Integrated DNA Technologies and Aldevron® Activate Strategic Partnership to Deliver Key CRISPR Components to Cell and Gene Therapy Developers
Aldevron, Jennifer Doudna, and Innovative Genomics Institute Collaborate on In Vivo CRISPR Therapies for Neurologic Diseases. Resulting paper published in Molecular Therapy.
FARGO, N.D. (August 29, 2023)— Aldevron, a leading global manufacturer of DNA, RNA and proteins used in cell and gene therapies and vaccine development, contributed to a publication in collaboration with 2020 Nobel Laureate Jennifer Doudna and the Innovative Genomics Institute (IGI) at the University of California, Berkeley to advance research on in vivo, CRISPR-based genome editing strategies to address genetic diseases of the central nervous system such as Huntington’s disease and ALS. Aldevron supplied custom CRISPR protein with ultra-low endotoxin, enabling direct delivery to the brain in mice. This partnership resulted in a paper, titled “Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs,” published in the journal Molecular Therapy, the official journal of the American Society of Gene and Cell Therapy.
Developing new CRISPR/Cas9-based genome editing tools will enhance innovative therapeutic products and the pathway to clinical programs. However, current obstacles in genome editing include high error rates, toxicity from production impurities, and strict regulatory concerns.