Eli Lilly has agreed to acquire eye disease specialist Adverum Biotechnologies, bucking a recent trend of big pharma ...

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

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

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

Gene therapy delivering protective vascular endothelial growth factor C into kidney filters reduced early diabetic kidney ...

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

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

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

Taysha Gene Therapies Inc. (NASDAQ:TSHA) is a clinical-stage biotechnology company that develops and commercializes ...

A one-time subretinal injection of Sura-vec at both high- and low-dose concentrations allowed more than half of patients with ...

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

However, in late September 2025, scientists successfully treated Huntington’s for the first time in a ground-breaking study.