Ractigen Therapeutics Receives FDA Orphan Drug Designation for the Novel Oligonucleotide Conjugate RAG-17
Friday, March 03, 2023
Ractigen Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RAG-17, a novel siRNA modality for the treatment of Amyotrophic Lateral Sclerosis (ALS).
The approval of RAG-17 to ALS patients can show significantly higher efficacy in patients with the SOD1 mutation, compared to the other modalities.
RAG-17 is a therapeutic siRNA designed to target and knockdown the expression of SOD1 in patients with pathogenic mutations known to cause ALS. The RAG-17 chemistry is based on Ractigen’s proprietary Smart Chemistry-Aided Delivery (SCAD) delivery platform, in which the siRNA is conjugated to an accessory oligonucleotide (ACO), enabling durable and potent activity in CNS tissues. Based on several preclinical studies, RAG-17 has a significantly higher potency on ALS disease models (e.g., hSOD1G93A mouse model) than benchmark compounds.
ALS is a severely disabling neurodegenerative disease without a curative treatment. Unfortunately, life expectancy of people diagnosed with ALS remains poor and the most patients die from respiratory failure within 3-5 years following diagnosis. Early symptoms typically include muscle cramps, twitching, weakness, and stiffness. Patients inevitably begin to experience problems with movement and speech, which eventually manifests into assisted breathing, paralysis, and inevitable death.
