Ractigen Therapeutics Introduces Novel Drug for ALS Treatment
Ractigen Therapeutics has unveiled RAG-17, an innovative siRNA modality designed to address Amyotrophic Lateral Sclerosis (ALS) treatment.
Recognizing its potential for treating a rare disease, the U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-17.
The FDA grants orphan drug status to drugs or biologics primarily intended for the treatment of diseases or conditions that impact less than 200,000 individuals in the United States.
ALS is a severe and incapacitating neurodegenerative disease that currently lacks a curative treatment. Unfortunately, individuals diagnosed with ALS face a bleak outlook, as the majority of patients experience respiratory failure and pass away within 3-5 years following their diagnosis.
Initial indications of ALS usually involve muscle cramps, twitching, weakness, and stiffness. Gradually, patients encounter challenges with motor skills and speech, leading to the need for assistance with breathing, paralysis, and ultimately, loss of life.
RAG-17 is a therapeutic siRNA specifically designed to target and reduce the expression of SOD1 in individuals affected by ALS-causing pathogenic mutations.
It utilizes Ractigen's proprietary Smart Chemistry-Aided Delivery (SCAD) platform, where the siRNA is linked to an accessory oligonucleotide (ACO). This unique formulation enables long-lasting and potent effects within the central nervous system (CNS) tissues.
Multiple preclinical studies have demonstrated that RAG-17 exhibits significantly greater potency against ALS disease models, such as the hSOD1G93A mouse model when compared to established compounds used as benchmarks.
