 3 HAELO trial. In its August update, the company set a U.S. launch target of early 2027. Here's how it works.
Aug 6
Prime Medicine Wins Arbitration Against Beam, Clearing the Way for Its AATD Prime Editor
An arbitration tribunal confirmed Prime Medicine's rights to PM647, a prime-editing therapy for alpha-1 antitrypsin deficiency, ending a dispute with Beam Therapeutics. Here's the science and what comes next.
Jul 8
FDA Draft Guidance Lets Gene-Editing Developers Build on What's Already Known
A June 2026 FDA draft guidance lets developers of genome-editing therapies reuse prior manufacturing, nonclinical and clinical knowledge. Here's why the platform approach could speed rare-disease cures.
Jun 3
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
bottom of page