UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...
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 ...
After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance ...
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.
Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...
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 ...
Zacks Investment Research on MSN
SRPT stock up as enrollment begins in DMD gene therapy safety study
Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in ...
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 ...
Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
NEW YORK and SAN SEBASTIÁN, Spain, March 17, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results