Ractigen Therapeutics Founder Dr. Long-Cheng Li Awarded 2024 Life Science Ice Breaking Award for Pioneering ALS Therapies

On December 30, 2024, Ractigen Therapeutics once again drew global attention for its significant breakthrough in the treatment of ALS (Amyotrophic Lateral Sclerosis), and Dr. Li Longcheng's team was honored.2024 Annual Life Sciences Breakthrough Award. This award was established by Mr. Cai Lei, an ALS patient and fighter, and his wife, Ms. Duan Rui, to honor scientists and teams who have made significant breakthroughs in ALS treatment and research and brought tangible changes to patients. This recognition highly affirms Ractigen Therapeutics' efforts in ALS drug development and technological innovation and highlights the company's commitment and determination to continuously advance ALS treatment globally.

Driving Innovative Breakthroughs in ALS Treatment

Amyotrophic lateral sclerosis (ALS), as a neurodegenerative disease that poses a serious threat to human life and health, has long been a medical challenge due to the lack of effective treatments, imposing a heavy burden on patients and their families. With the strong support of Mr. Cai Lei and his team, Ractigen Therapeutics, driven by deep compassion for patients and a strong sense of social responsibility, has swiftly taken action in the field of ALS treatment. Leveraging its self-developed small nucleic acid drug research and development system and combining it with the globally leading SCAD™ central nervous system delivery technology, Ractigen Therapeutics has rapidly initiated and advanced its R&D pipeline in the ALS field, achieving several breakthrough milestone accomplishments that have brought new hope to patients.

Among them, RAG-17 is an innovative double-stranded small interfering RNA (siRNA) drug used for treating carriersSOD1Amyotrophic Lateral Sclerosis with Gene Mutation. Preclinical studies show that the drug works by inhibitingSOD1Gene expression reduces the generation of toxic proteins, protects neuronal function, and can slow disease progression while significantly improving motor ability. In investigator-initiated trials (IIT), RAG-17 demonstrated good safety and tolerability, as well as encouraging clinical efficacy. Currently, the drug has entered Phase I clinical trials and successfully completed the first patient dosing.

At the same time, RAG-21 isFUSDeveloped for ALS patients with gene mutations, it has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). Through the RNA interference (RNAi) mechanism, RAG-21 can precisely knock down.FUSGene expression significantly reduces toxicityFUSProtein production. Preclinical study data show that the drug has the potential to alleviate motor neuron degeneration and improve disease prognosis, providingFUS-ALS, this rapidly progressive motor neuron disease, brings new hope for treatment.

Outstanding Technological Innovation and Academic Contributions

As the discoverer and pioneer of the RNA activation (RNAa) phenomenon, Dr. Li Longcheng has achieved remarkable success in scientific research and medical innovation. Having originally worked as a clinician, he served as an associate professor and independent PI at the University of California, San Francisco, as well as a distinguished professor at Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and director of the Central Laboratory, accumulating extensive research experience and profound academic knowledge. In 2006, Dr. Li Longcheng first discovered and named the "RNA activation" phenomenon, opening up an entirely new field of study. To date, he has published over 80 papers, which have been cited more than 13,800 times, and has been listed on the "Highly Cited Chinese Researchers" list for three consecutive years, holding six U.S. patents.

In 2017, Dr. Li Longcheng founded Ractigen Therapeutics, dedicated to developing breakthrough small nucleic acid drugs and innovative therapies. With its self-developed next-generation RNA activation platform and internationally leading small nucleic acid drug delivery technology, the company has built a highly differentiated R&D pipeline. Ractigen has made significant progress in the field of rare disease drug development, providing unique treatment options for patients worldwide. To date, Ractigen Therapeutics has received three orphan drug designations and one rare pediatric disease designation from the U.S. FDA, demonstrating the company’s commitment and care for patients with rare diseases.

This time, winning the Life Science Breakthrough Award is not only a high recognition of Dr. Li Longcheng's personal contributions but also an acknowledgment of the achievements made by the Ractigen team in the field of ALS treatment. The award comes with a prize of 5 million RMB, fully demonstrating the attention and support from Mr. Cai Lei, Ms. Duan Rui, and all sectors of society towards ALS treatment research.

Dr. Li Longcheng stated:"I am deeply honored and gratified to receive this prestigious award. This is not only an affirmation of our team's past efforts but also an encouragement for us to continue creating new treatment possibilities for ALS and other rare disease patients in the future. Ractigen will remain committed to technological innovation and patient needs, striving to bring more tangible changes to patients worldwide." He further emphasized: "We have decided to invest the full RMB 5 million prize into accelerating the advancement of our ALS R&D pipeline, in hopes of contributing our modest efforts to breakthroughs in this field."

In the future, Ractigen will continue to push technological boundaries, accelerate the development of RAG-17 and RAG-21, and expand its innovative drug research and development into more disease areas, bringing more hope and benefits to patients.

Life Science Ice Breaking AwardThe Life Science Ice Breaking Award was established by Mr. Cai Lei, an ALS fighter, and his wife, Ms. Duan Rui. It aims to encourage and honor scientists, biopharmaceutical companies, technology enterprises, and doctors who have made outstanding contributions to ALS research and life-saving efforts in China (including Hong Kong, Macao, and Taiwan). The award seeks to advance scientific research and development in the ALS field and gather strength to overcome this medical challenge. As the first life science award issued by a patient organization, its most important criterion for evaluation is the actual contribution to patients—whether it truly achieves life-saving results for them. The total prize money for 2024 is 5 million RMB, jointly supported by Breaking Ice Future Company and the Charity Trust for Conquering ALS, uniting care and strength from all sectors of society to inject lasting momentum into solving this medical challenge.

Ractigen Therapeutics Founder Dr. Long-Cheng Li Awarded 2024 Life Science Ice Breaking Award

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.