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

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

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

The companies will work together by using the codon-optimized BBS1 AAV9 vector to minimize the vision loss caused by the ...

Taysha Gene Therapies offers hope for Rett syndrome with innovative treatment, strong trial results, and promising regulatory ...

After spending years developing its endoscopic device treatment for patients with Type 2 diabetes, Fractyl Health is now ...

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

Natural insecticide developer has won $3 million of US military grants to tackle the problems of mosquito-borne diseases, ...

VectorBuilder's partner, Ucello, announced that its CD19-targeting umbilical cord blood-derived allogeneic CAR-T cell therapy ...

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