gene-therapy

Taysha Gene Therapies has finished dosing 17 patients in the pivotal REVEAL trial of its Rett syndrome gene therapy TSHA-102 and reported longer-term Phase 1/2 data, setting up a planned BLA filing after a six-month interim analysis.

The FDA approved expanded use of Vertex's CRISPR therapy Casgevy for patients as young as 2 with sickle cell disease or transfusion-dependent beta thalassemia, making roughly 5,500 more U.S. children eligible.

A Shanghai-built AAV gene therapy delivering two dopamine-synthesis genes to the putamen was safe in 10 patients, with motor improvements at one year.

Lonvoguran ziclumeran met its primary endpoint with an 87% reduction in monthly attacks, the first Phase 3 win for an in vivo CRISPR therapy.

The FDA's June 2 draft guidance lets genome-editing sponsors leverage prior CMC, nonclinical, and clinical knowledge across products, with a 90-day comment window.

Scribe's ELXR platform silences PCSK9 in the liver without cutting DNA; a single low dose sustained LDL reductions for roughly 18 months in primates.

Eli Lilly acquired Engage Bio and its Tethosome non-viral DNA delivery platform for up to $202 million, aiming to overcome the immune and redosing limits of viral gene therapies.

Fractyl Health received authorization in the Netherlands to run the world's first clinical trial of an AAV-based GLP-1 gene therapy for type 2 diabetes, targeting patients who cannot sustain daily injections.

An AAV9 gene-regulation therapy delivered durable, dose-dependent seizure control and early signs of neurodevelopmental rescue through 52 weeks, just as the pivotal study begins dosing.

Regenxbio's gene therapy RGX-202 met the primary endpoint of its pivotal Phase III AFFINITY DUCHENNE trial, with 93% of participants reaching the microdystrophin expression threshold.

Regeneron's one-time intracochlear gene therapy for OTOF-related deafness cleared the FDA 61 days after BLA filing, the first gene therapy product approved under the Commissioner's National Priority Voucher pilot program.

Intellia Therapeutics will report topline Phase 3 results for lonvoguran ziclumeran in hereditary angioedema on April 27, 2026 — the first Phase 3 readout for any in vivo CRISPR candidate.