#China #ALS Research #Ractigen Therapeutics #Nantong, Suzhou #Dr. Long-Cheng Li

Ractigen Therapeutics' Founder Receives Prestigious Award

Ractigen Therapeutics recently announced that its founder and CEO, Dr. Long-Cheng Li, has been awarded the esteemed 2024 Life Science Ice Breaking Award. This accolade, established by ALS advocate Mr. Cai Lei and his wife, Ms. Duan Rui, aims to recognize remarkable advancements in ALS research that significantly benefit patients. Dr. Li’s pioneering work in RNA therapeutics has had a profound impact on the development of treatments for this challenging disease, emphasizing the critical role of scientific innovation in addressing pressing healthcare needs.

ALS, or Amyotrophic Lateral Sclerosis, is a severe neurodegenerative condition characterized by the progressive degeneration of motor neurons. This ultimately leads to muscle weakness, and in advanced stages, respiratory failure. Despite significant investments in research over decades, effective therapies remain elusive, underscoring the importance of Ractigen's breakthroughs in this space.

A highlight of Ractigen's efforts is the innovative siRNA therapy, RAG-17, specifically targeting the SOD1 gene—known to play a crucial role in some subtypes of ALS. RAG-17 employs the proprietary SCAD™ delivery platform developed by Ractigen, enhancing the efficient delivery of the therapeutic agent to the central nervous system. Early preclinical trials indicate that RAG-17 effectively reduces toxic SOD1 protein levels, thereby extending survival while preserving motor function in animal models. Currently, RAG-17 is undergoing Phase I clinical trials, reassuring ALS patients about the potential for viable treatment options in the near future.

In addition to RAG-17, Ractigen has developed RAG-21, targeting mutations in the FUS gene associated with more aggressive forms of ALS. The U.S. FDA has granted Orphan Drug Designation to RAG-21, highlighting its potential to fulfill the critical needs of patients facing this aggressive variant of the disease.

Dr. Li's contributions extend well beyond ALS. Through his groundbreaking discovery of RNA Activation (RNAa), he has opened new pathways for gene expression regulation and has garnered international recognition for his contributions to RNA science. Under his leadership, Ractigen has built a robust pipeline of RNA-based therapies that are poised to tackle various unmet medical needs, including neuromuscular disorders, metabolic diseases, and cancers.

The Life Science Ice Breaking Award, which comes with a prize of RMB 5 million, symbolizes the commitment and aspirations of the ALS community and advocates for real progress in treatment solutions. By focusing on tangible patient impact, it champions efforts to turn scientific accomplishments into therapies that can transform lives.

In Dr. Li's words, "This award is a profound honor and a testament to the commitment of our team at Ractigen. It motivates us to continue our mission of developing transformative therapies that address the root causes of diseases like ALS. Together, we remain committed to advancing science and delivering meaningful solutions to patients around the world."

Ractigen Therapeutics continues to push forward with its mission, enhancing the development of RAG-17 and RAG-21, while also seeking new, innovative treatments for rare diseases. Through its resolute commitment to RNA-based drug development, Ractigen aims to bring brighter prospects to patients grappling with unmet medical needs.

About the Award

The Life Science Ice Breaking Award, began by ALS patient leader Mr. Cai Lei, is aimed at celebrating endeavors in ALS research, particularly those that directly enhance patient lives in China and beyond, including Hong Kong, Macau, and Taiwan. As a first-of-its-kind recognition initiated by a patient community, the award underlines the societal imperative to speed up innovation in the realm of ALS treatment, fostering a climate of collaboration and forward-thinking.

About Ractigen Therapeutics

Ractigen Therapeutics is a leading company in the small activating RNA (saRNA) domain, harnessing RNA activation to facilitate the up-regulation of gene expression. Its innovative approach allows targeted therapies to restore normal protein functions. As a pioneering company globally, it is making significant strides in clinical development for both small activating RNA (saRNA) and small interfering RNA (siRNA) treatments. Ractigen also specializes in advanced delivery systems, both liver-targeted and extra-hepatic, providing new treatment options for diseases that have been traditionally difficult to address.

For more detailed insights into their work, please visit www.ractigen.com.