Opus Genetics' Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by Opus Genetics (IRD).
Over the past two years, Opus Genetics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
OPGx-BEST1. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
OPGx-BEST1 FDA Regulatory Events
OPGx-BEST1 is a drug developed by Opus Genetics for the following indication: For the treatment of bestrophin-1 (BEST1)-related IRD.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- OPGx-BEST1
- Announced Date:
- August 18, 2025
- Indication:
- For the treatment of bestrophin-1 (BEST1)-related IRD.
Announcement
Opus Genetics announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD.
AI Summary
Opus Genetics IRD, a clinical-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration has accepted its Investigational New Drug (IND) application for OPGx-BEST1. This gene therapy is designed to treat bestrophin-1 (BEST1)-related inherited retinal disease, also known as Best disease or vitelliform macular dystrophy. Mutations in the BEST1 gene cause progressive vision loss and can lead to blindness.
With IND clearance, Opus Genetics plans to start a Phase 1/2 trial in the second half of 2025. The multi-center, open-label study will assess the safety, tolerability, and early signs of effectiveness after a single subretinal injection of OPGx-BEST1. Researchers will measure changes in visual function and retinal structure to explore the therapy’s biological activity.
OPGx-BEST1 uses Opus Genetics’ proprietary AAV-based platform to deliver a working copy of the BEST1 gene directly into retinal pigment epithelium cells. Preclinical studies showed restored BEST1 protein expression and improved retinal function in disease models, supporting the move into human trials.
Read Announcement
Opus Genetics FDA Events - Frequently Asked Questions
As of now, Opus Genetics (IRD) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Opus Genetics (IRD) has reported FDA regulatory activity for OPGx-BEST1.
The most recent FDA-related event for Opus Genetics occurred on August 18, 2025, involving OPGx-BEST1. The update was categorized as "FDA Accepted," with the company reporting: "Opus Genetics announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related IRD."
Currently, Opus Genetics has one therapy (OPGx-BEST1) targeting the following condition: For the treatment of bestrophin-1 (BEST1)-related IRD..
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:IRD) was last updated on 8/22/2025 by MarketBeat.com Staff
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