After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice.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

Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene ...

The top vaccine and biologics drug official in the U.S. Food and Drug Administration will step down in April. The agency made ...

The Progeria Research Foundation ("PRF"), a nonprofit research organization dedicated to developing treatments and the cure for Hutchinson-Gilford Progeria Syndrome ("Progeria"), and Forge Biologics, ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

Shares of uniQure BV (NASDAQ:QURE) surged 36% on Monday after reports emerged that the head of the U.S. Food and Drug ...

UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question ...

Cure Rare Disease (CRD), a non-profit biotechnology organization, today announced a landmark partnership with the LGMD2L Foundation. This collaboration is backed by a generous commitment of $7.65 ...

Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But ...

After a heart attack, the heart struggles to recoup and maintain energy. One-third of patients develop heart failure as a ...