Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

Gene therapy experts and advocates warning of the sector’s many challenges found a receptive audience in FDA leadership at a ...

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

The Platform Technology Designation, which predates the current FDA leadership, is designed to streamline the drug ...

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing ...

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

The FDA has designated the viral vector rAAVrh74, which Sarepta Therapeutics, Inc. (NASDAQ:SRPT) uses in its gene therapy ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

A Falmouth teen, Jayden Wilsey, is the first to receive gene therapy to treat sickle cell at Boston Children's Hospital.

During an FDA panel discussion, the US health secretary prioritised easier routes to market for curative therapies.

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...