Regeneron is adding its name to Tessera Therapeutics’ unique in vivo gene writing program, inking a deal that lets it in on a ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease.

This year has seen the approval of several first-in-class therapies for HAE, but in a fragmented space, experts question ...

Initial safety results for the full cohort of 19 indicated that the treatment was well tolerated. The only notable adverse ...

The FDA approved an intrathecal form of Novartis’ spinal muscular atrophy gene therapy Zolgensma on Monday, broadening access ...

Oliver Chu was born with a rare genetic condition called Hunter syndrome that impacts physical and mental development. The ...

Novartis’ Itvisma wins FDA approval as the first gene replacement therapy for spinal muscular atrophy (SMA) patients age 2 ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

Three-year-old Oliver becomes first Hunter syndrome patient to receive gene therapy, showing remarkable progress a year after treatment.

Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...

Assistant Professor of Pharmaceutical Sciences KC Mei standing in his lab at the School of Pharmacy at Binghamton University, State University of New York. BINGHAMTON, N.Y. -- A pharmacy professor at ...

A patient who received an experimental gene therapy developed by Rocket Pharmaceuticals has died following treatment, the company disclosed Tuesday. According to Rocket, the patient experienced what’s ...