Rznomics announces result of New Drug Pre-clinical Treatment for Retinal Dysplasia' at American Society of Gene & Cell Therapy (ASGCT)

RNA Editing 'RZ-004' pipeline plans To apply for FDA Clinical Trial at year-end of 2023.

Rznomics, announced on the 22nd that it has announced the results of a pre-clinical study of its pipeline "RZ-004," a candidate for treatment for retinal pigment degeneration, at the American Society of Gene & Cell Therapy (ASGCT) held in Los Angeles.

RZ-004 received a lot of attention on its potential as a new treatment through the announcement of the Association for Research in Vision and Ophthalmology (ARVO) last month. About a month later, the American Society of Gene & Cell Therapy selected it as oral presentation topic. Research results such as material optimization and pre-clinical test results have been announced.

The presentation was made by Dr. Ji-hyun Kim, in-charge of genetic diseases at Rznomics. RZ-004 has an RNA editing mechanism that removes mutated rhodopsin ribonucleic acid (RNA) and replaces it with a normal rhodopsin gene be in the pre-clinical stage of completion. Pre-clinical results that can enter clinical trials, such as efficacy, safety, and toxicity test results confirmed by animal models with various diseases, have been accumulated by the company.

The effectiveness was demonstrated through electroretinogram (ERG) and optical coherence tomography (OCT). It also confirmed safety by using rodent, pig models and non-human primates. It is planning to apply for clinical trials with the U.S. Food and Drug Administration (FDA) at the end of the year.

Dr. Ji-hyun Kim said, "The results of RZ-004 were selected as the topic of oral presentation by ASGCT, the most prestigious academic society in the field of gene and cell therapy," adding, "I am happy for this opportunity to announce RZ-004 to the world."

Seong-wook Lee, CEO of Rznomics, said, "RZ-004's target rhodopsin mutation-mediated autosomal dominant Retinitis Pigmentosa is very demanding because currently there is no treatment," adding, "we will do our best to provide patients with innovative treatment opportunities as soon as possible."