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Opus Genetics (Raleigh, North Carolina) has announced new preclinical data from studies evaluating gene therapies for OPGx-001 and OPGx-002.

Opus Genetics Inc., a private ocular gene therapy company based in Raleigh, North Carolina, has announced new preclinical data from studies evaluating gene therapies, termed OPGx-001 and OPGx-002, aimed to address Leber congenital amaurosis (LCA).  The company has reported that preparation for IND-enabling trials of OPGx-001 and OPGx-002 have “conducted to determine eligibility, therapeutic window, and possible outcome measures for gene therapy for LCA5 and RDH12 inherited retinal diseases”. In addition, safety evaluations for the subretinal delivery of an AAV8 vector containing LCA5 or RDH12 were performed in non-human primates (NHP).


Opus Genetics is supported by the Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments and cures for the entire spectrum of retinal degenerative diseases. The company’s lead programs are licensed from the University of Pennsylvania and will focus on treatments to address mutations in genes that cause different forms of Leber congenital amaurosis (LCA). According to the company, “AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies. The company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients.”


According to Opus’s lead program, OPGx-001, is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein. Preclinical data, including animal and human iPSC models, have demonstrated preservation of visual function when OPGx-001 was administered prior to peak disease severity, indicating the potential for a broad therapeutic window for patients with LCA5. The OPGx-001 treatment expects to file an IND in early 2022 and enter the clinic in mid-2022.  In addition, Opus’s second program, OPGx-002, aims to restore protein expression and halting functional deterioration in patients with retinal dystrophy caused by mutations in the retinal dehydrogenase (RDH12) gene.  Again, preclinical studies of OPGx-002 in cellular and animal models have demonstrated functional improvement and efficacy, including the restoration of RDH12 activity to approaching wild type levels in animal models with mutations in the RDH12 gene. Finally, the company anticipates an IND in the next 12-18 months


Following the announcement, Ash Jayagopal, Ph.D., Chief Scientific Officer of Opus commented that, “patients with Leber congenital amaurosis due to mutations of the LCA5 or RDH12 genes experience rapid retinal degeneration, resulting in vision loss in early childhood.  The detection of preserved photoreceptors in LCA patients signals a therapeutic opportunity to target the mutation and potentially restore structure and function through gene augmentation. In addition, the encouraging dose-ranging results in the primate model suggest subretinal delivery of Opus’ AAV8-based gene therapies are safe and inform the doses to be used in our toxicology studies, a key step on our path toward the clinic for OPGx-001 and OPGx-002.”