Aldevron News: Gene Editing

Advancing Direct Enzyme Delivery for Therapeutic Genome Editing In Vivo

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.  

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In the News: Pharma's Almanac - The Pivotal Role of Plasmid DNA

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.

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In the news: Pharma's Almanac - CRISPR RNPs and the Future of Cell and Gene Therapy

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.

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Webinar Highlights Potential of Researcher-CMO Collaboration in Gene Editing Therapy

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.

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Aldevron Releases GMP-Grade SpyFi™ Cas9 Nuclease

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.

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Aldevron to Provide New GMP Cas9 Nuclease Through Agreement with Integrated DNA Technologies

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 SpyFiTM Cas9 Nuclease—which is patented by IDT. The product will be manufactured for research, clinical, and commercial use.  

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Aldevron Announces Availability of GMP SpCas9

Aldevron, a leading global provider of contract plasmid DNA manufacturing, protein production and antibody discovery services, announces the availability of GMP-grade SpCas9 nuclease for use in clinical development programs.  Aldevron’s GMP SpCas9 is designed to accelerate gene editing clinical trials and commercial applications by providing an immediate enzyme supply; thereby eliminating months typically associated with contract GMP production and testing.

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