Press Releases September 2, 2026 07:00 AM

Opus Genetics to Present OPGx-BEST1 Cohort 1 Clinical Data in Investor Webcast and at Upcoming Leading Ophthalmology Conferences

Opus Genetics to Present Promising Phase 1/2 OPGx-BEST1 Gene Therapy Data in Investor Webcast and Key Ophthalmology Conferences

By Leila Farooq
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OPUS

Opus Genetics announced it will present topline data from Cohort 1 of its Phase 1/2 study of OPGx-BEST1 gene therapy targeting BEST1-related inherited retinal diseases. The data presentation is scheduled for September 9, 2026, followed by full clinical datasets at major ophthalmology conferences. OPGx-BEST1 uses an AAV vector to deliver a functional BEST1 gene to retinal cells to treat diseases like BVMD and ARB. The company is advancing multiple gene therapy programs addressing inherited retinal disorders.

Opus Genetics to Present OPGx-BEST1 Cohort 1 Clinical Data in Investor Webcast and at Upcoming Leading Ophthalmology Conferences
OPUS
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Key Points

  • Opus Genetics to disclose topline Phase 1/2 Cohort 1 data for OPGx-BEST1 gene therapy in an investor webcast on Sept 9, 2026.
  • Full clinical datasets and analyses will be presented later that year at leading ophthalmology conferences including Euretina and Retina in Rio.
  • OPGx-BEST1 targets BEST1 mutations causing inherited retinal diseases such as Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB), addressing a significant unmet medical need in ophthalmology and gene therapy sectors.

Topline data from Phase 1/2 Cohort 1 of OPGx-BEST1 gene therapy program to be presented during investor conference call and webcast on September 9 at 8:00 a.m. ET

Full clinical dataset and additional analyses evaluating key measures of visual function to be presented at upcoming medical meetings

RESEARCH TRIANGLE PARK, N.C., Sept. 02, 2026 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (“Opus” or the “Company”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced plans to present topline clinical data from Cohort 1 of its Phase 1/2 study of OPGx-BEST1 during an investor conference call and webcast on Wednesday, September 9, 2026, at 8:00 a.m. ET.

The Company also plans to present the full clinical dataset and additional analyses from the OPGx-BEST1 program at several upcoming medical conferences.

Investor Conference Call and Webcast

Opus Genetics will host an investor conference call and webcast on Wednesday, September 9, 2026, at 8:00 a.m. ET to present topline data from Cohort 1 of the Phase 1/2 clinical study of OPGx-BEST1.

Date: Wednesday, September 9, 2026
Time: 8:00 a.m. ET
Webcast: Opus Genetics Investor Events

A live webcast and accompanying presentation will be available in the Investors section of the Company’s website. A replay will be available following the event.

Upcoming Conference Presentations

Euretina Innovation Spotlight (EIS)

Date: Wednesday, September 30, 2026

Time: 14:20-14:56 CEST

Presenter: Ben Yerxa

Title: Advancing the Future of IRDs: Opus Genetics Clinical Development Update

Euretina 2026

Date: Sunday, October 4, 2026

Time: 13:15-13:21 CEST 

Presenter: Mark Pennesi, M.D., Ph.D., Professor of Ophthalmology, School of Medicine, Casey Eye Institute, School of Medicine

Title: Initial Results from Cohort 1 of a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for the Treatment of BVMD and ARB Due to BEST1 Mutations

Retina in Rio 

Date: Wednesday, October 14, 2026

Title: Structure-Function Correlation of EZ-RPE Thickness and MAIA Microperimetry in BEST1-Related Retinopathy: Results from the Phase 1/2 BIRD-1 Study of OPGx-BEST1 Gene Therapy

Presenter: George Magrath, M.D, CEO, Opus Genetics

Title: Near Normalization of Cone Sensitivity using Gene Augmentation in Children with LCA5

Presenter: Rodrigo Jorge, M.D., Ph.D., Head of Ophthalmology Department at University of São Paulo

FLORetina Presentation

Title: Phase 1/2 Cohort 1 3M Results of OPGx-BEST1 Gene Therapy in Patients with BVMD and ARB Due to BEST1 Mutations

Presenter: Aaron Nagiel, M.D., Ph.D.

About OPGx-BEST1

OPGx-BEST1 is an investigational gene therapy designed to address the underlying genetic cause of BEST1-related inherited retinal diseases, including BVMD and ARB. OPGx-BEST1 uses an AAV vector to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The ongoing BIRD-1 study is an adaptive, open-label Phase 1/2 clinical trial evaluating the safety and efficacy of single-eye subretinal administration of OPGx-BEST1 in adults with BVMD or ARB.

About Opus Genetics

Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RDH12, MERTK, RHO, CNGB1 and NMNAT1. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com.

Contacts

Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
[email protected]

Media
Kimberly Ha
KKH Advisors
917-291-5744
[email protected]


Risks

  • Early clinical data from Phase 1/2 Cohort 1 may not demonstrate clear clinical efficacy or sufficient safety, slowing development progress and affecting valuation.
  • Gene therapy for rare inherited retinal diseases faces complex regulatory and scientific challenges that could delay approvals or commercialization.
  • Competitive landscape includes multiple gene therapy candidates for IRDs, presenting risks related to market adoption and differentiation.

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