Rznomics Inc. Gains Australian Approval for RNA Therapy Trial for Retinitis Pigmentosa

Rznomics Inc., a biopharmaceutical company based in South Korea, has announced a significant development in their efforts to treat autosomal dominant Retinitis pigmentosa (ADRP) with rhodopsin mutation. The company has received approval from the Australian Therapeutic Goods Administration (TGA) to commence a Phase 1/2a clinical trial for their gene therapeutic candidate, RZ-004.

ADRP is a genetic disorder that causes the degeneration of photoreceptor cells in the retina, leading to progressive vision loss and eventual blindness. It is primarily caused by various genetic defects, with mutations in the rhodopsin gene being the most prevalent. The diversity of mutation sites in rhodopsin, over 150 identified to date, has posed significant challenges in developing effective treatments for this form of Retinitis pigmentosa.

RZ-004 is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver a rhodopsin RNA-targeting trans-splicing ribozyme, along with wild type rhodopsin, into the patient’s cells. This innovative approach allows for the reprogramming of pathogenic mutant Rhodopsin mRNA into its normal form. By targeting the conserved upstream region of the rhodopsin mutations, RZ-004 has the potential to treat all different types of rhodopsin mutations with a single treatment.

Dr. Seong-Wook Lee, the Chief Executive Officer of Rznomics, highlighted the importance of this milestone, noting that the Clinical Trial Notification (CTN) is a crucial step towards addressing the significant unmet medical needs of patients with ADRP due to rhodopsin mutation. Dr. Lee expressed the company’s commitment to developing RNA-based biopharmaceuticals aimed at treating various rare and challenging diseases. He emphasized the anticipation surrounding the clinical trial, which will evaluate the safety and efficacy of RZ-004 as a potential treatment option for those afflicted by this retinal disease.

Rznomics Inc., established in 2017, focuses on the development of RNA-based gene therapies for cancer and other incurable conditions. The company’s core technology revolves around RNA editing, utilizing a trans-splicing ribozyme that can specifically target and cleave disease-causing RNA. This enzyme also has the capability to replace the targeted RNA with therapeutic RNA, thereby inducing the expression of therapeutic genes in cells that express the target RNA. This RNA replacement platform is being explored for a range of indications, including various malignant diseases such as hepatocellular carcinoma and glioblastoma, as well as other conditions like Retinitis pigmentosa, Rett syndrome, and Alzheimer's disease.

The initiation of the clinical trial for RZ-004 represents a promising advancement in the treatment of ADRP with rhodopsin mutation. If successful, this therapy could offer a much-needed solution for patients suffering from this debilitating condition, demonstrating the potential of RNA-based therapeutics in tackling genetic disorders.