The U.S. Food and Drug Administration has placed a clinical hold on Intellia Therapeutics' two late-stage trials testing an ...

A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their high cost limits ...

BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending ...

Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ...

Japan's Ministry of Health, Labor and Welfare (MHLW) has granted orphan regenerative medicine product designation to Kyowa ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

Oncologists' experience with CGT has increased, with more patients treated and earlier lines of therapy being utilized.

UniQure's therapy, called AMT-130, reduced disease progression by 75% at 36 months in patients who received a high dose.

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

To solve this critical challenge, he founded Nephrogen in 2022, a biotech startup that uses AI and advanced screening to ...

Tom Golisano donates a record $50 million to University of Maryland Medical System for children's hospital enhancements.