CRF awards four global grants to advance CHM research toward new clinical trial endpoints and therapeutic approaches—driving progress toward treatments.
SPRINGFIELD, MA, UNITED STATES, March 9, 2026 /EINPresswire.com/ — The Choroideremia Research Foundation (CRF) is pleased to announce its latest round of scientific research funding, awarding four new grants to advance understanding and treatment options for choroideremia (CHM), a rare inherited retinal disease. These projects reflect the Foundation’s commitment to investing in diverse, high-impact research around the world.
Award recipients are as follows:
Alice Yang Zhang, MD
Department of Ophthalmology, University of North Carolina at Chapel Hill
STUDY: Cross-Sectional Validation of Patient-Reported Outcome Measures and Correlation with Visual Function and Structural Outcomes in Choroideremia
AIM: This study aims to characterize the consistency and reliability of CHM patient-reported outcomes (PROs) in various domains of the Michigan Retinal Degeneration Questionnaire (MRDQ) and the Michigan Vision-related Anxiety Questionnaire (MVAQ). Further, it will be determined if these measures correlate with clinical outcomes such as visual acuity, visual field, fundus autofluorescence, and OCT imaging. By establishing the validity of PROs as potential patient-centered surrogate measures of visual function tests, this work can provide additional endpoints for use in future clinical studies and trials.
Randy Wheelock Award: $50,000
David Gamm, MD, PhD
Director, McPherson Eye Research Institute
Professor, Department of Ophthalmology and Visual Sciences
University of Wisconsin-Madison
Chris Ahern, PhD
Professor, Molecular Biology and Biophysics
University of Iowa
STUDY: Advancing precision ace-tRNA readthrough technology for the treatment of choroideremia
AIM: Engineered transfer RNA (ace-tRNA) technology is a novel and highly effective strategy to overcome the wide array of nonsense mutations affecting one-third CHM patients. The proposal will determine the efficacy, accuracy, and stability of next-generation ace-tRNA molecules to restore substantial
production of REP1 in human induced pluripotent stem cell (hiPSC)-derived retinal pigmented epithelial cells and retinal organoids. These pre-clinical studies are designed to support a future investigational new drug application (IND) that will employ ace-tRNA technology for the treatment of CHM.
John Oster Leadership Award: $125,000
Rob W.J. Collin, PhD
Professor, Department of Human Genetics, Radboud University Medical Center, Nijmegen, The Netherlands
STUDY: Splicing modulation therapy for choroideremia
AIM: This study aims to develop an antisense oligonucleotide (ASO)-based splicing modulation therapy tailored to the c.1359C>T variant of CHM. A successful outcome will deliver a potent (safe and efficacious) ASO that can be further developed (in vivo safety, GMP manufacturing) in preparation of therapeutic intervention. The long-term aim is to determine if ASOs can be developed as personalized treatments to stop further progression of choroideremia.
GRANT: €150,000
Maureen McCall, PhD
Professor, Department of Ophthalmology and Visual Sciences
University of Louisville
STUDY: Generation and characterization of a novel porcine model of Choroideremia
AIM: A clinically relevant minipig model will aid in the development of validated, reproducible, safe, and effective treatments for human patients. Dr. McCall will collaborate with previous CRF Wheelock Awardee Dr. Bhanu Telugu (U. Missouri) to determine if inactivating the CHM gene in male pigs replicates the pathogenesis of choroideremia. CRF contributed to the purchase of a mobile veterinary OCT scanner to support this important work.
GRANT: $10,000
###
About Choroideremia
Choroideremia (CHM) is a rare inherited form of blindness affecting approximately 1 in 50,000 people. Due to its x-linked inheritance pattern males are most severely affected with females usually experiencing much milder visual impairment. Symptoms begin in early childhood with night blindness and restriction of visual field being the earliest noticeable effects, eventually progressing to complete blindness. An estimated 6,000 people in the United States and 10,000 in the European Union are impacted by Choroideremia. There are currently no approved treatments for Choroideremia.
About the Choroideremia Research Foundation Inc.
The Choroideremia Research Foundation (CRF) was founded in 2000 as an international fundraising and patient advocacy organization to stimulate research on CHM. Since its inception, the CRF has provided approximately $6 million in research awards and is the largest financial supporter of CHM research worldwide. Research funded by the CRF has led to the development of a CHM animal model, the pre-clinical production of gene therapy vectors currently in clinical trials, and the CRF Biobank which stores tissue and stem cell samples donated by CHM patients. For more information, visit curechm.org
Reagan Devinney
Choroideremia Research Foundation Inc
+1 800-210-0233
email us here
Legal Disclaimer:
EIN Presswire provides this news content “as is” without warranty of any kind. We do not accept any responsibility or liability
for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this
article. If you have any complaints or copyright issues related to this article, kindly contact the author above.
