Opus Genetics, Inc. develops ophthalmic therapies for inherited retinal diseases as a clinical-stage biopharmaceutical company. Its pipeline includes AAV-based gene therapy 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.
Company news commonly covers clinical and preclinical data, FDA regulatory interactions, scientific conference presentations, financing arrangements and annual financial results. Updates also include ophthalmic small-molecule development tied to Phentolamine Ophthalmic Solution 0.75% and collaboration activity related to that asset.
Opus Genetics (Nasdaq: IRD) received FDA clearance to begin clinical development of OPGx-RDH12, its investigational gene therapy for inherited childhood blindness. The therapy targets RDH12-associated Leber congenital amaurosis, a rare retinal disease causing progressive vision loss from early childhood. Opus plans to initiate a Phase 1/2 trial this quarter, advancing its third gene therapy program into clinical development.
The Global RDH12 Alliance committed up to $1.6 million toward development under a collaboration established in July 2025. Eligible costs are reimbursed as activities are completed, subject to diligence obligations and milestones.
Opus Genetics (Nasdaq: IRD) received FDA approval for phentolamine ophthalmic solution 0.75% to treat presbyopia, age-related difficulty focusing on nearby objects, in adults.
The supplemental approval expands the uses of RYZUMVI, already marketed in the United States to treat drug-induced pupil dilation. It will trigger a milestone payment to Opus Genetics. Under a global licensing agreement, Viatris, through an affiliated entity, holds exclusive U.S. commercialization rights.
The approval was supported by the pivotal Phase 3 VEGA-2 and VEGA-3 trials. Both met their primary and all key secondary efficacy endpoints, with no treatment-related serious adverse events reported. In the presbyopia safety information, the most common ocular adverse reactions were eye redness (21%) and irritation at the application site (16%).
Opus Genetics (IRD) presented clinical findings showing improved visual function in participants receiving its OPGx-BEST1 and OPGx-LCA5 gene therapies. In the Phase 1/2 BEST1 low-dose cohort, three of four evaluable participants (75%) showed improved retinal light sensitivity in treated areas surrounding damaged tissue. Five participants were treated, with three to six months of follow-up.
For LCA5, three adults showed sustained visual acuity improvements through 24 months; three adolescents showed improved cone-mediated vision, with sensitivity reaching normal ranges through six months. Both groups showed improved light sensitivity compared with untreated eyes. BEST1 high-dose Cohort 2 completed enrollment with 11 participants. LCA5 advanced to Phase 3 at the low dose without further dose escalation; its natural history and treatment cohorts are fully enrolled. Opus expects Phase 3 dosing to begin in the fourth quarter of 2026.
Opus Genetics (Nasdaq: IRD) announced that management will present at the Chardan Annual Healthcare Conference on October 20, 2026.
The presentation is scheduled for 10:00 a.m. ET. A link to the live and archived webcast will be available on the company’s website under Investors: Events.
Opus Genetics (IRD) reported positive low‑dose Cohort 1 data from its Phase 1/2 BIRD‑1 trial of OPGx‑BEST1 in BEST1‑related retinal disease and alignment with FDA on a potential pivotal endpoint.
All five participants treated at 1.5 x 10⁹ vg/eye showed clinically meaningful visual function improvements, with structural retinal improvements in four. Best‑corrected visual acuity improved in 3 of 5 participants, low‑luminance visual acuity and contrast sensitivity each improved in 2 of 5, and 3 of 4 evaluable participants showed improved retinal sensitivity on microperimetry. No serious adverse events, dose‑limiting toxicities, intraocular inflammation, or notable lab/vital‑sign issues were observed; all treatment‑related adverse events were mild or moderate. The FDA agreed that ≥3 dB microperimetry improvement anchored to patient‑reported outcomes may serve as a pivotal endpoint, supporting planning for Phase 3 dosing in 2027. Cohort 2 at 4.5 x 10⁹ vg/eye has over‑enrolled to eight participants, with dosing completion expected in Q4 2026 and 3‑month topline data in Q2 2027. The company expects its cash runway to extend into 2029.
