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."