3). This end-to-end platform leverages Pfizer’s extensive rare disease drug development expertise to advance gene therapy development timelines, support global gene therapy trials, and engage ...

Gene therapy trailblazer Ark Therapeutics was dealt a blow last December, when the European Medicines Agency (EMEA) rejected the London-based company's marketing application for its adenoviral ...

The company will discontinue development of its original ALS gene therapy candidate VY9323 amid toxic effects in nonhuman ...

OTO, is one of several early-stage gene therapies being developed to treat relatively straight-forward causes of genetic ...

Ocugen, Inc. (NASDAQ: OCGN) announced a significant milestone with the U.S. Food and Drug Administration (FDA), gaining ...

The team notes that different forms of SCN1B gene expression may result in different outcomes for the therapy. However, the proof-of-concept is the first step toward a gene replacement therapy for ...