NEW YORK – The first patient has been dosed in Ractigen Therapeutics' first-in-human trial for a drug targeted toward a genetic form of amyotrophic lateral sclerosis (ALS), the biopharmaceutical company said Monday.
The study is testing RAG-17 as a treatment for SOD1-ALS, in which the neurodegenerative disease is caused by mutations in the SOD1 gene. While overall rare, SOD1-ALS is one of the most common genetic forms of ALS, accounting for about 2 percent of ALS cases globally.
RAG-17 is a small interfering RNA (siRNA) drug that's designed to target and knock down expression of the SOD1 protein in the central nervous system of ALS patients in an effort to prevent motor neuron degradation and delay disease progression. The siRNA drug is conjugated to an accessory oligonucleotide and injected intrathecally.
For the trial, which is being conducted at Beijing Tiantan Hospital in China, investigators will test RAG-17 in ALS patients with a confirmed SOD1 mutation.
"We are extremely pleased to initiate this first-in-human trial of RAG-17, marking our transformation into a clinical-stage company" said Long-Cheng Li, founder, president, and CEO of Ractigen, which is based in Jiangsu, China.