Prime Medicine, Inc. (Nasdaq: PRME) has announced a preclinical program for alpha-1 antitrypsin deficiency (AATD), expanding its liver-focused gene editing pipeline. The company anticipates filing an investigational new drug (IND) or clinical trial application (CTA)...
Verve Therapeutics has not escaped the doom that seems to be engulfing the gene editing sector of late. The company said Thursday that Vertex Pharmaceuticals handed back the rights to a preclinical-stage gene editing program for liver disease. The two companies had...
Credit: Unsplash/CC0 Public Domain A collaborative effort between investigators at the National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID) and Massachusetts General Hospital (MGH) demonstrates the potential of precise...
*Nicolás Sandoval-Villegas, Zoltán Ivics, The best of both worlds: AAV-mediated gene transfer empowered by LNP delivery of Sleeping Beauty transposase for durable transgene expression in vivo, Molecular Therapy, Volume 32, Issue 10, 2024, Pages 3211-3214, ISSN...
Revolutionizing CAR T Cell Therapy with Lipid Nanoparticles Chimeric antigen receptor (CAR) T cell therapy has transformed cancer treatment by turning a patient’s own T cells into powerful cancer-fighting agents. However, as the technology advances, there is an...
