Fractyl Health is developing a shot that would program the body to make more of the GLP-1 hormone naturally, a risky bet that it can provide a longer-lasting benefit than blockbuster weight-loss drugs ...

Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...

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 ...

For the first time, the tools to rewrite human biology are moving from speculative fiction into regulated clinics and ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

Researchers in the United Kingdom say they have successfully trialed what could become the world's first gene therapy for Huntington's disease – a fatal neurodegenerative disorder that is typically ...

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 ...

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 ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...

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.