Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular dystrophy.

Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...

Currently, in the 2025-2027 "Fast, Forward" phase, ClearPoint aims to grow into a $500 million market opportunity, expand to 150 activated customers, and support the first commercial cell and gene ...

Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would advance one day.

Last year, the FDA approved a gene therapy that can be administered directly to the brain. Developed by PTC Therapeutics, Kebilidi is an AAV gene therapy indicated for the treatment of adult and ...

PTC Therapeutics’ Kebilidi™ (US) / Upstaza™ (ex-US) (eladocagene exuparvovec), an AAV vector-based gene therapy indicated for adults and children with aromatic12 L-amino acid decarboxylase ...

The company has license and collaboration agreement with Novartis Pharmaceuticals Corporation to develop PTC518 Huntington's disease program. It markets Upstaza, a gene therapy with the brand name ...

In addition to the recent approval of KEBILIDI for use in the US with ClearPoint cannulas, PTC Therapeutics recently licensed its Huntington's disease therapy (PTC-518).

Gene therapy using a recombinant adeno-associated viral vector carrying the AIPL1 gene improved visual acuity and preserved retinal structure in young children with AIPL1-associated retinal dystrophy.