Gene therapy represents a paradigm shift in modern medicine, offering unprecedented opportunities to combat diseases at their genetic origin.

The FDA has extended the review for the BLA for clemidsogene lanparvovec for the treatment of MPSII, also known as Hunter Syndrome.

Down syndrome is also the most common genetic cause of cognitive dysfunction. But little research has explored how gene editing tools could potentially be used to treat this condition. A new study ...

Opus Genetics (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and small molecule therapies for other ...

The FDA has postponed its decision date for Regenxbio’s Hunter syndrome gene therapy to review additional longer-term ...

A number of recent clinical trials have suggested that gene therapy could help treat diseases of the eye that cause vision loss. This Special Feature explores the current evidence and outstanding ...

Company expects to complete BLA submission in the fourth quarter of 2025NOVATO, Calif., Aug. 18, 2025 (GLOBE NEWSWIRE) -- ...

A number of recent clinical trials have suggested that gene therapy could help treat diseases of the eye that cause vision loss. This Special Feature explores the current evidence and outstanding ...