Translating preclinical CRISPR editing in Tay-Sachs and Sandhoff rodent models to human clinical trials

Symposium & Working Group

Thursday June 1, 2023

Time Session
2:00pm – 2:10pm Welcome
Heather Gray-Edwards, DVM, PhD
Co-chair, NTSAD Scientific Advisory Council
2:10pm – 2:50pm Keynote: Advances in the Therapeutic Application of CRISPR/Cas9 for In Vivo Genome Editing
Sean Burns, MD
Vice President, Disease Biology, Intellia Therapeutics
2:50pm – 3:20pm Preclinical programs with an eye to “compassionate use”. Experiences from N=1 INDs.
Lauren Black, PhD
Distinguished Scientist, Charles River
3:20pm – 3:30pm Break
3:30pm – 4:00pm In Vivo Base Editing of the Human Late-Onset Tay Sachs Disease Mutation Delays Symptom Onset and Substantially Prolongs Life-Span
Rick Proia, PhD
Senior Investigator, Genetics and Biochemistry Branch, NIDDK
4:00pm – 4:30pm Rewriting the Code: Pioneering Individualized Gene Editing Techniques for Tay-Sachs Disease Treatment
Zhenya Ivakine, PhD
Scientist, Program in Genetics and Genome Biology, The Hospital for Sick Children, Toronto
4:40pm – 5:00pm Discussion: How do we move gene editing for Tay-Sachs and Sandhoff into the clinic?