The FDA has released the rejection letter explaining its recent refusal of Regenxbio’s gene therapy for the rare disease Hunter syndrome, providing further details into the agency’s issues with the ...

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

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

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 ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

A new approach to gene therapy for retinitis pigmentosa may help patients with advanced disease regain vision regardless of which genetic mutation is causing the condition. An optogenetic therapy ...