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SUMMARY:Duke Genomic Seminar Series
DESCRIPTION:A platform approach to develop and deploy CRISPR-Cas based experimental therapies for inborn errors of immunity in an academic/nonprofit setting \nThursday\, February 8 | 2-3 p.m. ET | Bryan Neurobiology Research Building\, Rm 103 \nSpeaker: Fyodor Urnov\, PhD\nProfessor of Molecular & Cellular Biology\, University of California\, Berkeley\nDirector of Technology & Translation\, Innovative Genomics Institute \nThe vast majority of diseases clinically tractable by genome editing using current-state technology are not the targets of active preclinical or clinical development. Addressing this issue will require more involvement of the academic/nonprofit sector\, but despite an existing charted path for advancing genome editing of specific cell types and tissues (such as T cells\, hematopoietic stem cells\, the liver\, and the retina) to the clinic\, there presently is only one open US IND for genome editing by an all-academic group. A key reason is that the current manufacturing\, regulatory\, and healthcare economics environment is not configured to maximize clinical impact of a technology like CRISPR-Cas. Under the framework that exists today\, a pediatric patient newly diagnosed with a terminal illness such as a severe inborn error of immunity due to a never-before-seen but clinically editable mutation and a life expectancy of < 1 yr will have to wait ~3.5 years until a gene editing medicine is engineered\, tested\, and manufactured at an approximate total cost of $9m. An actionable path through this status quo is to develop and clinically derisk a dedicated nonclinical development path for use in academic/nonprofit settings in N=1/rare situations of dire medical need. From a technology standpoint\, this will require comprehensive leveraging of the intrinsically platform nature of CRISPR-Cas gene editing. The Innovative Genomics Institute in close partnership with clinicians at UCSF and UCLA is developing such a platform focused on inborn errors of immunity\, where ca 112\,000 known patients suffering from 505 currently known diseases lack even a single open trial for genome editing open at the present time. \nTo learn more\, visit Duke’s Genomic Seminar Series page. \nPlease note: This seminar series occurs quarterly with some events occurring virtually only and others in-person with streaming options.
URL:https://gmkb.org/event/duke-genomic-seminar-series/
LOCATION:Duke Neurobiology Research Center\, 311 Research Drive\, Durham\, NC\, 27710\, United States
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