Mirimus' RNAi Technology Helps Advance New Treatment for Liver Tumors and Acute Chronic Liver Diseases

Leveraging Mirimus RNAi technology, a study led by Tuebingen University shows that a new drug has the potential to revolutionize liver surgery and liver transplantation.

NEW YORK, March 18, 2024 /PRNewswire-PRWeb/ -- Mirimus, a pioneer in RNAi therapeutic design and CRISPR/Cas9 gene editing technology, proudly announces its role in advancing the development of HRX-215, a novel MKK4 inhibitor that showcases promising results in liver regeneration. This advancement, detailed in CELL journal through collaboration with Prof. Dr. Lars Zender and HepaRegeniX, signifies a hopeful future for liver tumor surgeries and effectively addresses acute and chronic liver disease treatments.

Mirimus' RNAi technology helped propel a significant breakthrough in the treatment of liver tumors and diseases. The development of the MKK4 inhibitor, HRX-215, which followed the mechanistic studies of MKK4 inhibition utilizing Mirimus' RNAi technology, is detailed in a comprehensive study published in CELL journal on March 14, 2024. The study, led by Prof. Dr. Lars Zender, Medical Director of the Department of Medical Oncology and Pneumology at Tuebingen University Hospital, in partnership with startup HepaRegeniX, demonstrates the potential of HRX-215 to increase liver cell regeneration, opening new avenues for previously infeasible liver surgeries and transplants.

The preclinical and phase I study results illustrate HRX-215's capacity to allow complete removal of advanced liver tumors without compromising liver functionality, a feat not possible before. Additionally, the drug shows promise in expanding the availability of life-saving liver transplants. A phase I trial involving 48 healthy volunteers confirmed the drug's safety and tolerability, marking a significant step forward in liver disease treatment.

Mirimus' innovative RNAi technology played a critical role in these developments, providing essential preclinical animal models for the drug's discovery and development process. "The RNAi technology from Mirimus helped to mitigate the risk and increase confidence to undertake a comprehensive drug discovery and development approach. Safety studies in RNAi animal models demonstrated that long term systemic suppression of MKK4 produced no side effects, and therefore boosted our confidence in pursuing a chemical approach to MKK4 inhibition," remarked Dr. Zender.

Dr. Prem Premsrirut, Founder and CEO of Mirimus, emphasized the collaboration's impact, stating, "We are extremely proud of our collaborative efforts that helped drive this first-in-human phase I trial in healthy volunteers. This is a true bench-to-bedside story that will continue through further clinical trials, with the long-term potential to help thousands of patients."

About Mirimus

Mirimus, an award-winning biotech company in Brooklyn, New York, was originally founded by Drs. Prem Premsrirut, Scott Lowe, Gregory Hannon, Stephen Elledge and Christof Fellmann in 2010 with technology developed at Cold Spring Harbor Laboratory that enabled scalable and cost-effective pipelines to generate RNAi mouse and rat models for evaluation of novel therapeutic targets in vivo. Over the years, Mirimus has continued to enhance its RNAi technologies and advance it beyond powerful research tools towards new therapeutic applications. Through strategic partnerships, Mirimus has developed first-in-class RNAi biologics that couple unique delivery modalities to its RNAi cargo. Owing to its new Optimus reporter model, Mirimus is able to readily assess the bioavailability, cell-specific delivery, cellular entry, endosomal escape, on-target efficiency and potential toxicities in vivo, to rapidly propel the development of novel RNAi therapeutic strategies. These innovations advance the development of novel RNAi therapeutics and provide critical tools for cutting-edge disease research for leading drug companies and academic institutions, reflecting the company's commitment to improving human health in a global scale.

Publication

First-in-class MKK4 inhibitors enhance liver regeneration and prevent liver failure

DOI: https://doi.org/10.1016/j.cell.2024.02.023

Media Contact

Sean Seibel, Mirimus, 1 (833) 647-4687, [email protected], www.mirimus.com

SOURCE Mirimus