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."