Gene therapies to treat rheumatoid arthritis have evolved slowly, but one is ready to move into human trials and an ...

Researchers have demonstrated, for the first time in the world using mice, the ability to overcome significant challenges in ...

Oxford, UK - 5 February 2025: OXB (LSE: OXB), a quality and innovation-led cell and gene therapy CDMO, today announces the ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

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.

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

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.

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