Opus Genetics 获 FDA 放行 OPGx-RDH12 IND，第三款 AAV 基因疗法本季度进入 1/2 期临床

FDA clearance enables clinical development of OPGx-RDH12 for RDH12-associated Leber congenital amaurosis, a rare inherited retinal disease that causes progressive vision loss beginning in early childhood
Strategic collaboration with the Global RDH12 Alliance supports the development of a potential treatment for this disease with high unmet need
RESEARCH TRIANGLE PARK, N.C., Oct. 21, 2026 (GLOBE NEWSWIRE) --
Opus Genetics, Inc.
(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 the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for OPGx-RDH12, an investigational gene therapy for the treatment of RDH12-associated Leber congenital amaurosis (RDH12-LCA), a rare inherited retinal disease that causes progressive vision loss and blindness beginning in early childhood.
The IND clearance enables Opus Genetics to advance OPGx-RDH12 into clinical development, marking an important achievement in the Company's collaboration with the Global RDH12 Alliance, which brings together patient advocacy organizations RDH12 Fund for Sight in the United States and Eyes on the Future in the United Kingdom. The partnership was established in July 2025 with the goals of accelerating development of OPGx-RDH12 and bringing a potential treatment to patients living with this devastating condition.
“We are pleased to advance our third gene therapy pipeline program into clinical development and plan to initiate a Phase 1/2 trial this quarter,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “RDH12-LCA causes progressive vision loss beginning at a very young age, underscoring the urgency of developing treatments that may preserve vision before further deterioration occurs. Receiving IND clearance for OPGx-RDH12 represents an important step toward bringing a potentially transformative gene therapy to children and families affected by this devastating disease. We are grateful for the collaboration and commitment of our partners at the Global RDH12 Alliance, which has been instrumental in advancing this program into the clinic.”
"Bringing a treatment to the members of the RDH12-LCA community has been the stated goal of The RDH12 Fund for Sight since its founding more than a decade ago,” said Mathew Pletcher, Ph.D., Board member of the RDH12 Fund for Sight and father to a 20-year-old child living with RDH12-LCA. "From the foundational work, funded by patient dollars, in the lab of Dr. Jean Bennett through today’s announcement, the RDH12-LCA community has been collaborating with partners to ensure the delivery a potentially vision-saving therapy to the clinic."
“This is what true co-development looks like,” said Silvia Cerolini, Chief Executive Officer of Eyes on the Future and mother of a child affected by RDH12-LCA. “Our RDH12 patient organizations and Opus have pooled resources, capabilities and risk to bring this rare gene therapy into the clinic, combining venture philanthropy, scientific innovation and a patient-driven sense of urgency. We have been working toward this moment for many years, and we are grateful to Opus for being a true partner. We believe this model can become a blueprint for bringing gene therapies to rare disease communities, and one that other patient organizations can follow.”
Under the strategic collaboration announced in July 2025, the Global RDH12 Alliance committed up to $1.6 million toward the development of OPGx-RDH12, with eligible development costs reimbursed as activities are completed subject to certain diligence obligations and milestones. The organizations are working together on the clinical and regulatory strategy for the program, integrating the perspectives and experiences of patients and families directly into its development.
About RDH12-LCA and OPGx-RDH12
RDH12-LCA is a rare form of childhood blindness affecting an estimated 30,900 people globally. Mutations in the RDH12 gene impair protein function in the retina, leading to early visual decline, often with structural retinal changes by age two, and rapid progression during adolescence. OPGx-RDH12 is an investigational adeno-associated virus (AAV)-based gene therapy designed to deliver a functional copy of the RDH12 gene directly to photoreceptor cells in the retina. Preclinical studies of OPGx-RDH12 in cell and mouse models have demonstrated restoration of RDH12 activity and functional improvements, supporting clinical evaluation of the therapeutic candidate.
About The RDH12 Fund for Sight
The RDH12 Fund for Sight is a registered 501(c)(3) not-for-profit organization based in the United States. The RDH12 Fund for Sight was founded for the express purpose of connecting and supporting families impacted by RDH12-LCA and to ensure that significant funds are devoted to the study and development of treatments for LCA caused by mutations in RDH12.
About Eyes on the Future
Eyes on the Future is a UK-based not-for-profit organization supporting research into rare diseases and eye diseases, with a specific focus on RDH12 inherited retinal dystrophies. Founded by Enrico and Silvia, whose 12-year-old child is affected by RDH12-LCA, Eyes on the Future is a registered company limited by guarantee in England and Wales (No. 13956181) and a registered charity (No. 1198330). More information is available at
www.eyesonthefuture.org.uk
.
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 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 OPGx-RDH12 cleared to enter the clinic, and additional candidates targeting, MERTK, RHO, CNGB1 and NMNAT1. The Company is based in Research Triangle Park, N.C. For more information, visit
www.opusgtx.com
.
Forward-Looking Statements
This press release contains certain statements that are not statements of historical fact and are forward-looking statements. In some cases, you can identify forward-looking statements by the following words: “aim,” “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “strive,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. Such statements include, but are not limited to, statements related to the Company’s plans for the clinical development of OPGx-RDH12, including the anticipated timing of initiation, and the design, of the Phase 1/2 clinical trial of OPGx-RDH12; the therapeutic potential of OPGx-RDH12, including its potential to preserve vision or otherwise treat RDH12-LCA; the significance of preclinical data for OPGx-RDH12 and whether such data will be predictive of clinical results; the Company’s collaboration with the Global RDH12 Alliance and the potential of this partnership to accelerate development of OPGx-RDH12 and bring a potential treatment to patients living with this devastating condition; the Company’s ability to satisfy diligence obligations and meet development milestones under its agreement with the Global RDH12 Alliance and the potential of the collaboration model to be replicated to bring other gene therapies to rare disease communities; and the Company’s expectations regarding its business prospects and results of operations. There can be no assurance that the Company’s planned Phase 1/b clinical trial of OPGx-RDH12 will be initiated on the anticipated timeline or at all, or that OPGx-RDH12 will be shown to be safe or effective or receive regulatory approval.. The forward-looking statements contained herein are subject to certain risks and uncertainties posed by many factors and events that could cause the Company’s actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2025, its Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, and in the Company’s other filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. These forward-looking statements are based upon the Company’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties. The Company undertakes no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
Contacts:
Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com
Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
Source: Opus Genetics, Inc.