Opus Genetics (NASDAQ: IRD) is quickly becoming one of the most closely watched players in inherited retinal disease (IRD) research. Recently, the company has made headlines with new partnerships, regulatory milestones, and forward-looking insights from its CEO as it advances a pipeline of gene therapies for rare inherited retinal diseases that can cause severe vision loss from childhood through adulthood.
Notable Clinical and Regulatory Momentum
Last week, Opus announced that the FDA cleared its IND application for OPGx-BEST1, a gene therapy for BEST1-related inherited retinal disease (IRD). This enables initiation of a Phase 1/2 clinical trial in the second half of 2025, evaluating safety, tolerability, and preliminary efficacy in patients with genetically confirmed BEST1 mutations.
“The FDA’s clearance of our BEST1 IND is a significant step forward for the IRD community and for our mission at Opus Genetics focused on restoring vision for patients,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “BEST1-related IRDs have no approved treatments today, leaving patients and families with uncertainty about the future of their vision. The OPGx-BEST1 trial will be our third ongoing clinical program, reflecting the depth of our pipeline and our commitment to advancing multiple therapies in parallel for patients with urgent, unmet needs.”
Opus also reported positive 12-month Phase 1/2 clinical data for its lead program, OPGx-LCA5, showing sustained vision gains in adult patients and early improvements in children. The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation, a key regulatory milestone that can expedite development and review. Pediatric data from the trial are expected in Q3 2025.
Beyond gene therapy, the company reported successful Phase 3 trial results for Phentolamine Ophthalmic Solution 0.75%, a partnered small-molecule therapy that could expand into new vision-related indications with supplemental FDA submissions later this year.
New Partnership to Advance Gene Therapy for RDH12-Related Blindness
In late July, Opus announced a collaboration with the Global RDH12 Alliance to accelerate development of OPGx-RDH12, a gene therapy candidate targeting RDH12 mutations that cause Leber congenital amaurosis (LCA). Leber congenital amaurosis is a rare IRD that causes progressive vision loss and blindness, often beginning in early childhood.
The Alliance, which unites advocacy groups in the U.S. and UK, will provide up to $1.6 million in non-dilutive funding to support development. The goal is to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) by late 2025. Preclinical studies have already demonstrated restoration of RDH12 activity and improved retinal function.
“This partnership represents a significant step forward,” said Dr. Mathew Pletcher, board member of the RDH12 Fund for Sight and parent of a child with the disease. “By combining our community’s perspectives with Opus’ gene therapy expertise, we can accelerate the transition of this therapy into the clinic.”
CEO Insights: AI and Efficiency in Rare Disease Trials
In an op-ed for “The Medicine Maker,” CEO Dr. George Magrath highlighted how artificial intelligence (AI) is reshaping the economics of clinical trials. By automating workflows, monitoring compliance in real time, and improving data accuracy, AI can cut costs and timelines significantly.
“We are pursuing multiple rare disease indications using a structured, sequential approach,” Magrath wrote. “The ability to manage seven assets simultaneously is a direct result of leveraging AI-driven efficiencies.”
For ultra-rare diseases like LCA and bestrophinopathy, a group of inherited eye mutations, these efficiency gains can make the difference between programs that reach patients and those that stall due to cost.
Featured Conversations: Shaping the Future of Gene Therapy
Opus’ leadership is actively engaging with the rare disease and ophthalmology communities.
- At the Chardon Virtual Ophthalmology Summit, Magrath underscored the company’s efficient development model, noting: “Our programs are designed to deliver meaningful clinical data with relatively modest capital, creating a solid investment thesis for shareholders.”
- On the Cell & Gene Podcast, Magrath explained why gene therapy is particularly well suited to IRDs, pointing to “structure-function dissociation,” where retinal cells remain intact despite genetic dysfunction. He emphasized the importance of early intervention to preserve vision and proper neural development, and described Opus’ strategy of building a sustainable business model capable of replicating success across multiple rare IRD mutations.
Investor Outlook
Opus ended Q2 2025 with $32.4 million in cash, which it expects to fund operations into the second half of 2026. Revenue was $2.9 million for the quarter, driven by collaborations with Viatris.
With multiple upcoming catalysts — including pediatric data from OPGx-LCA5, initiation of the OPGx-BEST1 clinical trial following FDA IND clearance, and regulatory filings for Phentolamine — investors will be watching closely.
This article contains sponsored content. Please see our disclaimer at: https://prismmarketview.com/disclaimer/
The post Opus Genetics Expands Clinical Pipeline with FDA Green Light for BEST1 Gene Therapy appeared first on PRISM MarketView.
