Casgevy sickle cell gene therapy is now FDA-approved for children as young as two, opening access for 5,500 more U.S. kids.

Jay Manuel and Pamela Chavez-Manuel, stars of TLC's "Jay & Pamela," candidly discuss living with OI Type III, ahead of the ...

Daniel Cressy, 23, has become the first person in the Gulf South to become functionally cured of sickle cell disease using ...

Casgevy also now indicated for transfusion-dependent beta thalassemia in children 2 years ...

Recent data published in NEJM demonstrates that CRISPR gene-editing therapy achieves complete clinical efficacy endpoints in ...

July 1 (Reuters) - The U.S. Food and Drug Administration approved expanded use of Vertex Pharmaceuticals' gene therapy in ...

Orca Bio, a commercial-stage biotechnology company committed to transforming the lives of patients through high-precision ...

I’m currently in the final stages of recovering from osteonecrosis of the jaw (ONJ). Getting a diagnosis took two months, all ...

Gene editing has delivered remarkable results against sickle cell disease. Now researchers are working to make these ...

Shannon Dingey was diagnosed with indolent systemic mastocytosis (ISM) at 16. She spent her teen and young adult years in and out of the hospital. But she says her life is "beautiful and purposeful." ...

CRISPR Therapeutics wins FDA pediatric approval for Casgevy, boosting market potential and pipeline upside despite slow uptake. Click here to read more.

Children who survive a stem-cell transplant only to watch their own donor cells attack their organs now have a ...