Rznomics Inc. Secures Australian Clinical Trial Notification for RHO-targeted Splicing Ribozyme-based RNA Editing Therapy for Retinitis Pigmentosa

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.

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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