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

An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...

A genetic variant carried by at least one-fifth of the population may do far more than raise the odds of developing Alzheimer ...

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 ...

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 ...

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 ...

The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring.  | The company is working ...

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.

Hemgenix for hemophilia B is temporarily unavailable, says maker CSL Behring ...

After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question ...

The 28th American Society of Gene & Cell Therapy (ASGCT) meeting kicks off in mid-May in New Orleans. The annual event, which is on pace toward a goal of 8,000 attendees, will be a whirlwind of ...