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

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

By reprogramming brain cells, a new gene therapy approach for Alzheimer's developed by UC San Diego researchers could address ...

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

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

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

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

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

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

FDA places clinical hold of RP-A501 for the treatment of patients with Danon disease due to a patient death. Click here to ...

New gene therapy improves the life of 18-year-old with Sickle Cell Disease which affects 5,000 New Jersey residents living ...

Unlike existing treatments for Alzheimer’s that target unhealthy protein deposits, the new approach developed at UC San Diego ...