CRISPR Cures 2033: Expanding the Public Health Impact of Gene Editing
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In this webinar, one of the pioneers of human genome editing—Dr. Fyodor Urnov—laid 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.
Dr. Urnov discussed the unique nature of CRISPR-Cas as a platform technology that brings the promise of leveraging nonclinical and manufacturing information from one disease indication to another, potentially without repeating redundant, costly, and time-consuming nonclinical studies. The non-viral delivery of CRISPR-Cas, including as a Cas9 ribonucleoprotein (RNP), is a strong case study, where key aspects of the manufacturing framework could be leveraged between disease indications. Dr. Urnov assessed the latest progress in clinical genome editing and discussed how creative academia-industry partnerships could enable further progress to develop and reduce real-world manufacturing innovation in the "CRISPR-Cas as a therapeutic platform" space.
This presentation is closely aligned with Dr. Urnov's December 2022 opinion piece in the New York Times, and with the recently released paper “Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs." published in Molecular Therapy, the official journal of the American Society of Gene and Cell Therapy. Aldevron leaders Tom Foti, VP/GM of our Protein Business Unit, and Tim Morris, Manager of Upstream Processing, are listed as contributors to the paper. Both Tom and Tim will help moderate audience questions with Dr. Urnov after his presentation.