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Publications & Presentations

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Program

BEST1

LCA5

MERTK

NMNAT1

RDH12

RHO

Conference

ARVO

Proceedings of the NASUSA

Proceedings of the NAS

Retinal Degeneration Conference

AOS

CTS

APAO

Macula Society

AEMB

AJOCR

Molecular Therapy

Human Gene Therapy

Cold Spring Harbor Perspectives in Medicine

British Journal of Ophthalmology

Investigative Ophthalmology and Visual Science

Drug Design, Development and Therapy

Nature Genetics

American Journal of Pathology

Molecular Therapy: Methods and Clinical Development

Human Molecular Genetics

AARVO

ASGCT

Retinal Imaging Biomarkers & Endpoints Summit

RD International Symposium

Foundation Fighting Blindness Retinal Therapeutics Innovation Summit

RHO
ARVO
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
Lead Author: Dufour.
LCA5
ARVO
May 2026
Restoration of Cone-Mediated Vision After Gene Augmentation in Children with LCA5
Lead Author: Aleman.
BEST1
ARVO
May 2026
Development of Cell-Based Expression and Functional Potency Assays for OPGx-BEST1 Gene Therapy
Lead Author: Siriwardena.
BEST1
ARVO
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
Lead Author: Pennesi.
RHO
ARVO
April 2026
Therapeutic Efficacy of a Mutation-Independent AAV Knockdown and Replacement Approach in a Swine Animal Model of Autosomal-Dominant Retinitis Pigmentosa (adRP)
Lead Author: Jaligampala.
RHO
Foundation Fighting Blindness Retinal Therapeutics Innovation Summit
April 2026
Safety and Efficacy of OPGx-RHO Silence-and-Replace Gene Therapy for RHO-adRP: Evidence Across Two Large Animal Models
Lead Author: Jayagopal.
RHO
ASGCT
April 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
Lead Author: Choudhary.
BEST1
Macula Society
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
Lead Author: Pennesi.
BEST1
APAO
February 2026
Gene Therapy for BEST1 Inherited Retinal Disease
Lead Author: Jayagopal.
LCA5
RD International Symposium
August 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
Lead Author: Jayagopal.
BEST1
CTS
June 2025
Current and Emerging Endpoints for IRDs
Lead Author: Jayagopal.
LCA5
Molecular Therapy
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
Lead Author: Aleman.

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