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SEONGNAM, South Korea, July 15, 2024 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, announced that it has received Clinical Trial Notification (CTN) from the Australian Therapeutic Goods Administration (TGA) for the initiation of a Phase 1/2a clinical trial evaluating RZ-004, a gene therapeutic candidate for autosomal dominant Retinitis pigmentosa with rhodopsin mutation. Retinitis pigmentosa is an inherited retinal disease in which photoreceptor cells in retina degenerates and leads to blindness. Autosomal dominant Retinitis pigmentosa is caused by numerous diverse genetic defects and mutations in rhodopsin is the most common cause of autosomal Retinitis pigmentosa. More than 150 mutation sites in rhodopsin have been identified and it causes difficulty in treating rhodopsin-mediated autosomal dominant Retinitis pigmentosa. RZ-004, AAV vector encoding a rhodopsin RNA-targeting trans-splicing ribozyme along with wild type rhodopsin, specifically targets and reprograms pathogenic mutant Rhodopsin mRNA into normal Rhodopsin mRNA, thereby leading to the therapeutic effect. Since RZ-004 targets conservative upstream region of the rhodopsin mutations, all different types of mutations in each patient could be treated with one therapy. "The Clinical Trial Notification (CTN) marks an important milestone in addressing the high unmet medical need of autosomal dominant retinitis pigmentosa with rhodopsin mutation," said Dr. Seong-Wook Lee, Chief Executive Officer of Rznomics. "Rznomics is committed to developing innovative RNA-based biopharmaceuticals for the treatment of various human rare and intractable diseases, and we look forward to the initiation of the clinical trial to evaluate the safety and efficacy profile of RZ-004 for providing a potential treatment option for patients suffering from the disease." About Rznomics Inc. Rznomics is a biopharmaceutical company founded in 2017 with the vision of developing RNA-based gene therapies for cancer and incurable diseases. Rznomics' core platform technology is RNA editing technology based on an RNA replacement enzyme, known as trans-splicing ribozyme, which can specifically target and cleave disease-causing RNA and simultaneously replace the whole part of targeted downstream RNA with therapeutic RNA to selectively and regulatedly induce therapeutic gene expression in cells expressing the target RNA. The RNA replacement enzyme platform is being developed for numerous indications such as malignant diseases including hepatocellular carcinoma and glioblastoma, retinitis pigmentosa, rett syndrome, and alzheimer's disease. For more information, please visit SOURCE Rznomics Inc.

기사원문 Rznomics gets approval from Clinical Trial Plan for Genetic Retinopathy Rznomics gets approval from Clinical Trial Plan for Genetic Retinopathy Rznomics announced on the 8th that it has received a notification from the Australian Federal Drug Administration (TGA) on the completion of the approval process for its phase 1 clinical trial plan for RZ-004, which is being developed as a treatment for hereditary retinal pigment degeneration. In order to conduct clinical trials of gene therapy drugs in Australia, it is necessary to undergo preliminary deliberation from the Office of the Gene Technology Regulator (OGTR) as a preliminary stage for approval of clinical plans. Rznomics completed the procedure in January of this year. This means that all deliberation and administrative procedures for clinical initiation in Australia have been completed. Retinitis Pigmentosa is a disease in which visual cells are damaged and the visual field becomes narrower, resulting in loss of vision. As pigment builds up in the retina, retinal function disappears, which is well known as a rare and incurable disease. It occurs in one out of every 3,500 to 4,000 people worldwide. RZ-004 removes the mutated Rhodopsin ribonucleic acid (RNA) and replaces it with a normal rhodopsin gene using the RNA trans-splicing ribozyme Editing Technology owned by RZ-004 to induce vision damage suppression and vision recovery. In particular, RZ-004 has a groundbreaking feature that can correct more than 150 different rhodopsin mutations known so far with a single treatment. Australia has excellent medical system and medical staff capabilities, the number of target patients, patient compliance, and support from regulatory agencies, said Sung-woo Hong, head of the development division of Rznomics. "We will expand our clinical development countries to the United States and Europe, starting with Australia." The approval of the clinical plan means that it has entered the clinical stage for genetic diseases that can make good use of the characteristics of the company's platform technology following the clinical entry of several anticancer drug pipelines, said Seong-wook Lee, CEO of Rznomics. "Since the target disease of RZ-004 is a disease with very high unmet medical demand without any approved treatment, we will do our best to provide treatment opportunities to suffering patients and improve their quality of life."

$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