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.

Genome editing enzymes such as Cas9 have been successfully employed to target different genetic disorders in model systems, and ex vivo approaches have demonstrated the clinical potential of this technology. However, the challenge of delivering such corrective enzymes in vivo represents a substantial barrier to therapeutic translation.  

Viral vectors—refined during their use in traditional gene therapy—represent the most widely-used delivery platform in preclinical work, and nanoparticles are quickly maturing into a comparably powerful vehicle. Direct delivery of pre-formed CRISPR enzymes has recently emerged as an appealing strategy for enabling therapeutic editing in vivo.  

Plasmid DNA was key to the development of biologic drug manufacturing. Today, it plays a critical role in the production of next-generation cell and gene therapies and vaccines. With its plasmid DNA manufacturing expertise, Aldevron has helped facilitate the advance of these important therapeutics. The company continues to invest in additional capacity and novel capabilities to support biopharma manufacturers into the future.

Aldevron and Integrated DNA Technologies, Inc. (IDT), a supplier of custom nucleic acid and genome editing products, have announced a license agreement whereby Aldevron will manufacture and distribute a S. pyogenes Cas9 variant—known as SpyFi^TM Cas9 Nuclease—which is patented by IDT. The product will be manufactured for research, clinical, and commercial use.