3RD ANNUAL SYMPOSIUM

AGENDA

  • 9:10am - 10:30am  SESSION 1 | Disease-Agnostic Editing and AI-Powered Patient Intelligence

    Chair/Intro: Anna Greka

                        • 9:10am - 9:30am: David Liu (Prime editing-installed suppressor tRNA)

                        • 9:30am - 9:35am: Q&A

                        • 9:35am - 9:50am: Nasha Fitter (Patient Data as a Therapeutic Discovery Engine)

                        • 9:50am - 9:55am: Q&A

                        • 9:55am - 10:25am: Panel Discussion (Meg Alexander, David Goldstein, Winston Yan)

                        • 10:25am - 10:30am: Q&A

  • 10:45am - 11:30am

    SESSION 2 | Target Discovery and Drug Design

    Chair: Caroline Uhler

                        • 10:45am - 11:00am: Olivia Wiles + Matt Brown (Optical Pooled Screening...)

                        • 11:00am - 11:05am: Q&A

                        • 11:05am - 11:25am: Andrew Adams (From Target Discovery to Clinical Impact)

                        • 11:25am - 11:30am: Q&A

  • 11:30am - 11:55am SESSION 3 | From Metabolic Node to Therapeutic Target

    Chair/Intro: Jillian Shaw

                        • 11:30am - 11:50am: Vamsi Mootha (Hypoxia as a Nodal Therapeutic Strategy)

                        • 11:50am - 11:55am: Q&A

  • 1:00pm - 2:00pm   SESSION IV: Mechanism Informed Therapeutic Development for Genetic Diseases

    Chair: Tim Yu

                        • 1:00pm - 1:15pm: Jay Thiagarjah (Small-molecule Discovery for MVID)

                        • 1:15pm - 1:20pm: Q&A

                        • 1:20pm - 1:35pm: Magdalena Riedl (Scalable Screening Approaches...)

                        • 1:35pm - 1:40pm: Q&A

                        • 1:40pm - 1:55pm: Cristina Jacobsen (Generalizable approaches to Collagenopathies)

                        • 1:55pm - 2:00pm: Q&A

  • 2:00pm - 2:40pm   SESSION V: Next-Generation Platforms for Disease Modeling

    Chair: Stacey Gabriel

                        • 2:00pm - 2:15pm: Sarah Morton (Cellular Barcoding at Scale)

                        • 2:15pm - 2:20pm: Q&A

                        • 2:20pm - 2:35pm: Sarra Merzouk (CellWorks: Cell Line Engineering...)

                        • 2:35pm - 2:40pm: Q&A

  • 3:00pm - 4:20pm   SESSION VI: CNBC Cures Panels

                        • 3:00pm - 3:30pm: Panel I: The Reimbursement Gap (Andrew Lo, William Shrank, George Yancopoulos)

                        • 3:30pm - 3:40pm: Q&A

                        • 3:40pm - 4:10pm: Panel II: Regulation, Advocacy, and Patient Advocates (Scott Gottlieb, Cat Lutz, Terry Pirovolakis)

                        • 4:10pm - 4:20pm: Q&A

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SPEAKERS

Anna Greka, MD, PhD

Broad Institute of MIT and Harvard

Jillian Shaw, PhD

Broad Institute of MIT and Harvard

Wendy Chung, MD, PhD

Boston Children’s Hospital

Join us for an inspiring event to discuss the latest in cutting-edge research, fierce advocacy, and synergistic collaborations within the rare disease community.

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