Gene Therapy

Shanghai Jiao Tong University is investigating neuroscientist Zilong Qiu after a 6-year-old girl died from a severe immune reaction in an experimental gene-editing trial that went undisclosed.

Jennifer Doudna's team used AI protein design to create synthetic TnpB nucleases that edited human genes more efficiently than the natural enzyme.

ARPA-H's new THRIVE program is funding seven teams to build reusable gene-editing platforms for rare pediatric diseases, aiming to move beyond one-patient-at-a-time therapies like the one made for infant KJ Muldoon.

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.

CARsgen's Claudin18.2-directed CAR-T satri-cel won Chinese approval for pretreated gastric cancer, becoming the first CAR-T cleared for any solid tumor.

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The FDA's June 2 draft guidance lets genome-editing sponsors leverage prior CMC, nonclinical, and clinical knowledge across products, with a 90-day comment window.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