Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

A groundbreaking gene therapy tested through a UCLA-led clinical trial has received approval from the U.S. Food and Drug ...

New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options. After research conducted as ...

With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients.

What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...

UF scientist develops safer gene therapy restoring vision in children with rare inherited eye diseases; pivotal trials begin this year.

A phase 1/2 trial of a novel viral vector gene therapy in adults with type 1 diabetes is scheduled to begin this year.

Gene therapy for sickle cell disease shows strong clinician support but faces barriers in referral practices, knowledge, and ...

Hemophilia usually is an inherited bleeding disorder in which the blood does not clot properly. Patients with severe hemophilia A may have to manage their condition with regular at-home injections or ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.