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.
As clustered regularly interspaced short palindromic repeats (CRISPR) therapies progress to the clinic, the key CRISPR editing components are primarily being delivered by recombinant virus, RNA, or as ribonucleoprotein (RNP), which is the active complex formed by the guide RNA and Cas9 protein. RNPs have several attractive properties as a delivery format, particularly in the manufacturing of ex vivo cellular therapies, the most rapidly growing segment of the CRISPR therapeutics market.
As a Project Scientist for the University of California, Los Angeles, Dr. Mark DeWitt has developed a phase 1 GMP-compatible protocol for correcting diseases that cause gene mutations using a CRISPR-Cas9 modular, non-viral gene editing platform. His specific research focuses on a treatment for sickle cell anemia that is more accessible and less costly than a bone marrow transplant—currently, the only way to address the disease.
Over the past year, DeWitt has partnered with Aldevron and Synthego, industry leaders in CRISPR-Cas9 nucleases and gRNA manufacturing, respectively.
Aldevron appoints presidents of its Antibody and Protein business units, strengthening its position as a global leader in biologics manufacturing
Brian Walters, President of its Antibody Business Unit, and Tom Foti now President of its Protein Business Unit
Aldevron has named Brian Walters, President of its antibody business unit and Tom Foti President of its protein business unit.
Ally Hauser of Aldevron has been promoted to Director of Client Relations for Protein Services. In her position, Hauser will lead new projects and initiatives to expand research grade and cGMP protein services to the benefit of our clients.
Aldevron announced today its new fermentation capacity of 1,000 liters and plans to significantly increase the Company’s manufacturing footprint in Madison, WI. The large-scale fermentation equipment is already installed and will be coming on-line in Q4 2019.
Product is the result of a partnership with Integrated DNA Technologies, Inc., and provides clinical stage clients with a critical raw material
Aldevron is announcing the release of GMP SpyFi Cas9 Nuclease for clinical and commercial applications. SpyFi Cas9 Nuclease, the trade name for Aldevron’s research grade and GMP products, is the direct result of a partnership with Integrated DNA Technologies, Inc. (IDT). The advantages of SpyFi Cas9 Nuclease include reduced off-target effects combined with clinically relevant on-target activity.