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 ...

First demonstration of single AAV muscle editing in non-human primates: NanoCas achieved in vivo editing efficiencies of up to 30% when targeting dystrophin in the skeletal muscle of cynomolgus ...

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

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.

Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular ...

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

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

ReiThera is proud to announce a new partnership with SillaJen through a comprehensive Master Service Agreement (MSA), which establishes ReiThera as the manufacturing partner for SillaJen’s innovative ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

The partners will adapt Eukarÿs' C3P3 engineered enzyme tech to work with adeno-associated virus vectors to boost biomanufacturing yields.