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RESEARCH TRIANGLE PARK, N.C. - Opus Genetics, Inc. (Nasdaq: IRD), a biotechnology firm specializing in gene therapies for inherited retinal diseases (IRDs), has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for their investigational gene therapy, OPGx-LCA5. The company, currently valued at $43.1 million, maintains a strong financial position with more cash than debt on its balance sheet, according to InvestingPro data. This therapy is designed to treat Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene.
The RMAT designation, announced today, is grounded in preliminary clinical data from an ongoing Phase 1/2 trial. The trial is assessing the safety and potential efficacy of OPGx-LCA5 in patients with severe vision loss caused by confirmed mutations in the LCA5 gene. The FDA’s recognition reflects the initial data’s promise and the significant need for treatment in this patient community.
"This is a major milestone for the LCA5 patient community and a strong validation of our early clinical data," stated George Magrath, M.D., CEO of Opus Genetics. The company is optimistic about the therapy’s potential to improve the lives of patients with this rare and debilitating form of inherited blindness. The market appears to share this optimism, with the stock showing a notable 9.54% gain over the past week, though trading at $0.95 remains well below analyst targets ranging from $6 to $15.
Additionally, Opus Genetics will participate in the FDA’s Initial Comprehensive Multidisciplinary RMAT Meeting, which aims to support the company’s development and manufacturing strategy. The firm has also been invited to join the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program. This program offers further guidance for accelerating CMC development for investigational new drugs.
The RMAT designation is part of a program that facilitates expedited development and review for regenerative medicine therapies that show potential to address serious or life-threatening diseases based on early clinical evidence. Benefits of the designation include early FDA interactions, development and manufacturing guidance, and the possibility of discussing surrogate endpoints for accelerated approval.
OPGx-LCA5 utilizes an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene to the outer retina. The therapy is currently under evaluation in a Phase 1/2 clinical trial led by Dr. Tomas Aleman at the University of Pennsylvania.
Opus Genetics focuses on developing AAV-based gene therapies for various IRDs, including bestrophinopathy, LCA, and retinitis pigmentosa. Their pipeline also features therapies for other ophthalmic conditions such as presbyopia and diabetic retinopathy.
This report is based on a press release statement from Opus Genetics, Inc.
In other recent news, Opus Genetics has reported promising results from its Phase 1/2 clinical trial of the gene therapy OPGx-LCA5, aimed at treating LCA5-related inherited retinal disease. A 16-year-old pediatric patient showed significant visual improvement after receiving a subretinal injection, with no adverse events reported. Additionally, Opus Genetics successfully closed a $20 million public offering and private placement, which will support corporate expenses and product development. The company sold shares and warrants, with Craig-Hallum Capital Group acting as the sole underwriter for the public offering. In terms of analyst activity, Craig-Hallum initiated a Buy rating on Opus Genetics with a $6 price target, citing the potential of its gene therapy pipeline. H.C. Wainwright also reiterated its Buy rating, maintaining an $8 price target, and expressed optimism about upcoming data presentations and regulatory interactions. Furthermore, Opus Genetics held its annual stockholder meeting where key governance proposals were approved, including the conversion of Series A Preferred Stock into Common Stock. These developments reflect the company’s ongoing efforts to advance its gene therapy treatments and strengthen its financial and operational standing.
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