Cargene Bio Announces Latest Advances in Novel siRNA Therapy CG-LR1 for Liver Cirrhosis

Dr. Tan Si Hui, Senior Director of Biology at CARGENE BIOPHARMA, will deliver an oral presentation at the 2023 Cold Spring Harbor Asia Liver Conference (Suzhou) to report the research progress of the self-developed small nucleic acid drug CG-LR1 in promoting liver regeneration and protecting liver function, along with a series of preclinical data.

At the same time, Dr. Torsten Wuestefeld, co-founder of CARGENE BIOPHARMA, will also give an oral presentation at this conference on the liver regeneration mechanism target screening platform they have developed, and showcase new targets screened from the platform.

CARGENE BIOPHARMA is an innovative drug discovery startup. Leveraging the capital, technology, and talent advantages of Shanghai, Boston, and Singapore, combined with the team's deep understanding of novel biological targets and years of industry experience in nucleic acid drug chemistry, CARGENE BIOPHARMA focuses on developing new nucleic acid drugs for liver and eye diseases. Currently, one of CARGENE BIOPHARMA’s key pipelines is CG-LR1, a novel liver regeneration-targeted nucleic acid drug that offers therapeutic hope to patients with chronic liver disease, particularly those with advanced cirrhosis, by enhancing and protecting liver function.

Liver cirrhosis is caused by factors such as alcohol, viruses, or non-alcoholic fatty liver disease. Long-term and repeated damage to liver cells leads to liver fibrosis, eventually resulting in cirrhosis. Liver failure caused by cirrhosis results in more than 2 million deaths globally each year, making it one of the leading causes of death worldwide. Nearly half of these deaths occur in China. Although a small number of patients can temporarily extend their lives through liver transplantation, the majority of patients are unable to receive a transplant. Approximately 14% of patients on liver transplant waiting lists die each year while waiting. There are over 100 million patients with cirrhosis worldwide, yet there are still no approved drugs that can address the symptoms and poor prognosis of cirrhosis. Patients are in urgent need of breakthrough new therapies.

CARGENE BIOPHARMA will also present at the conference on how to use innovative liver regeneration targets to make irreversible cirrhosis reversible. The two experts presenting this report have both been working in the field of regenerative biology for many years.

Dr. Torsten Wuestefeld is the co-founder and scientific advisor of CARGENE BIOPHARMA. He is also the principal investigator at the Genome Institute of Singapore (A*STAR), a guest researcher at the National Cancer Center Singapore, an adjunct professor at the School of Biological Sciences, Nanyang Technological University, and a member of the steering committee for the Novo Nordisk, GIS, and National University of Singapore joint laboratory. As an expert in liver research and diseases, he has published over 120 papers and holds multiple invention patents. With decades of experience in translating research findings into practical applications and industry collaborations, Dr. Wuestefeld developed a target discovery platform that identified an MKK4 kinase inhibitor, which has been licensed by HepaRegeniX (Germany) and showed positive results in Phase 1 clinical trials.

Dr. Tan Si Hui is the Senior Director of Biology at CARGENE BIOPHARMA and a member of the founding team. She graduated with a bachelor's degree from the University of Wisconsin, USA, and earned her Ph.D. from Stanford University, USA. Dr. Tan has over a decade of experience in cancer biology and regenerative biology, with publications in the American journals *Nature* and *Science*. She has also been awarded the highest honor for young scholars in Singapore—the President’s Young Scientist Award.

The specific schedule for the oral report is as follows:

Reporting Session: Reporter

Oral Report Section 4

Wednesday, September 13, from 9:00 AM to 12:00 PM

Dr. Torsten Wuestefeld

Agency for Science, Technology and Research, Genome Institute of Singapore

Oral Report Section 7

Thursday, September 14th, from 2 PM to 5 PM

Dr. Si Hui Chen

CARGENE BIOPHARMA

2023 Cold Spring Harbor Asia Liver Conference (Suzhou) "Liver Development, Metabolism, Disease and Cancer" will be held from September 11 to September 15, 2023. The conference topics cover many of the latest discoveries, with main themes including: 1. Liver biology and cell lineage tracing, 2. Hepatogenic cells and iPS cells, 3. Liver metabolism and pathways, 4. Alcoholic fatty liver disease, 5. Non-alcoholic fatty liver disease and NASH, 6. New technologies in liver research, 7. Mechanisms of hepatocarcinogenesis, 8. Immunotherapy for liver cancer, 9. Clinical translational research in liver diseases.

Meeting link: https://csh-asia.org/?content/1372