Ractigen Therapeutics Wins 2024 Life Science Ice Breaking Award for Breakthrough 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, along with his wife, Ms. Duan Rui, to honor scientists and teams who have achieved 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, showcasing the company's dedication and determination to continuously advance global ALS treatment.

Dr. Li Longcheng stated:"I am deeply honored and gratified to receive this prestigious award. This not only affirms our team's past efforts but also inspires 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 addressing 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 directly into accelerating the advancement of our ALS research pipeline, hoping to contribute our modest efforts toward breakthroughs in this field."

Amyotrophic lateral sclerosis (ALS), as a neurodegenerative disease that poses a severe 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 care for patients and a strong sense of social responsibility, has swiftly taken action in the field of ALS treatment. Leveraging its independently 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 milestone breakthroughs that have brought new hope to patients.

Among them,RAG-17 is an innovative double-stranded small interfering RNA (siRNA) drug designed to treat 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 an investigator-initiated trial (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 isFUSGene mutation-type ALS patients have 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 downFUSGene 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.

Ractigen Therapeutics is a platform-based new drug research and development company based in China and targeting the global market, committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is the world's only small nucleic acid drug company that simultaneously develops small activating RNA and small interfering RNA drugs and has advanced both categories into clinical trials. It is also one of the few companies globally that possesses liver-targeted and extrahepatic small nucleic acid drug delivery technologies, having developed multiple internationally leading proprietary small nucleic acid drug delivery platform technologies with independent intellectual property rights, such as SCAD™ and LiCO™.

Based on RNA activation technology and the independently developed Smart-TTC saRNA drug development platform, Ractigen Therapeutics has established a highly differentiated small nucleic acid drug pipeline. The indications cover neurodegenerative diseases, neuromuscular diseases, tumors, metabolic and hematological diseases, etc., providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas. To date, Ractigen 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.

As the discoverer and pioneer of the RNA activation (RNAa) phenomenon, Dr. Li Longcheng, founder and CEO of Ractigen, has achieved remarkable success in scientific research and medical innovation. A former clinical doctor, he served as an associate professor and independent PI at the University of California, San Francisco, as well as a distinguished professor and director of the central laboratory at Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, accumulating extensive research experience and profound academic expertise. 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.

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 within the territory of the People's Republic of China (including Hong Kong, Macao, and Taiwan regions). The award seeks to promote 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 crucial evaluation criterion 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 Conquer ALS Charitable Trust, uniting care and strength from all sectors of society to inject lasting momentum into solving this medical puzzle.

1. Ractigen Official WeChat Account