Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at the MDA (Muscular Dystrophy ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

Developers of gene therapies to treat knee osteoarthritis are taking the familiar intra-articular route to deliver proteins and molecules directly to the site of the disease.

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...

Clinical-stage biotechnology company Jaguar Gene Therapy has completed dosing in Cohort I of its first-in-human clinical trial investigating JAG201 for patients with SHANK3 haploinsufficiency, a ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Neurogene received FDA Breakthrough Therapy Designation for NGN-401 in Rett Syndrome, driving a >25% share price surge. Click ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...