Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...

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 research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

Groundbreaking advancements in gene therapy at UTMB represent new possibilities for patients living with rare genetic ...

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 ...

AskBio Inc., an RTP-based gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has received U.S. Food and Drug Administration (FDA) acceptance of its ...

Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...

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.