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 and LYNX-3. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
OPGx-BEST1 FDA Regulatory Timeline and 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:
- May 7, 2026
- Indication:
- For the treatment of bestrophin-1 (BEST1)-related IRD.
Announcement
Opus Genetics, Inc. announced the completion of enrollment in Cohort 1 of its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy.
AI Summary
Opus Genetics announced completion of enrollment in Cohort 1 of its Phase 1/2 OPGx‑BEST1 gene therapy study. Target enrollment included participants with both dominant and recessive forms of BEST disease. The adaptive, open‑label trial tests single‑eye subretinal delivery of OPGx‑BEST1 at up to two dose levels in adults. Five participants were enrolled — three with Best Vitelliform Macular Dystrophy (BVMD) and two with Autosomal‑Recessive Bestrophinopathy (ARB). Four have been dosed; the fifth is scheduled this month.
Three‑month topline results from Cohort 1 are expected in September 2026, with presentation planned at a medical conference. Outcomes will include OCT, microperimetry, best‑corrected and low‑luminance visual acuity, and contrast sensitivity. OPGx‑BEST1 uses Opus’s AAV gene platform to deliver a functional BEST1 gene to RPE cells, aiming to restore BEST1 protein, preserve photoreceptors and improve retinal function. BEST1‑related inherited retinal diseases affect about 22,000 people worldwide and have no approved treatments.
Read Announcement- Drug:
- OPGx-BEST1
- Announced Date:
- February 27, 2026
- Indication:
- For the treatment of bestrophin-1 (BEST1)-related IRD.
Announcement
Opus Genetics, Inc. announced today new clinical data from its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy, presented at the 49th Annual Meeting of the Macula Society, in San Diego, California.
AI Summary
Opus Genetics announced new clinical data from its ongoing Phase 1/2 study of OPGx-BEST1 gene therapy, presented at the 49th Annual Meeting of the Macula Society. The presentation reported 3-month results from the first adult (sentinel) participant, showing the subretinal dose was well tolerated and demonstrated early signs of biological activity. Company leadership said these initial findings are encouraging and represent an important early milestone for patients with BEST1-related retinal disease.
OPGx-BEST1 uses an AAV-based approach to deliver a working copy of the BEST1 gene to retinal pigment epithelium (RPE) cells, aiming to restore BEST1 protein function and help preserve photoreceptors. The adaptive, open-label trial tests single-eye subretinal injection at up to two dose levels in adults with BVMD or ARB. Recruitment is ongoing at two U.S. sites, with more planned; two participants are enrolled so far and 3-month Cohort 1 results are expected mid-2026. The full presentation will be available on the Opus Genetics website.
Read Announcement- Drug:
- OPGx-BEST1
- Announced Date:
- December 9, 2025
- Indication:
- For the treatment of bestrophin-1 (BEST1)-related IRD.
Announcement
Opus Genetics announced that the Independent Data Monitoring Committee (IDMC) issued a positive recommendation to continue as planned in the Company's Phase 1/2 BEST1 clinical trial (BIRD-1), which is a multi-center, adaptive, open-label, dose-exploring study evaluating OPGx-BEST1 in participants with Best disease.
AI Summary
Opus Genetics announced that the Independent Data Monitoring Committee (IDMC) gave a positive recommendation to continue the company’s Phase 1/2 BEST1 clinical trial (BIRD-1) as planned. The IDMC completed its pre-specified safety review of one-month data from the sentinel participant and recommended advancing enrollment and dosing of additional participants without modification. Opus will proceed to dose the next four participants, and the company said the result supports its confidence in the program.
BIRD-1 is a multi-center, adaptive, open-label, dose-exploring study testing OPGx-BEST1 in people with Best disease (BVMD) or autosomal-recessive bestrophinopathy. Treatment is a single subretinal injection in one eye, with two dosing cohorts. The trial will evaluate safety, tolerability, and early signs of efficacy using functional and anatomical endpoints. OPGx-BEST1 uses an AAV gene-therapy approach to deliver a working BEST1 gene to retinal pigment epithelium cells to try to restore BEST1 protein and improve retinal function.
