Genomic Odyssey to Ocular Therapy: A First-in-Class Individualized Treatment for Retinitis Pigmentosa

Emily McCourt, MD
Ponzio Family Endowed Chair for Pediatric
Ophthalmology
Chief of Pediatric Ophthalmology
Associate Professor, Ophthalmology

Timothy W. Yu, MD, PhD
Associate Professor
Pediatrics
Harvard Medical School
Principal Investigator of the Yu Lab

Learning Objectives

At the conclusion of this presentation, participants should be able to:

• Evaluate the utility of Whole Genome Sequencing (WGS) in identifying treatable targets for rare genetic diseases.
• Describe the translational pathway for developing customized antisense oligonucleotide (ASO) therapies.
• Analyze the clinical, safety, and regulatory challenges associated with bespoke genomic medicines.

Dr. Subramanian reports financial relationship as a consultant for ACELYRIN, Alexion Pharma, Amgen, GenSight Biologics, Invex Therapeutics, Kriya Therapeutics, Neurophth Therapeutics and Viridian Therapeutics, an investigator for Amgen, GenSight Biologics, Immunovant, Invex Therapeutics, Neurophth Therapeutics and Viridian Therapeutics, recipient of a research grant from Amgen and a DSMB member for Argenx. Dr. Yu reports a financial relationship as a consultant with Servier Pharmaceuticals, SynaptixBio, and Third Rock Ventures and as a speaker with Biomarin and Lilly. All of the other planners, faculty, or individuals in control of content for this educational activity have no relevant financial relationship(s) to disclose with ineligible companies whose primary business is producing, marketing, selling, reselling, or distributing healthcare products used by or on patients.

The University of Colorado Anschutz School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACME) to provide continuing medical education for physicians.

The University of Colorado Anschutz School of Medicine designates this live activity for a maximum of AMA PRA Category 1.5 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.