The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

While the company is prioritizing RJVA-001 and a lead program in obesity, it's pausing some R&D efforts and laying off 17 percent of its workforce.

Gene therapy using modified blood stem cells helped five adults with severe hemophilia A reach therapeutic levels of clotting ...

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.

The World Health Organization (WHO) congratulates Niger for having met the criteria for onchocerciasis elimination, making it ...

Financial writer recommends Voyager Therapeutics, Inc. for high-risk investors due to promising preclinical Alzheimer's ...

The Purdy family is working with researchers to develop an entirely new clinical trial they hope will save Paxton and other ...

With permission from a data safety monitoring committee, the firm will begin treating a second cohort with AMT-162 in the EPISOD1 trial in early 2025.

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

Initial interim 52-week results from Phase 2b Population Extension cohort of PRISM clinical trial to be presented by Dante Pieramici, M.D., a ...