Opus Genetics (IRD) will present topline data from Cohort 1 of its Phase 1/2 OPGx-BEST1 gene therapy study during an investor conference call and webcast on Wednesday, September 9, 2026, at 8:00 a.m. ET.
The company will later share the full clinical dataset and additional analyses on visual function at multiple ophthalmology meetings, including Euretina Innovation Spotlight on September 30, 2026, Euretina 2026 on October 4, 2026, Retina in Rio on October 14, 2026, and a FLORetina presentation. These sessions will cover initial clinical results, structure-function correlations in BEST1-related retinopathy, and related gene augmentation findings in inherited retinal diseases.
Longeveron (NASDAQ: LGVN) appointed Nirav Jhaveri, CFA, as Chief Financial Officer, principal financial officer and principal accounting officer, joining the executive leadership team and succeeding Marie Washburn, who returns to her prior role as Corporate Controller.
According to Longeveron, Jhaveri brings over 25 years of experience in capital markets, corporate finance, business development and investor relations, including more than 15 years in the biotech sector and prior CFO roles at Opus Genetics (Nasdaq: IRD), Insilico Medicine (HKEX: 3696.HK) and Journey Medical (Nasdaq: DERM). The company also highlighted ongoing development of its stem cell therapy laromestrocel, with top-line data from a Phase 2b trial in HLHS expected in September 2026.
Opus Genetics (Nasdaq: IRD) reported that its Board’s Compensation Committee approved equity awards under the company’s 2021 Inducement Plan, as amended, as a material inducement to hiring three new non-executive employees. The grants were made in accordance with Nasdaq Listing Rule 5635(c)(4).
The equity awards consist of options to purchase an aggregate of 310,000 shares of Opus Genetics common stock, with an exercise price equal to the fair market value on the grant date. The options vest over four years: 25% on the one-year anniversary of each hire date, and the remaining 75% in quarterly installments thereafter, subject to possible acceleration or forfeiture under the applicable award agreements.
Opus Genetics (Nasdaq: IRD) reported second quarter 2026 license and collaborations revenue of $0.8 million, down from $2.9 million a year earlier, and a net loss of $8.1 million, or $0.08 basic and $0.14 diluted per share. R&D expenses rose to $11.2 million from $6.0 million, while G&A expenses were $6.0 million versus $5.8 million.
As of June 30, 2026, cash and cash equivalents were $88.8 million, and the company expects existing cash plus potential note funding to support operations into 2029. Opus Genetics completed Cohort 1 enrollment in its OPGx‑BEST1 Phase 1/2 trial with 3‑month topline data targeted for the second week of September 2026 and finalized the registrational Phase 3 OPGx‑LCA5 trial design with enrollment complete and dosing planned for Q4 2026. Additional gene therapy programs targeting RDH12, MERTK and RHO are expected to enter clinical testing between Q4 2026 and the second half of 2027.
Opus Genetics (Nasdaq: IRD) announced completion of patient enrollment in its registrational Phase 3 trial of OPGx-LCA5, an investigational gene therapy for LCA5-associated inherited retinal disease. The study design was developed with the U.S. FDA and aligned through the Rare Disease Evidence Principles (RDEP) program.
According to Opus Genetics, the one-time subretinal therapy is being evaluated for safety and efficacy using a six-month run-in period where patients serve as their own control. Dosing is expected to begin in Q4 2026, with topline six-month efficacy data targeted by year-end 2027, potentially supporting a Biologics License Application with 12-month durability data submitted during review. OPGx-LCA5 holds FDA Rare Pediatric Disease, Orphan Drug, and RMAT designations and may qualify for a Rare Pediatric Disease Priority Review Voucher upon approval.