GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for the agency to ...

New one-dose treatment edits a gene in the liver, disabling the ANGPTL3 protein and significantly lowers cholesterol.

Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...

"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...

OCU410 controlled lesion growth 46% better than placebo in dAMD-related GA.

Nanoscope Therapeutics has received Pioneering Regenerative Medical Product (Sakigake) and Orphan Drug designations for ...

The company has made more than 50 deals for its proprietary technology as it looks forward to a potentially “transformative” ...

Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due to ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of addiction from narcotics treatments, a breakthrough which could provide hope for ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

Scientists from UC Davis Center for Surgical Bioengineering, the MIND Institute and UC Berkeley’s Murthy Lab are developing ...