Clinical Trials
3
1 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
2016
Not yet recruiting
1
33.3%
Recruiting
2
66.7%
No approval data available
- Ractigen Therapeutics received U.S. FDA IND clearance for RAG-1C, a first-in-class small activating RNA (saRNA) therapy for proliferative vitreoretinopathy (PVR), following prior clearance from China's CDE in March 2025. - RAG-1C utilizes the proprietary LiCO™ platform to activate the p21 gene, inducing cell cycle arrest to inhibit cellular proliferation and myofibroblast transformation without cellular toxicity. - PVR affects 5–10% of all rhegmatogenous retinal detachment cases and up to 40% of severe ocular trauma cases, with no FDA-approved pharmacological therapies currently available. - The upcoming Phase I trial will evaluate safety, tolerability, pharmacokinetics, and preliminary efficacy in patients undergoing retinal detachment surgery at high risk of developing PVR.
- Ractigen Therapeutics closed over $31 million in new financing led by Guozhong Capital to accelerate its clinical-stage small activating RNA (saRNA) pipeline. - Proceeds will fund Phase 2 trials for RAG-01 in NMIBC and RAG-17 in ALS, and advance RAG-18 toward IND filing for Duchenne muscular dystrophy. - RAG-01 achieved a 67% complete response rate in Phase 1 for BCG-unresponsive NMIBC, while RAG-17 demonstrated ~60% CSF SOD1 reduction in ALS patients. - The company's proprietary SCAD™ and LiCO™ delivery platforms enable extrahepatic RNA delivery to CNS, muscle, heart, bladder, and eye tissues.
- Ractigen Therapeutics' first-in-class saRNA therapy LiCO-saUcp1 reduced fat mass by 45% in diet-induced obese mice while fully preserving lean muscle mass, unlike semaglutide which caused 19% lean mass loss. - Treated animals sustained a 46% fat mass reduction for two months after drug withdrawal, eliminating the rapid weight rebound typically seen with incretin therapies. - When combined with semaglutide, LiCO-saUcp1 synergistically achieved a 69% reduction in fat mass without worsening GLP-1-associated muscle depletion. - The therapy dose-dependently reduced liver triglycerides by up to 79%, demonstrating significant resolution of hepatic steatosis.
- Ractigen Therapeutics reported positive Phase I data for RAG-17, a novel siRNA therapeutic targeting SOD1-ALS, demonstrating an 81% reduction in plasma neurofilament light chain levels. - The single intrathecal dose showed favorable safety profile with no serious adverse events and achieved 58.1% reduction in cerebrospinal fluid SOD1 protein by Day 90. - Preliminary clinical data from the highest dose cohort showed functional stabilization with zero decline in ALSFRS-R scores at Day 90 in all assessed participants. - Phase II trials are now actively dosing participants following the encouraging biomarker and clinical stabilization signals from the Phase I study.
- Ractigen Therapeutics has dosed the first patient in an investigator-initiated trial of RAG-18, a small activating RNA therapeutic for Duchenne Muscular Dystrophy at Peking Union Medical College Hospital. - RAG-18 utilizes RNA activation technology to target the UTRN gene and upregulate utrophin protein as a functional replacement for missing dystrophin in DMD patients. - The therapy has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation in 2024, with preclinical data showing effective mitigation of muscle damage. - This first-of-its-kind saRNA approach could potentially treat all DMD patients regardless of specific genetic mutation location, addressing limitations of current exon-skipping therapies.
- Ractigen Therapeutics has dosed the first patient in a Phase I clinical trial of RAG-17, a siRNA therapy for ALS patients with SOD1 mutations. - The Phase I trial is a randomized, double-blind, placebo-controlled study evaluating safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy. - RAG-17 leverages Ractigen's SCAD platform to enhance delivery of siRNA into the central nervous system, targeting the SOD1 gene. - Preclinical and IIT data suggest RAG-17 is well-tolerated and improves motor function, supported by Orphan Drug Designation from the FDA.
- Ractigen Therapeutics' RAG-21, a novel siRNA therapy targeting the FUS gene, has received FDA Orphan Drug Designation for amyotrophic lateral sclerosis (ALS). - RAG-21 leverages RNA interference to reduce FUS protein levels, addressing motor neuron degeneration in FUS-ALS, a particularly aggressive ALS subtype. - Preclinical studies demonstrate RAG-21's efficacy and safety in mitigating FUS cytoplasmic mis-localization and aggregation, offering hope for improved outcomes. - The FDA's designation provides incentives, including tax credits and marketing exclusivity, to accelerate RAG-21's development for this rare and severe disease.
- The FDA has granted Orphan Drug Designation to Ractigen Therapeutics' RAG-21 for amyotrophic lateral sclerosis (ALS) treatment, specifically targeting the FUS subtype. - RAG-21 is a small interfering RNA (siRNA) therapy designed to reduce toxic FUS protein production by targeting FUS mRNA transcripts in the central nervous system. - Preclinical data suggests RAG-21 can mitigate motor neuron degeneration and improve outcomes for patients with FUS-ALS, a severe and rapidly progressing form of ALS. - RAG-21 shares a similar administration method with Ractigen's RAG-17, which has shown promising early clinical data in SOD1-mutated ALS patients.
- Ractigen Therapeutics' RAG-18, a small activating RNA (saRNA) therapy, has been granted Orphan Drug Designation by the FDA for treating Duchenne and Becker muscular dystrophy. - RAG-18 previously received Rare Pediatric Disease Designation, making it the first saRNA therapy to achieve both designations, highlighting its potential for treating rare genetic conditions. - The therapy aims to increase utrophin production by targeting the UTRN gene, offering a functional substitute for the deficient dystrophin protein in DMD and BMD patients. - Preclinical data presented at OTS 2023 demonstrated RAG-18's ability to induce utrophin expression and ameliorate muscle damage in a mouse model of DMD.
