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

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

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

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

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 ...

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

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

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

After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...

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 ...