Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them ...

Ocugen has a valuable IP portfolio, which should give it funding options. OCGN’s cash runway remains the main risk. Check out ...

Greater numbers of circulating stem cells increase the efficacy of in vivo gene therapy techniques, potentially reducing the price tag of lifesaving treatments.

Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing ...

The startup, which is backed by the venture arms of Sanofi, Roche and Novartis, is using dual adeno-associated viruses to ...

How can early decisions in cell and gene therapy clinical development impact downstream progress toward commercialization and ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.

For example, comprehensive data about cystic ... be among the 10% who don’t benefit from modulator therapies. Can a gene therapy work no matter the mutation? While there’s little chance ...

Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the ...

The infant, named KJ, initially received a low dose of the therapy when ... this may be the first example of a new treatment that can be tweaked to treat a wide range of genetic disorders in ...

A baby born with a rare and dangerous genetic disease is ... The first CRISPR therapy approved by the U.S. Food and Drug Administration, for example, treats sickle cell disease, a painful blood ...