Read Announcement- Drug:
- OPGx-BEST1
- Announced Date:
- November 13, 2025
- Target Action Date:
- Q1 2026
- Estimated Target Date Range:
- January 1, 2026 - March 31, 2026
- Indication:
- For the treatment of bestrophin-1 (BEST1)-related IRD.
Announcement
Opus Genetics announced that the Initial data expected in Q1 2026
AI Summary
Opus Genetics announced that it has dosed the first participant in its OPGx-BEST1 Phase 1/2 trial for Best disease. OPGx-BEST1 is a one-time gene therapy given by a subretinal injection that aims to deliver a working BEST1 gene to retinal pigment epithelium (RPE) cells. The therapy is designed to restore the protein that helps regulate ion flow in the retina and could slow or prevent vision loss in patients with BEST1-related conditions.
The multi-center, adaptive, open-label BIRD-1 study will test safety, tolerability, and early signs of benefit using functional and structural eye measures. The trial is being run with clinical and surgical teams at leading retina centers. Importantly, Opus said initial data from this study are expected in the first quarter of 2026, offering an early read on safety and preliminary efficacy.
Read Announcement- Drug:
- OPGx-BEST1
- Announced Date:
- November 13, 2025
- Indication:
- For the treatment of bestrophin-1 (BEST1)-related IRD.
Announcement
Opus Genetics, announced that the first participant has been dosed in the Company's OPGx-BEST1 Phase 1/2 clinical trial for Best disease (BEST1).
AI Summary
Opus Genetics announced that the first participant has been dosed in its OPGx-BEST1 Phase 1/2 clinical trial for Best disease (vitelliform macular dystrophy). Best disease is a rare, inherited retinal disorder caused by mutations in the BEST1 gene that harm the retinal pigment epithelium (RPE) and can lead to progressive vision loss. OPGx-BEST1 is an AAV-based gene therapy given as a one-time subretinal injection designed to deliver a functional BEST1 gene to RPE cells and restore their function.
The multi-center, adaptive, open-label BIRD-1 study will evaluate safety, tolerability, and early signs of benefit in people with Best disease or autosomal-recessive bestrophinopathy, using two dosing cohorts and measures of visual function and retinal structure. The trial is led by Dr. Mark Pennesi with surgical teams including Drs. Kenneth Fan and Charles Wykoff. Opus expects initial data from the study in the first quarter of 2026 and calls the dosing milestone an important step toward treatments that could preserve or restore vision for affected families.
Read Announcement- 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
LYNX-3 FDA Regulatory Events
LYNX-3 is a drug developed by Opus Genetics for the following indication: Keratorefractive Patients With Visual Disturbances Under Mesopic, Low-Contrast Conditions.
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:
- LYNX-3
- Announced Date:
- September 4, 2025
- Indication:
- Keratorefractive Patients With Visual Disturbances Under Mesopic, Low-Contrast Conditions
Announcement
Opus Genetics, Inc announced that the first patient has been dosed in LYNX-3, the Company's pivotal Phase 3 clinical trial evaluating Phentolamine Ophthalmic Solution 0.75% in treating significant, chronic night driving impairment in keratorefractive patients with reduced mesopic vision.
AI Summary
Opus Genetics announced dosing of the first patient in LYNX-3, a pivotal Phase 3 clinical trial testing Phentolamine Ophthalmic Solution 0.75% for chronic night driving impairment in patients who have undergone keratorefractive surgery and have reduced mesopic vision.
LYNX-3 is a multicenter, randomized, double-masked, placebo-controlled study enrolling about 200 adults with documented low-light vision issues such as glare, halos, and starbursts after LASIK, PRK, SMILE or RK.
Participants will receive once-daily evening eye drops of Phentolamine 0.75% or placebo for two weeks. The trial’s main goal is to measure the percentage of patients who gain at least three lines of improvement in low-contrast vision under dim light after 15 days.
Secondary measures include patient reports of driving difficulty and night-vision disturbances, as well as overall binocular visual function and safety assessments.
In low-light conditions, Phentolamine works by reducing pupil size to block aberrant peripheral light rays while preserving retinal contrast sensitivity.
Read Announcement
Free IRD Stock Alerts