Tuesday, 7 February 2023 to Wednesday, 8 February 2023

CRISPR In Drug Discovery ... When CRISPR IS the Drug

Tue7 Feb04:50pm(40 mins)
Where:
Auditorium
Plenary Keynote:

Abstract

Evidence from multiple ongoing clinical trials proves that genome editing can attain robust disease-modifying outcomes in multiple indications including sickle cell disease, transfusion-dependent beta-thalassemia, and TTR amyloidosis. Current regulatory and manufacturing frameworks, however, are not scaled to expand the clinical footprint of CRISPR to a substantial fraction of Mendelian disease, even in settings where a charted path exists to advance editing to the clinic (such as inborn errors of immunity). Such an expansion will require the engineering and clinical development of a fundamentally new approach to designing, developing, and deploying CRISPR-based genomic medicines. The academic/nonprofit sector is a key venue for this effort, with nonmalignant hematology as an actionable disease space for reducing such an approach to practice in the next 3-5 years.

Hosted By

ELRIG

The European Laboratory Research & Innovation Group Our Vision : To provide outstanding, leading edge knowledge to the life sciences community on an open access basis

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