On November 19, 2024, Ractigen Therapeutics announced that its self-developed FUS gene-targeting small interfering RNA (siRNA) therapy, RAG-21, has successfully received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of Amyotrophic Lateral Sclerosis (ALS).The medical full name of ALS is Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease. Its core mechanism lies in the irreversible degeneration of motor neurons, leading patients to progress from mild muscle weakness to complete paralysis, and eventually respiratory failure.Each year, approximately 50,000 to 100,000 people worldwide are diagnosed with ALS, and the majority of patients have a life expectancy of only 2 to 5 years.The causes of ALS are complex and diverse. Currently identified pathogenic factors include gene mutations, protein metabolism disorders, mitochondrial dysfunction, etc. However, it is precisely these complex and diverse pathogenic mechanisms that make the treatment of the disease particularly difficult.Currently, there are only two drugs approved globally for the treatment of ALS: Riluzole and Edaravone. While these two drugs can slow disease progression, their effects are limited and they cannot reverse neuronal damage.RAG-21 is a siRNA therapy specifically targeting FUS gene mutations, effectively reducing the level of FUS protein and addressing the root cause of motor neuron degeneration. The drug functions through the RNA interference (RNAi) mechanism, alleviating the mislocalization and abnormal aggregation of FUS protein by lowering its levels.Preclinical studies show that RAG-21 has a significant knockdown effect and good safety. As a novel RNA therapy, RAG-21 brings new hope for improving the prognosis of FUS-ALS patients, who currently have no disease-modifying treatments available. This is the company's second FDA Orphan Drug Designation in the ALS field, following the previous designation of RAG-17 for SOD1-ALS, demonstrating the company’s firm commitment to conquering ALS.Orphan Drug Designation Granted by FDA Aims to Encourage Development of Drugs and Biologics for Rare Diseases (Diseases affecting fewer than 200,000 people in the United States). Companies receiving Orphan Drug Designation are entitled to several incentives, including seven years of market exclusivity from the date of marketing approval and a waiver of the new drug application fee.In April this year, Mr. Cai Lei and Ractigen Therapeutics established a joint laboratory for ALS drug development. Both parties will further integrate advantageous resources in the field of ALS research and treatment, share scientific research achievements, and accelerate the research and clinical application of ALS drugs.Dr. Longcheng Li, founder and CEO of Ractigen, stated: “RAG-21’s receipt of FDA Orphan Drug Designation highlights the urgent need to develop innovative therapies for ALS, particularly for patients with FUS gene mutations. FUS-ALS is one of the most severe and rapidly progressing subtypes of ALS, and currently, there are no effective treatment options available. We are committed to developing innovative therapies like RAG-21 to provide meaningful treatment options for patients with ALS and other life-threatening rare diseases.”
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