LCA5

Priority Review Voucher would be issued upon approval of OPGx-LCA5 Aug. 20, 2024 -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPD) for its ocular gene therapy OPGx-LCA5 to treat patients with the inherited retinal disease LCA5. OPGx-LCA5 is an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). “We are thrilled to receive Rare Pediatric Disease designation from the FDA for our OPGx-LCA5 gene therapy. This important milestone brings us closer to delivering a potential treatment for patients with LCA5,” said Ben Yerxa, Ph.D., chief executive officer of Opus. “At Opus, we’re committed to advancing therapies that help treat patients with inherited retinal diseases, and this designation further validates the potential impact of our innovative ocular gene therapy approach. We look forward to providing updates on the Phase 1/2 clinical trial evaluating OPGx-LCA5 soon.” LCA5 is a form of early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. Currently, there are no approved treatments for individuals with LCA5-related vision loss. OPGx-LCA5 is currently being studied in an open-label, dose-escalation Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in 15 patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. For more information on the trial, visit clinicaltrials.gov (NCT05616793). The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. By obtaining this designation, Opus has access to valuable incentives and support from the FDA during its development program. With this designation, OPGx-LCA5 will be eligible to receive a priority review voucher upon approval for any subsequent marketing application that can be sold or transferred to other companies. 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 is an early-onset severe inherited retinal dystrophy. Studies in LCA5 patients 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 at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in 15 patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’ venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com. The content above comes from the network. if any infringement, please contact us to modify.

$1M TRAP award to support preclinical safety study for gene therapy vector targeting rhodopsin- RHO-adRP Additional project and operational funding to support MERTK gene therapy IND-enabling studies RESEARCH TRIANGLE PARK, N.C., June 13, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced it has received $1.7 million in project-based funding from the Foundation Fighting Blindness to help advance two preclinical candidate programs. “Opus is immensely grateful for the generous support from the Foundation Fighting Blindness, which will catalyze our efforts in pioneering treatments for inherited retinal diseases. This $1.7 million funding infusion makes a significant impact to help accelerate the development of these two preclinical candidates,” said Ben Yerxa, Ph.D., chief executive officer of Opus. “Together, we strive toward a future where therapies to help treat patients with inherited retinal diseases are readily available.” Opus received a $1M Translational Research Acceleration Program (TRAP) award to conduct a preclinical safety study for a gene therapy vector designed to target rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP). The safety study will be conducted in the established canine animal model at the University of Pennsylvania School of Veterinary Medicine (Penn Vet). The Company anticipates this is the last preclinical study required before the gene therapy will enter clinical trials. RHO-adRP is one of the most common IRDs, estimated to affect approximately one in 51,000 people, or more than 6,000 people, in the United States alone. Additionally, Opus received approximately $700,000 in project-based and operational funding to support the preclinical development of a novel viral vector for treating retinitis pigmentosa due to mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene. The Company is collaborating with the Foundation to begin IND-enabling studies for a newly designed adeno-associated virus (AAV) viral vector to replace mutated MERTK genes in the retinal pigmented epithelial (RPE) cells of the retina. The initial funding will provide the resources for testing the vector in an established animal model of the disease and to conduct early safety assessments in larger animals. MERTK mutations cause a rod-cone dystrophy with early macular atrophy, and retinitis pigmentosa is the most common retinal phenotype. “We're excited to announce our funding commitment to Opus, a trailblazer in inherited retinal disease therapeutics and a company created based on our mission of ultimately curing blindness caused by retinal degenerative diseases. This investment highlights our steadfast dedication to hastening innovative solutions for those combating inherited retinal diseases,” said Jason Menzo, chief executive officer of the Foundation Fighting Blindness. “In collaboration with Opus, we're propelling forward promising clinical candidate programs with the potential to revolutionize the lives of those affected by these challenging conditions.” About Foundation Fighting BlindnessEstablished in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $816 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information. About Opus GeneticsOpus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’ venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com. Media Contact:Gina Mangiaracina6 Degrees gmangiaracina@6degreesPR.com

OPGx-LCA5 will advance to the next highest dose in mid-2024 based on positive safety and efficacy data OPGx-LCA5 is well-tolerated and demonstrated clear signs of biological activity March 26, 2024 -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). Based on positive safety and efficacy data from the first cohort of three adult patients, the Company will advance OPGx-LCA5 into the next highest dose. Opus anticipates initiating the next cohort mid-2024. There are also future plans to expand the study population to include subjects 13 years or older. “In the first cohort, OPGx-LCA5 has been well-tolerated and demonstrated clear signs of biological activity through 90 days, warranting continued evaluation in the next highest dose,” said principal investigator Tomas S. Aleman, M.D., from the Center for Advanced Retinal and Ocular Therapeutics (CAROT), of the Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania. “Moreover, early anecdotal and VR challenge test feedback is encouraging and indicates that some of the patients, who have been nearly totally blind all their lives, are now able to see and identify objects for the first time.” LCA5 is a form of an early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. Currently, there are no approved treatments for individuals with LCA5-related vision loss. “Based on these early clinical data, we’re excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5,” said Ben Yerxa, Ph.D., chief executive officer of Opus. “We’d like to thank the study participants and their families and the incredible efforts of the team at the University of Pennsylvania for reaching this clinical milestone, and we look forward to progressing the trial as we continue to dose escalate.” 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 is an early-onset severe inherited retinal dystrophy. Studies in LCA5 patients 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 at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in nine patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Opus Genetics is a clinical-stage gene therapy company for inherited retinal diseases with a unique model and purpose. Backed by Foundation Fighting Blindness’ venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous orphan retinal diseases. Its AAV-based gene therapy portfolio, including a derisked LCA5 lead program currently in a Phase 1/2 clinical trial, tackles some of the most neglected forms of inherited blindness while creating novel orphan manufacturing scale and efficiencies. Based in Research Triangle Park, N.C., the company leverages knowledge of the best science and the expertise of pioneers in ocular gene therapy to transparently drive transformative treatments to patients. For more information, visit www.opusgenetics.com. Thecontent above comes from the network. if any infringement, please contact us to modify.