Press Releases April 27, 2026 08:00 AM

Opus Genetics to Participate in Leading Medical Conferences in May 2026

Opus Genetics to Present Progress on Gene Therapy Programs at Key Ophthalmology Conferences in May 2026

By Priya Menon IRD
Opus Genetics to Participate in Leading Medical Conferences in May 2026
IRD

Opus Genetics announced that its leadership team will present encouraging preliminary and preclinical data on its gene therapy programs for inherited retinal diseases at multiple prestigious medical conferences in May 2026. This includes updates on Phase 1b/2a clinical trials for LCA5 and BEST1 gene therapies, highlighting advancements toward durable, one-time treatments aimed at restoring vision and preventing blindness in patients with genetic retinal disorders.

Key Points

  • Opus Genetics will present preliminary data from Phase 1b/2a OPGx-BEST1 clinical trial and preclinical work at conferences like ARVO, ASGCT, and Retina World Congress in May 2026.
  • Focus remains on developing durable, one-time gene therapies targeting inherited retinal diseases such as LCA5, BEST1, and RHO-related disorders.
  • The company also advances a small-molecule therapy, Phentolamine Ophthalmic Solution, progressing through regulatory review and Phase 3 trials for eye conditions beyond mydriasis.
  • The biotechnology sector, especially gene therapy and ophthalmology subsectors, is impacted as this reflects progress toward innovative treatments for rare genetic diseases.

RESEARCH TRIANGLE PARK, N.C., April 27, 2026 (GLOBE NEWSWIRE) -- Opus Genetics (Nasdaq: IRD) (“Opus Genetics” 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 that members of its leadership team will participate in several leading ophthalmology and industry conferences.

“We look forward to sharing progress across our programs at ARVO and other leading meetings this May, including encouraging findings from our LCA5 and BEST1 gene therapy programs,” said Dr. George Magrath, Chief Executive Officer, Opus Genetics. “At ARVO, we will present previously disclosed preliminary data from our ongoing Phase 1b/2a OPGx-BEST1 study, alongside new preclinical and translational work advancing our pipeline. These meetings provide an important opportunity to engage with the ophthalmology community as we work to develop durable, one-time treatments for inherited retinal diseases.”

Retinal Therapeutics Innovation Summit 2026

  • Presentation Title: Safety and Efficacy of OPGx-RHO Silence-and-Replace Gene Therapy for RHO-adRP: Evidence Across Two Large Animal Models
  • Date: May 1, 2026
  • Location: Denver, CO

Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting

  • Presentation Title:
    • Restoration of Cone-Mediated Vision After Gene Augmentation in Children with LCA5
    • Preliminary Results from Adult Participants in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for ARB and BVMD due to BEST1 Mutations
    • Development of Cell-Based Expression and Functional Potency Assays for OPGx-BEST1 Gene Therapy
  • Date: May 3–7, 2026
  • Location: Denver, CO

American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

  • Presentation Title:
    • Therapeutic Platform for BEST-1-associated Blindness: Anticodon-Engineered tRNA and Gene Augmentation
    • 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
  • Date: May 11–15, 2026
  • Location: Boston, MA

Retina World Congress

  • Presentation Title: Retina Unplugged: Inherited and Rare Retinal Diseases
  • Date: May 14–17, 2026
  • Location: Fort Lauderdale, FL

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 RHO, CNGB1, RDH12, NMNAT1, and MERTK. Opus Genetics is also advancing a small-molecule therapy, Phentolamine Ophthalmic Solution 0.75%, beyond its approved use for pharmacologically induced mydriasis, with a supplemental new drug application under review for presbyopia and an ongoing Phase 3 pivotal trial for mesopic, low contrast conditions after keratorefractive surgery (dim light disturbances). 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]

Source: Opus Genetics


Risks

  • Clinical data presented are preliminary and from early-phase trials, so efficacy and safety outcomes remain uncertain and subject to further validation.
  • Regulatory approvals and commercial viability of gene therapies and small-molecule drugs are uncertain and could impact future development.
  • Competition among gene therapy developers in inherited retinal diseases is intense, potentially affecting market positioning and investor sentiment.

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