Four domestic and international technologies for 'RNA and gene therapy' announced at KMB on the 22^nd

[KMB 2023] A session of 'RNA and gene therapy' co-hosted by Rznomics will be held..Four speakers, including CEO Seong-wook Lee, Professor Hyuk-jin Lee, and CEO Dong-ki Lee, will be announced

There will be a place to see the development of new drugs for RNA and gene therapy in Korea, including RNA interference (RNA interference), lipid nanoparticles (LNP), RNA-splicing ribozyme, and RNA aptamer. Rznomics will co-host the "RNA and Gene Therapy" session on the 22nd at the Korea Microbiological Biotechnology Association (KMB 2023) held as an international symposium to commemorate the 50th anniversary of its foundation at the Gyeongju Hwabaek Center for three days from the 21st. In this session, Bruce Sullenger, a professor at Duke University, and researchers above Dong-ki Lee's Olix Pharmaceutical are expected to participate in the session to share innovative research results and the latest knowledge. The first speaker, Professor Bruce Sullenger, is a professor of surgery at Duke University's University of Medicine in the U.S. and vice chairman of the recently established Society for RNA Therapeutics. Professor Sullenger has been developing nucleic acid treatments for cardiovascular disease, stroke, and cancer treatment for more than 30 years in the field of RNA treatments.

At this conference, Professor Sullenger will present preclinical and clinical resistance data of nuclease-resistive modulated RNA aptamer (BB-301) that binds to VWF (Won Willebrand Factor) as a treatment target for acute ischemic stroke (AIS) diseases.

So far, the only AIS treatment approved by the U.S. Food and Drug Administration (FDA) is the rtPA (recombinant tissue plasminogen activator), and it is limitedly used within 4.5 hours of stroke due to the risk of causing cerebral hemorrhage. rtPA administration is limited to patients within 6% of the total. Among them, the role of VWF in the overall blood clot process has recently been revealed.

 

The last speaker, CEO Seong-wook Lee, is a professor of bio-convergence engineering at Dankook University and served as the president of the Korean Nuclear Acid Society and the Korean Genetic Cell Therapy Society. CEO Lee is a leading researcher in the field of trans-splicing ribozyme technology, and has been researching the field for more than 20 years to optimize the technology, and based on this, he founded Rznomics.

Rznomics will announce the development of rare incurable treatments such as cancer, degenerative and genetic diseases, and the results of preclinical candidates for new drugs based on RNA replacement enzymes. RNA replacement enzyme technology is a concept that removes target RNA while expressing a therapeutic gene (transgen).

Last year, Rznomics was approved by the FDA and the Ministry of Food and Drug Safety to develop phase 1/2a of the RNA replacement enzyme candidate "RZ001" for liver cancer patients, and in May this year, it was approved to expand the adaptation to glioblastoma. RZ-001 is a mechanism that targets telomerase (hTERT) RNA that is specifically expressed by cancer cells by delivering ribonucleic acid replacement enzymes to adenovirus vectors and at the same time expresses genes that induce anticancer action.

In the second half of this year, Rznomics plans to submit a clinical trial plan (IND) for phase 2 clinical trials to evaluate the effect of RZ-001 and immuno-cancer drugs in liver cancer and "RZ-004," a candidate for retinitis pigmentationosa (RP) treatment, an ophthalmic genetic disease.

Seong-wook Lee, CEO of Rznomics, said, "I recently participated in conferences and business conferences such as the American Society for Gene Cell Therapy (ASGCT), the American Society for Clinical Oncology (ASCO), and BIOUS. I hope this will serve as an opportunity to seek international industry-academic cooperation and development."