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

A 5-year-old boy from Center City has faced rare odds since birth and now this warrior is in a real life race to rewrite his story.

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

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

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

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

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

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 ...

STEMCELL Technologies, Canada’s largest biotechnology company, is pleased to announce the acquisition of Cellular Highways ...

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

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 ...