Study aims to evaluate the safety and feasibility of RAG-17 in human patients and clinically validate Ractigen’s proprietary CNS delivery platform enabling new oligonucleotides therapeutics in the CNS
JIANGSU, CHINA (June 5, 2023) – Ractigen Therapeutics, a biopharmaceutical company focused on the discovery, development, and commercialization of novel oligonucleotide therapeutics, today announced the dosing of its first patient in the Company’s First in Human (FIH) clinical trial for ALS patients with SOD1 mutation. RAG-17 is a siRNA drug designed to provide potent and durable knockdown of SOD1 protein in the central nervous system (CNS) of ALS patients to prevent motor neuron degradation and delay disease progression. While delivery to tissue has been historically difficult for siRNAs, RAG-17 employs a novel conjugate referred to as an Smart Chemistry-Aided Delivery (SCAD) that enables broad biodistribution and tissue uptake throughout the CNS.
The FIH trial is designed to evaluate the safety, pharmacokinetics, pharmacodynamics (reduction of CSF biomarkers) and immunogenicity of RAG-17 in ALS patients with a confirmed SOD-1 mutation. The study is being conducted at Beijing Tiantan Hospital.
“We are extremely pleased to initiate this first in human trial of RAG-17, marking our transformation into a clinical-stage company” said Dr. Long-Cheng Li, Ractigen’s Founder, President and Chief Executive Officer. “This milestone highlights our commitment to building and strengthening our pipeline and clinical validation of our novel CNS delivery platform. Given our mission to deliver innovative medicines to patients with high, unmet needs, we look forward to continued enrollment and dosing of patients in this trial” added Dr. Li.
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 death. Over 50 genes have been linked to ALS in which about 20% of all genetically defined cases are associated with mutation to the SOD1 gene.
RAG-17 is a therapeutic siRNA designed to knockdown the expression of SOD1 in patients with pathogenic mutations known to cause ALS. RAG-17 utilizes 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 following intrathecal (IT) injection. Based on several preclinical studies, RAG-17 has a significantly higher potency on ALS disease models (e.g., hSOD1G93A mouse model) than benchmark compounds.
Ractigen Therapeutics was founded in 2017 by pioneers of the RNA activation (RNAa) technology including Dr. Long-Cheng Li and his longstanding colleagues Dr. Robert Place and Dr. Moorim Kang. Ractigen is a late-stage preclinical pharmaceutical company devoted to creating groundbreaking therapies based on oligonucleotide technologies including RNAa and RNAi. Supporting therapeutic development, Ractigen Therapeutics has also created a portfolio of delivery platforms including its SCAD system, which allows enhanced biodistribution throughout CNS and empowers a pipeline of first-in-class candidate medicines for patients with unmet genetic disorders. The Company recently completed +$50M Series A/A+ venture financing rounds with participation from reputed investors including SDIC Venture Capital, Hillhouse Venture Capital, Eisai Co., LC Ventures, CSSD Capital, Xianghe Capital, CCB Healthcare Growth Fund, Boyi Capital, and Longman Capital.