NANTONG and SUZHOU, China, Dec. 30, 2024 — Ractigen Therapeutics proudly announces that its Founder and CEO, Dr. Long-Cheng Li, has been honored with the 2024 Life Science Ice Breaking Award. This award, established by Mr. Cai Lei, a prominent ALS patient and advocate, along with his wife Ms. Duan Rui, recognizes groundbreaking contributions to ALS research and treatment that delivers tangible benefits to patients. The recognition underscores Dr. Li’s pioneering work in RNA-based therapeutics and Ractigen’s significant impact on advancing ALS drug development.
ALS (Amyotrophic Lateral Sclerosis) is a devastating neurodegenerative disease with no effective cure. It severely affects motor neurons, leading to progressive muscle weakness and eventual respiratory failure. Despite decades of research, ALS remains one of the most challenging areas in healthcare. Ractigen is committed to addressing this challenge by leveraging advanced RNA science to develop therapies targeting the genetic roots of ALS.
A cornerstone of Ractigen’s efforts is RAG-17, a siRNA therapy specifically designed to target the SOD1 gene, a key driver in certain ALS subtypes. By leveraging Ractigen’s proprietary SCAD™ delivery platform, RAG-17 achieves efficient and durable delivery to the central nervous system, where it reduces toxic SOD1 protein levels, slows disease progression, and preserves motor neuron function. Preclinical studies have demonstrated significant efficacy in extending survival and improving motor performance in animal models. Now undergoing Phase I clinical trials, RAG-17 marks a promising step forward in the development of effective ALS therapies.
Complementing this is RAG-21, a novel therapy targeting FUS gene mutations linked to a more aggressive form of ALS. Recognized with Orphan Drug Designation by the U.S. FDA, RAG-21 signifies a critical advancement in meeting the unmet needs of ALS patients by addressing the underlying causes of their condition.
Dr. Li's contributions to RNA therapeutics extend significantly beyond ALS. As the discoverer of RNA Activation (RNAa), he has pioneered a new frontier in gene expression regulation, earning international acclaim for his research and scientific innovations. His leadership has propelled Ractigen to develop a diverse pipeline of RNA-based therapies, employing innovative delivery platforms and targeting diseases with unmet medical needs, including neurodegenerative and neuromuscular disorders, metabolic and ocular diseases, and cancers.
The Life Science Ice Breaking Award, with a prize of RMB 5 million, reflects the collective hope and dedication of patient communities and advocates to driving meaningful change in ALS research and treatment. By prioritizing real-world patient impact, the award emphasizes the importance of translating scientific innovation into life-changing therapies.
“This award is a profound honor and a testament to the commitment of our team at Ractigen,” said Dr. Li. “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 is dedicated to advancing its pipeline, including RAG-17 and RAG-21, while exploring innovative treatments for rare diseases. Through its groundbreaking work in RNA-based drug development, the company strives to bring hope and healing to patients facing unmet medical needs.
About Life Science Ice Breaking Award
The Life Science Ice Breaking Award, established by prominent ALS patient and advocate Mr. Cai Lei and his wife Ms. Duan Rui, is dedicated to recognizing outstanding contributions to ALS research and patient care within China, including Hong Kong, Macau, and Taiwan. As the first life science award initiated by a patient organization, it emphasizes tangible impact on patient lives, encouraging therapies and innovations that address critical medical needs. By fostering collaboration and innovation, the award aims to inspire breakthroughs in ALS treatment and galvanize efforts toward overcoming this devastating disease.
About Ractigen Therapeutics
A leader in small activating RNA (saRNA) drug development, Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing RNA activation (RNAa) mechanism to up-regulate endogenous gene expression. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen is the only company globally advancing both small activating RNA (saRNA) and small interfering RNA (siRNA) therapies into clinical development. Additionally, it is among the few worldwide with expertise in both liver-targeted and extra-hepatic oligonucleotide delivery systems. Ractigen's cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation.
For more information, please visit our website: www.ractigen.com.
