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

Clinical-stage biotechnology company Jaguar Gene Therapy has completed dosing in Cohort I of its first-in-human clinical ...

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

Still, broad uptake has long been an uphill battle for the gene therapy as it competes with a variety of treatments including, Roche’s blockbuster Hemlibra, Sanofi’s newer sales driver Altuviiio and ...

Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or ...

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

Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) is one of the top gene therapy stocks to buy according to hedge funds.

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

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

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.

Nemours Children’s Hospital is now a qualified treatment center to offer a transformative gene therapy to treat sickle cell ...