Shares of Phio Pharmaceuticals Corp. soared 291% Jan. 13 on news that two patients with cutaneous squamous cell carcinoma had a complete response following treatment with the company’s Intasyl siRNA gene silencing candidate PH-762.
Canary Cure Therapeutics (Canary Global Inc.) has released preclinical results for its lead candidate, CCT-217, an siRNA therapeutic targeting cannabinoid CB1 receptor (CB1R) and zinc finger protein 423 (ZFP423) to treat obesity.
Hangzhou Sino-US Huadong Medicine Co. Ltd. and Synerk Pharmatech (Suzhou) Co. Ltd. have entered into a strategic collaboration in which the two parties will jointly develop the small nucleic acid (siRNA) drug SNK-2726.
CSPC Pharmaceutical Group Ltd. has gained clinical trial approval from China’s National Medical Products Administration (NMPA) for SYH-2062 injection, a double-stranded small interfering RNA (siRNA) drug to treat hypertension.
Wave Life Sciences Ltd. has submitted a clinical trial application (CTA) for WVE-007, an investigational GalNAc-conjugated small interfering RNA (siRNA) designed to silence inhibin βE (INHBE) gene expression, for the treatment of obesity.
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
Voyager Therapeutics Inc.’s recent selection of a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease brought renewed attention to the target, which continues to intrigue a substantial lineup of developers. Bellwether data rolled out this fall from UCB SA and Roche AG at the Clinical Trials in Alzheimer’s Disease meeting in Madrid.
Researchers from E-Therapeutics plc presented preclinical data for ETX-312, a GalNAc-conjugated short interfering RNA (siRNA), produced using the Galomic platform and being developed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
Researchers from Suzhou Siran Biotech Co. Ltd. presented the discovery and preclinical characterization of SA-1211, an N-acetylgalactosamine (GalNAc)-conjugated siRNA dimer targeting both hepatitis B virus (HBV) and PD-L1 gene expression, being developed as a potential new therapeutic candidate for the treatment of chronic hepatitis B (CHB).
Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).
