A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated ...

Osaka, Japan - A research team at The University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus ...

A total of 15 vector-related adverse events occurred ... These data support the long-term efficacy and safety of AAV gene therapy for severe hemophilia B. (Funded by the U.K. Medical Research ...

Ocugen has a valuable IP portfolio, which should give it funding options. OCGN’s cash runway remains the main risk. Check out ...

Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...

RNA gene scissors (CRISPR-Cas13) are gaining significant attention as a next-generation gene therapy with fewer side effects.

It was found that those with severe hemophilia B have less than 1% of normal factor IX activity, often experiencing spontaneous bleeding that can cause joint damage or life-threatening events.

A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...

OXB (LSE: OXB) (“the Company”), a global quality and innovation-led cell and gene therapy CDMO, provides an update to ...

Siegfried Priglinger, MD discussed research he presented focused on pediatric patients with inherited retinal diseases with ...

The company’s intein-based technology is initially aimed at Stargardt disease, a type of macular degeneration.