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BioWorld - Sunday, February 1, 2026
Home » siRNA

Articles Tagged with ''siRNA''

Illustration of siRNA structure
Cardiovascular

Huadong Medicine and Synerk to jointly develop siRNA drug

Dec. 30, 2024
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.
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Cardiovascular

SYH-2062 cleared for clinic in China for hypertension

Dec. 24, 2024
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.
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Illustration of double helix
Endocrine/metabolic

Wave files clinical trial application for WVE-007

Dec. 24, 2024

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. 


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Neurology/psychiatric

ATL-201, a di-siRNA, reduces seizures in KCNT1-driven epilepsy

Dec. 12, 2024
Pathogenic variants in the KCNT1 gene, which encodes potassium channel subfamily T member 1, cause a severe childhood developmental epileptic encephalopathy.
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Illustration of tau accumulating in a neuron cell.

UCB/Roche lead Voyagers in Alzheimer’s tau campaign

Dec. 6, 2024
By Randy Osborne
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.
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Liver and DNA
Endocrine/metabolic

New siRNA therapy candidate, ETX-312, shows efficacy in MASH mouse model

Dec. 5, 2024
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).
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Hepatitis B virus
Infection

SA-1211, a dual-target siRNA with promising antiviral activity in AAV-HBV mice

Dec. 4, 2024
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).
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Illustration of siRNA structure
Neurology/psychiatric

Ractigen Therapeutics’ RAG-21 designated orphan drug for ALS

Nov. 20, 2024
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).
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John Maraganore, co-founder and executive chair, City Therapeutics
Drug design, drug delivery & technologies

Alnylam’s founder brings in a $135M series A for City Therapeutics

Oct. 14, 2024
By Lee Landenberger
The founding CEO of Alnylam Pharmaceuticals Inc. is now leading the charge with newly launched City Therapeutics Inc., which just completed a $135 million series A financing. City’s executive chair, John Maraganore, will be in familiar territory as the new company plans to develop RNAi-based medicines using next-generation siRNA engineering. He expects dozens of these therapies to reach the market in a relatively short period of time, not just from City Therapeutics but from other companies. It’s a period in the development timeline that he finds reminiscent of the rise and development of monoclonal antibodies.
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John Maraganore, co-founder and executive chair, City Therapeutics
Newco news

Alnylam’s founder brings in a $135M series A for City Therapeutics

Oct. 11, 2024
By Lee Landenberger
The founding CEO of Alnylam Pharmaceuticals Inc. is now leading the charge with newly launched City Therapeutics Inc., which just completed a $135 million series A financing. City’s executive chair, John Maraganore, will be in familiar territory as the new company plans to develop RNAi-based medicines using next-generation siRNA engineering. He expects dozens of these therapies to reach the market in a relatively short period of time, not just from City Therapeutics but from other companies. It’s a period in the development timeline that he finds reminiscent of the rise and development of monoclonal antibodies.
Read More
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