Ractigen Therapeutics has received orphan drug designation from the US Food and Drug Administration (FDA) for its new therapeutic siRNA, RAG-17, to treat Amyotrophic Lateral Sclerosis (ALS).
RAG-17 has been designed to act on and knockdown SOD1 expression in patients with pathogenic mutations that are known to cause ALS.
The chemistry of the therapeutic is based on the company’s Smart Chemistry-Aided Delivery (SCAD) delivery platform, in which it is conjugated to an accessory oligonucleotide (ACO), thereby enabling prolonged and potent activity in CNS tissues.
RAG-17 is said to have a significantly higher potency on ALS disease models compared to benchmark compounds based on several preclinical studies.
Ractigen Therapeutics founder, president and CEO Dr Long-Cheng Li said: “The FDA’s decision to grant orphan drug designation is an important and valuable milestone for RAG-17, and highlights the significant unmet medical need for people living with this severe disease.
“We are eager to bring RAG-17 to ALS patients as soon as possible, as we believe this therapy can have significantly higher efficacy in patients with the SOD1 mutation, compared to the other modalities.”
The severely disabling neurodegenerative disease ALS has no curative treatment and life expectancy of people diagnosed with ALS remains poor.
The US FDA’s orphan drug designation provides companies with development incentives, including a marketing exclusivity for seven years from the date of market approval and a waiver of the New Drug Application fee.
Last January, Ractigen closed a Series A+ financing round totalling $30m led by SDIC Venture Capital with participation from Eisai Co, LC Ventures, CSSD Capital, and Xianghe Capital to advance serval programmes from preclinical to clinical phase.