RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

As cell and gene therapies move into more hospitals and cancer centers, health system leaders are learning that clinical success is only part of the equation. These treatments may offer life-changing ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...

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 ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

The FDA has approved Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening immune disorder. Waskyra is indicated for pediatric patients 6 ...