While vepdegestrant outperformed a standard treatment option in a subtype of patients, the benefit on progression-free ...

Intellia’s stock fell 23% after one patient in its Phase III MAGNITUDE trial developed severe liver toxicity that later ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Preliminary data from ongoing Phase 1/2 trials show BEAM-302 is well tolerated and achieves dose-dependent, durable ...

Researchers engineered a new therapeutically enhanced, naturally derived particle for powerful gene therapies to tackle our ...

Cardiovascular disease and kidney disease are two of the most urgent issues in global public health. In the United States ...

While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of ...

Intellia Therapeutics’ stock may have taken a hit in the wake of details of a grade 4 adverse event in a phase 3 trial, but ...

Taysha Gene Therapies, Inc. (NASDAQ: TSHA) has announced pivotal progress in its TSHA-102 gene therapy program for Rett ...

FDA places clinical hold of RP-A501 for the treatment of patients with Danon disease due to a patient death. Click here to ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

A New Jersey biotech company has paused a gene therapy trial after a participant had an unexpected adverse event and later died from a sudden, severe infection.  Rocket Pharmaceuticals said the ...