BEST1
May 2026 :
Therapeutic Platform for BEST1-Associated Blindness: Anticodon-Engineered tRNA and Gene Augmentation
Kabra, et al., ASGCT » View Details
May 2026 :
Development of Cell-Based Expression and Functional Potency Assays for OPGx-BEST1 Gene Therapy
Siriwardena, et al., ARVO » View Details
May 2026 :
Preliminary Results from Adult Participant in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for ARB and BVMD Due to BEST1 Mutations
Pennesi, et al., ARVO » View Details
February 2026 :
Preliminary Results from an Adult Participant in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for the Treatment of BVMD and ARB Due to BEST1Mutations
Pennesi, Macula Society » View Details
May 2025 :
Consideration of Endpoints for the First Interventional Gene Therapy Clinical Study in Inherited Retinal Diseases Caused by BEST1 Mutations
Tucker, et al., AOS » View Details
May 2024 :
Development of OPGx-BEST1 AAV gene therapy for the treatment of bestrophinopathies
Choudhary, et al., ARVO » View Details
September 2023 :
Development Of Opgx-BEST1 AAV Gene Therapy for the Treatment of Bestrophinopathies
Choudhary, et al., Retinal Degeneration Conference » View Details
September 2018 :
Mutation-Independent Rhodopsin Gene Therapy by Knockdown and Replacement with a Single AAV Vector
Cideciyan, Artur V., et al., Proceedings of the National Academy of Sciences » View Details
LCA5
May 2026 :
Restoration of Cone-Mediated Vision After Gene Augmentation in Children with LCA5
Aleman, et al., ARVO » View Details
September 2025 :
One Year Results from a Phase 1/2 Study of OPGx-LCA5 Subretinal Gene Therapy for the Treatment of LCA5-LCA in Adult Participants
Jayagopal, RD International Symposium » View Details
June 2025 :
Recovery of Cone-Mediated Vision in Lebercilin-Associated Severe Retinal Ciliopathy (LCA5) after Gene Therapy: One Year Results of a Phase Ib/IIa Trial
Aleman, et al., Molecular Therapy » View Details
May 2025 :
Utilizing Virtual Reality Guided Multi-Luminance Orientation and Mobility Testing for Assessment of Visual Function
Jayagopal, Retinal Imaging Biomarkers & Endpoints Summit » View Details
May 2025 :
Recovery of Cone-Mediated Vision in a Severe Ciliopathy after Gene Augmentation: One Year Results of a Phase Ib/IIa Trial for LCA5-LCA
Aleman, ARVO » View Details
January 2022 :
Relative preservation of the extramacular retina in LCA5-associated Leber congenital amaurosis
O’Connor, et al., American Journal of Ophthalmology Case Reports » View Details
May 2020 :
Treatment Potential for LCA5-Associated Leber Congenital Amaurosis
Uyhazi et al., Investigative Ophthalmology and Visual Science 2020 » View Details
March 2018 :
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness
Song et al., Molecular Therapy 2018 » View Details
RDH12
May 2025 :
Evaluation of Ocular Tolerability of OPGx-RDH12 by Subretinal Delivery in Cynomolgus Primates
Jayagopal, et al., ARVO » View Details
May 2021 :
Gene Therapy for Rdh12-Associated Retinal Diseases Helps to Delay Retinal Degeneration and Vision Loss
Bian, J., et al., Drug Design, Development and Therapy » View Details
December 2019 :
Natural History and Genotype-Phenotype Correlations in RDH12-Associated Retinal Degeneration
Fahim, A. T., et al., Advances in Experimental Medicine and Biology » View Details
November 2019 :
Detailed Clinical Characterisation, Unique Features, and Natural History of Autosomal Recessive RDH12-Associated Retinal Degeneration
Fahim, A. T., et al., British Journal of Ophthalmology » View Details
November 2019 :
Development of a Gene Therapy Vector for RDH12-Associated Retinal Dystrophy
Feathers, K. L., et al., Human Gene Therapy » View Details
October 2018 :
RDH12 Mutations Cause a Severe Retinal Degeneration With Relatively Spared Rod Function
Aleman et al., Investigative Ophthalmology and Visual Science 2018 » View Details
NMNAT1
November 2021 :
Reduced nuclear NAD+ drives DNA damage and subsequent immune activation in the retina
Brown et al., Human Molecular Genetics 2021 » View Details
May 2021 :
Mutant Nmnat1 leads to a retina-specific decrease of NAD+ accompanied by increased poly(ADP-ribose) in a mouse model of NMNAT1-associated retinal degeneration
Greenwald et al., Human Molecular Genetics 2021 » View Details
July 2020 :
Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration
Greenwald et al., Molecular Therapy: Methods and Clinical Development, 2020 » View Details
July 2016 :
Mouse Models of NMNAT1-Leber Congenital Amaurosis (LCA9) Recapitulate Key Features of the Human Disease
Greenwald et al., American Journal of Pathology 2016 » View Details
September 2012 :
NMNAT1 mutations cause Leber congenital amaurosis.
Falk et al., Nature Genetics 2012 » View Details
RHO
May 2026 :
Nonclinical efficacy and toxicity study of GMP-grade vector OPGx-RHO (scAAV2/5-RHO820-shRNA820) delivered by subretinal injection in a canine model of RHO-adRP
Choudhary, et al., ASGCT » View Details
May 2026 :
Safety and Efficacy of OPGx-RHO Silence-and-Replace Gene Therapy for RHO-adRP: Evidence Across Two Large Animal Models
Jayagopal, et al., Foundation Fighting Blindness Retinal Therapeutics Innovation Summit 2026 » View Details
May 2026 :
Nonclinical Efficacy and Toxicity Study of GMP-Grade Vector OPGx-RHO (Scaav2/5-RHO820-Shrna820) Delivered by Subretinal Injection in a Canine Model of RHO-adRP
Dufour, et al., ARVO » View Details
May 2026 :
Therapeutic Efficacy of a Mutation-Independent AAV Knockdown and Replacement Approach in a Swine Animal Model of Autosomal-Dominant Retinitis Pigmentosa (adRP)
Jaligampala, et al., ARVO » View Details
July 2014 :
Gene Augmentation for AdRP Mutations in RHO
Lewin, Alfred S., et al., Cold Spring Harbor Perspectives in Medicine >> » View Details
January 2012 :
Long-Term Rescue of Retinal Structure and Function by Rhodopsin RNA Replacement with a Single Adeno-Associated Viral Vector in P23H RHO Transgenic Mice
Mao, Haoyu, et al., Human Gene Therapy » View Details
MERTK
May 2025 :
Evaluation of MERTK Gene Therapy in RCS Rats Following a Single Bilateral Subretinal Injection
Choudhary, et al., ASGCT » View Details
May 2025 :
Evaluation of MERTK Gene Therapy in RCS Rats Following a Single Bilateral Subretinal Injection
Choudhary, et al., ARVO » View Details
