Opus Genetics Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for OPGx-LCA5 Gene Therapy Candidate
- FDA RMAT designation received for OPGx-LCA5 gene therapy, potentially accelerating development and review
- Encouraging early clinical data from Phase 1/2 trial
- Additional FDA support through RMAT Meeting and CMC Development program participation
- None.
Insights
FDA RMAT designation for Opus Genetics' OPGx-LCA5 signals regulatory momentum and enhanced development pathway for this rare blindness gene therapy.
The FDA's grant of Regenerative Medicine Advanced Therapy (RMAT) designation to Opus Genetics' OPGx-LCA5 gene therapy represents a significant regulatory achievement that could substantially accelerate development timeframes. This designation is exclusively awarded to regenerative medicine therapies showing meaningful preliminary clinical evidence addressing serious conditions with unmet medical needs. The designation confers three critical advantages: expedited regulatory review procedures, enhanced engagement with FDA officials through early interactions, and specialized guidance for streamlining development and manufacturing pathways.
Particularly noteworthy is Opus's dual invitation to participate in the FDA's Initial Comprehensive Multidisciplinary RMAT Meeting and the Chemistry, Manufacturing and Controls Development and Readiness Pilot (CDRP) program. These supplementary regulatory engagements provide invaluable guidance opportunities that could optimize both clinical development strategy and manufacturing processes—the latter being especially crucial for gene therapies where production complexity often creates bottlenecks.
For an ultra-rare indication like LCA5-mediated Leber Congenital Amaurosis, these regulatory advantages could meaningfully compress development timelines while potentially reducing costs. The designation effectively acknowledges both the promise of Opus's preliminary clinical data and the significant unmet need in this patient population, positioning the company's lead program for enhanced regulatory support throughout its continued development.
RMAT designation validates Opus's early clinical data for LCA5 gene therapy, offering hope for patients with this untreatable inherited blindness.
The RMAT designation granted to Opus Genetics' OPGx-LCA5 represents meaningful validation for patients with LCA5-associated Leber Congenital Amaurosis, an ultra-rare inherited retinal disease that typically causes severe vision impairment from infancy. This condition results from mutations in the LCA5 gene, which encodes the protein lebercillin—critical for photoreceptor ciliary function. When this protein is dysfunctional, progressive and severe vision loss occurs.
While the press release doesn't provide specific efficacy metrics, the FDA's decision to grant RMAT designation indicates that preliminary data from Opus's ongoing Phase 1/2 trial demonstrated sufficient promise to warrant enhanced regulatory support. This suggests the therapy is showing signals of potential benefit in this previously untreatable condition. The designation specifically acknowledges both the therapy's early clinical evidence and the significant unmet need in this patient population.
The development of gene therapies for inherited retinal diseases has advanced significantly in recent years, establishing proof-of-concept for ocular gene delivery approaches. For LCA5 patients who currently have no approved treatment options, this regulatory milestone represents important progress, though significant clinical development work remains ahead. The ultra-rare nature of the condition creates both clinical trial challenges (small patient populations) and regulatory opportunities (potential for approval with smaller datasets), making the RMAT designation particularly valuable for this development program.
- FDA RMAT designation recognizes encouraging early data from Opus’ Phase 1/2 trial in patients with LCA5, an ultra-rare inherited retinal disease
RESEARCH TRIANGLE PARK, N.C., May 06, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmic disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, its investigational gene therapy for the treatment of Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene.
The RMAT designation for OPGx-LCA5 is based on early clinical evidence from Opus’s ongoing Phase 1/2 open-label, dose-escalation trial, which is evaluating the safety and potential efficacy of OPGx-LCA5 in patients with severe vision loss due to confirmed mutations in the LCA5 gene. The FDA’s decision indicates recognition of the strength of this initial data and the unmet need in this patient population.
“The FDA’s decision to grant RMAT designation to OPGx-LCA5 is a major milestone for the LCA5 patient community and a strong validation of our early clinical data,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “We’re encouraged by the potential of OPGx-LCA5 to meaningfully impact patients living with this ultra-rare and debilitating form of inherited blindness, and we look forward to continued collaboration with the FDA to accelerate its development.”
In addition to the RMAT designation, Opus has been invited to participate in the FDA’s Initial Comprehensive Multidisciplinary RMAT Meeting to support Opus’ development and manufacturing strategy. Lastly, Opus has been invited to join the FDA’s Chemistry, Manufacturing and Controls (CMC) Development and Readiness Pilot (CDRP) program, which provides additional guidance for accelerating CMC development of products under an investigational new drug application.
The RMAT designation program offers the potential for expedited development and review of regenerative medicine therapies that demonstrate the potential to address serious or life-threatening diseases based on preliminary clinical evidence. The designation provides sponsors with early interactions with the FDA, guidance on efficient development and manufacturing, and the opportunity to discuss surrogate endpoints to support accelerated approval.
About OPGx-LCA5
OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial with Dr. Tomas Aleman at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.
About Opus Genetics
Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing therapies to treat patients with inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders. Our pipeline includes adeno-associated virus (AAV)-based investigational gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. Our most advanced investigational gene therapy program, which has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. OPGx-LCA5 is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial, with encouraging early data. Our pipeline also includes BEST1 investigational gene therapy, designed to address mutations in the BEST1 gene, which is associated with retinal degeneration. The pipeline also includes Phentolamine Ophthalmic Solution
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning our interactions with the FDA, our participation in the CDRP program, and the effects of RMAT designation for OPGx-LCA5.
These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our 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 our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our 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. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
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- Clinical data referenced in this press release is preliminary and related to a relatively small group of patients, and, as a result, data that initially appears promising may be revised, updated, or invalidated at a later data readout and/or may ultimately not be capable of duplication in additional patients;
- Failure to successfully integrate our businesses following our acquisition of former Opus Genetics Inc. (the Opus Acquisition) could have a material adverse effect on our business, financial condition and results of operations;
- The Opus Acquisition significantly expanded our product pipeline and business operations and shifted our business strategies, which may not improve the value of our common stock;
- Our gene therapy product candidates are based on a novel technology that is difficult to develop and manufacture, which may result in delays and difficulties in obtaining regulatory approval;
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Contacts:
Corporate
Nirav Jhaveri
CFO
ir@opusgtx.com
Investor Relations
Corey Davis, Ph.D.
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FAQ
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