Clinical-stage pharmaceutical company Ractigen Therapeutics announced on Tuesday that the first patient has been dosed in a Phase I clinical trial of RAG-17, a new siRNA therapy targeting Amyotrophic Lateral Sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) gene mutations.

Conducted in China, the randomised, double-blind, placebo-controlled trial will assess the safety/tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of RAG-17 in subjects with SOD-ALS.

The US Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to the product in March 2023, followed by approval of the Investigational New Drug (IND) application.

Dr. Long-Cheng Li, founder and CEO of Ractigen Therapeutics, said: "The first patient dosed in the RAG-17 trial marks a pivotal milestone in our mission to combat ALS, one of the most devastating neurodegenerative diseases. This achievement underscores our unwavering commitment to advancing RNA-based therapies that have the potential to transform the lives of patients and families affected by rare and severe conditions like ALS."