"FDA Grants Orphan Drug Designation to RAG-21 for Treatment of ALS
[...]The FDA granted an orphan drug designation to RAG-21 (Ractigen Therapeutics) for the treatment of amyotrophic lateral sclerosis (ALS). Preclinical data showcases RAG-21's potential to mitigate motor neuron degeneration and improve disease outcomes for patients."
"RAG-21 is an siRNA therapy specifically designed to target FUS-ALS, one of the most aggressive subtype of ALS. By leveraging RNA interference (RNAi), RAG-21 safely and effectively reduces FUS protein levels, addressing the root cause of motor neuron degeneration in FUS-ALS. Preclinical studies have demonstrated its potent efficacy and safety, mitigating FUS cytoplasmic mis-localization and aggregation. As a novel RNA-based therapy, RAG-21 offers renewed hope for improving outcomes in patients suffering from this challenging form of ALS, which currently lacks effective treatment options. This designation marks our second FDA ODD for treating ALS, following RAG-17 for SOD1-ALS, reflecting our commitment to conquering ALS."
"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’s cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation."
"Ractigen’s operations are strategically located across various sites in China: Our main headquarters, situated in the bioBAY area of Suzhou, spans 2200 square meters and is outfitted with state-of-the-art equipment and facilities, including certified BSL-2 biology laboratories and a certified SPF animal facility. In Rudong, Jiangsu, China, we operate a oligonucleotide manufacturing facility housed in a four-story building within the modern Industry Park of Life and Health Science. Additionally, we maintain a business development office in the Zhangjiang High Technology Park in Shanghai."
[...]The FDA granted an orphan drug designation to RAG-21 (Ractigen Therapeutics) for the treatment of amyotrophic lateral sclerosis (ALS). Preclinical data showcases RAG-21's potential to mitigate motor neuron degeneration and improve disease outcomes for patients."
"RAG-21 is an siRNA therapy specifically designed to target FUS-ALS, one of the most aggressive subtype of ALS. By leveraging RNA interference (RNAi), RAG-21 safely and effectively reduces FUS protein levels, addressing the root cause of motor neuron degeneration in FUS-ALS. Preclinical studies have demonstrated its potent efficacy and safety, mitigating FUS cytoplasmic mis-localization and aggregation. As a novel RNA-based therapy, RAG-21 offers renewed hope for improving outcomes in patients suffering from this challenging form of ALS, which currently lacks effective treatment options. This designation marks our second FDA ODD for treating ALS, following RAG-17 for SOD1-ALS, reflecting our commitment to conquering ALS."
"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’s cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation."
"Ractigen’s operations are strategically located across various sites in China: Our main headquarters, situated in the bioBAY area of Suzhou, spans 2200 square meters and is outfitted with state-of-the-art equipment and facilities, including certified BSL-2 biology laboratories and a certified SPF animal facility. In Rudong, Jiangsu, China, we operate a oligonucleotide manufacturing facility housed in a four-story building within the modern Industry Park of Life and Health Science. Additionally, we maintain a business development office in the Zhangjiang High Technology Park in Shanghai."
