Aldevron News: CRISPR

WEBINAR: CRISPR Genome Editing Solutions from Discovery to Clinic

Among current techniques for clinical genome editing, the CRISPR-associated gene editing system has emerged as a highly advantageous method to drive development of new gene and cell therapies. Through evolving routes of direct delivery of the CRISPR-ribonucleoprotein (RNP) complex, consisting of single guide RNA (sgRNA) and Cas nucleases, a non-viral approach has become increasingly in demand for gene-editing clinical trials. However, drug developers face significant challenges when initiating their clinical programs, which includes the design and production of the target-specific guides, the production of reliable, high-quality nucleases, and the ability to characterize the RNP complex for the efficient and safe delivery of a genomic therapy.
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WEBINAR: CRISPR Cures 2033: Expanding the Public Health Impact of Gene Editing

In this live webinar, one of the pioneers of human genome editing—Dr. Fyodor Urnov—will lay out the urgent challenge facing the clinical genome editing community in terms of expanding the public health impact of CRISPR-Cas. A formidable financial, logistical, and regulatory hurdle in all genetic therapies is attaining scalable clinic-grade manufacture of the experimental therapeutic. The current costs, timelines, and requirements in that space make the vast majority of genetic disease intractable in a practical sense.  
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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.

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Webinar: Genome Editing Tools Beyond Discovery

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.

Thursday, November 3, 2022

8am PT, 11 am ET, 17:00 CET

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