Gene and cell therapy developers are contending with the implications of the evolving regulatory environment while trying to progress programs as quickly as possible to address unmet needs and secure first-mover advantage in competitive indications. Regulators are facilitating efforts to truncate development timelines through a series of designations that can expedite programs while also making efforts to implement standardization.
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.
Aldevron recently announced its upcoming virtual ribbon cutting event, which will take place on Wednesday, February 10 at 12:00 PM CST. Its focus will be the company's investments in key areas, expanding Aldevron's ability to enable clients' breakthroughs in the emerging field of genetic medicine, further impacting patients and achieving the company's mission.
The Joint Session of the State of North Dakota’s 67th Legislative Assembly held its opening sessions earlier this week, and featured Governor Doug Burgum’s 2021 State of the State address. Burgum reflected on the progress and challenges of the past year, calling for bold action and investments in infrastructure and other priorities to position the state for a bright future.
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 Announces Presentation of "Standardized Plasmids for Viral Vector Production" at BIO International Convention
Aldevron is scheduled to present, exhibit and conduct one-on-one meetings at the Biotechnology Innovation Organization’s (BIO) International Convention, June 3 – 6, 2019, in Philadelphia. Aldevron’s James Brown, VP Corporate Development, is speaking on standardized helper plasmids for viral vector production.