Expanding the Public Health Impact of Gene Editing
CRISPR-Cas shows promise in leveraging nonclinical and manufacturing information from one disease indication to another, potentially without repeating redundant, costly, and time-consuming nonclinical studies. That’s the driving message in a webinar that featured a pioneer in human genome editing, Dr. Fyodor Urnov, Scientific Director at the Innovative Genomics Institute (IGI).
In this GEN webinar from October 12, developed with IGI and Aldevron, Dr. Urnov laid out the challenges facing the clinical genome editing community for expanding the public health impact of CRISPR-Cas. Urnov pointed out that attaining scalable clinic-grade manufacture of a therapeutic faces several financial, logistical, and regulatory hurdles. Costs, timelines, and regulatory requirements make treating the vast majority of genetic disease intractable in a practical sense.
As part of the discussion, Urnov pointed to the unique nature of the non-viral delivery of CRISPR-Cas through complexing with ribonucleoproteins (RNPs), where key aspects of the manufacturing framework could be leveraged to address multiple “rare” disease indications.
His assessment of the progress in clinical genome editing showed how further gains would be enabled by creative academia-industry partnerships—partnerships developed to build real-world manufacturing innovation in the "CRISPR-Cas as a therapeutic platform" space.
Aldevron’s Tom Foti, Vice President & General Manager, and Tim Morris, Manager of Upstream Development, both of the company’s Protein Business Unit, joined Urnov after his presentation, expressing their support for those creative partnerships, sparking further discussion during the Q&A session.
Urnov stressed that integration of R&D and partnership spaces must begin now to enable progress in the development area, and how it all must be supported with a robust, yet easily navigated, regulatory structure.
To learn more about Dr. Urnov’s views on CRISPR Cures 2033, along with additional perspective from the manufacturing area from Foti and Morris, you can watch the full webinar on demand.
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- Visit our CRISPR solutions web page
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