May 1, 2023

Eric Pierce Receives Proctor Medal for Outstanding Achievements in Retinal Research

Eye On the Cure Research News

During his Proctor Award Lecture, Dr. Pierce reviewed encouraging clinical trial results for Editas’ CRISPR/Cas9 treatment for people with LCA10

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Eric Pierce, MD, PhD, a world-renowned clinician-scientist dedicated to eradicating inherited retinal diseases (IRDs), was honored with the prestigious Proctor Medal on April 24 at the 2023 annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in New Orleans.

The Proctor Medal, which honors outstanding research in ophthalmology, was established in 1949 as a memorial to Dr. Francis I. Proctor, an ophthalmologist who conducted extensive research on the etiology and treatment of trachoma, a contagious bacterial eye infection.

Dr. Pierce’s groundbreaking research efforts, many of which have been funded by the Foundation Fighting Blindness, have focused on understanding the genetic causes of IRDs and developing therapies to save and restore vision for people affected by them. His achievements include the identification of several IRD genes and investigative roles in several clinical trials for emerging therapies, including the study at the Children’s Hospital of Philadelphia that led to the FDA-approved RPE65 gene therapy, LUXTURNA®. He is also recognized for being a mentor to several up-and-coming IRD clinician-scientists and laboratory investigators. Dr. Pierce served as chair of the Foundation’s Scientific Advisory Board from 2005 to 2015.

During his Proctor Award Lecture, Dr. Pierce reviewed results from the BRILLIANCE Phase 1/2 clinical trial launched by Editas for EDIT-101, a CRISPR/Cas9, gene-editing therapy designed to correct, or snip out, a deep-intronic mutation (IVS26) in the gene CEP290, a mutation that leads to Leber congenital amaurosis 10 (LCA10). Though LCA10  causes severe vision loss in children, significant retinal structure remains in patients for a relatively long period of time, making the condition a good candidate for genetic therapies.

In the Phase 1/2 clinical trial, EDIT-101 had a favorable safety profile with no report of serious adverse reactions. The treatment also showed evidence of efficacy with 12 of 14 trial participants demonstrating improvement in at least one of four measures of visual function. Six of 14 participants had improvement in at least two measures. The visual functions evaluated in the trial were: best corrected visual acuity, navigation of a multi-luminance mobility course, and full-field retinal sensitivity. The participants also completed a visual function questionnaire (the National Eye Institute’s VFQ-25).

A BRILLIANCE principal investigator, Dr. Pierce deemed the trial a success thanks to the meaningful improvements in functional vision for most patients. He also noted that EDIT-101 was one of the first CRISPR/Cas9 treatments directly administered to humans. The treatment, contained in a human-engineered virus known as an AAV, was injected underneath the patients’ retinas.

Dr. Pierce is now working with Editas to identify an industry partner to move the therapy forward.

During his lecture, Dr. Pierce also reviewed the impressive progress that’s been made in identifying IRD genes, including the Berman-Gund Lab’s discovery of rhodopsin in 1989, the first gene to be associated with an IRD. With 281 genes now linked to genetic retinal conditions, approximately 60 percent of patients get a definitive diagnosis when genetically tested with a broad panel. Dr. Pierce’s lab is part of a Foundation-funded consortium working to identify the remaining genes, and hard-to-find mutations in known genes, that cause IRDs.

Dr. Pierce is currently the director of the Harvard Ophthalmology Ocular Genomics Institute (OGI); the William F. Chatlos Professor of Ophthalmology at Harvard Medical School; and director of the Berman-Gund Laboratory for the Study of Retinal Degeneration at Mass Eye and Ear, the first research center launched by the Foundation. He received his medical degree from Harvard Medical School and doctorate in biochemistry from University of Wisconsin-Madison.