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BioWorld - Wednesday, December 17, 2025
Home » Topics » Drugs » RNAi

RNAi
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Liver over digital lens background
Gastrointestinal

Bispecific siRNA knocks down YAP1, WWTR1 with single guide strand

Dec. 15, 2025
No Comments
Yes-associated protein (YAP), encoded by Yes1-associated transcriptional regulator (YAP1) and transcriptional coactivator with PDZ-binding motif (TAZ), encoded by WW domain containing transcription regulator 1 (WWTR1), are two crucial paralog transcriptional regulators of the Hippo pathway regulating organ size, tissue homeostasis and disease progression.
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Illustration of siRNA structure
Drug design, drug delivery & technologies

Peptidream reveals milestone in Alnylam collaboration

Dec. 15, 2025
No Comments
Peptidream Inc. has announced a preclinical development milestone in its collaboration with Alnylam Pharmaceuticals Inc. under their siRNA conjugate discovery collaboration.
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Illustration of glucose molecules in a bloodstream
Endocrine/metabolic

Junevity raises funds to advance siRNA metabolism program

Dec. 4, 2025
No Comments
Junevity Inc. has raised $10 million in new funding, bringing its seed financing to a total of $20 million. The new funding will enable Junevity to advance JUN-01, its lead siRNA program for type 2 diabetes and obesity, through IND-enabling and initial clinical studies.
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Liver disease
Gastrointestinal

Tangram seeks clinical trial clearance for TGM-312 for MASH

Nov. 26, 2025
No Comments
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
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Redemplo packaging

Arrowhead’s plozasiran cleared for FCS; Ionis patent duel awaits

Nov. 18, 2025
By Jennifer Boggs
No Comments
Arrowhead Pharmaceuticals Inc. won U.S. FDA approval of plozasiran in familial chylomicronemia syndrome (FCS), the second drug to gain clearance for use in the rare genetic disease following the late 2024 nod for Ionis Pharmaceuticals Inc.’s Tryngolza (olezarsen), setting up a battle in the marketplace even as the firms wage a patent dispute regarding the two RNA-based therapies.
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Illustration of clot forming in blood vessel
Hematologic

City Therapeutics seeks clinical trial clearance for CITY-FXI

Nov. 11, 2025
No Comments
City Therapeutics Inc. has submitted a clinical trial application (CTA) to the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a phase I study of CITY-FXI, an investigational RNAi therapeutic targeting factor XI (FXI) for the treatment of thromboembolic diseases.
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RNA strand

Lilly moves deeper into RNAi with $1.2B Sanegene obesity deal

Nov. 11, 2025
By Tamra Sami
No Comments
In a deal worth $1.2 billion, Suzhou Sanegene Bio Inc. and Eli Lilly and Co. are partnering to advance RNAi candidates for metabolic diseases based on Sanegene's tissue selective delivery technology.
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RNA strand

Lilly moves deeper into RNAi with $1.2B Sanegene obesity deal

Nov. 10, 2025
By Tamra Sami
No Comments
In a deal worth $1.2 billion, Suzhou Sanegene Bio Inc. and Eli Lilly and Co. are partnering to advance RNAi candidates for metabolic diseases based on Sanegene's tissue selective delivery technology.
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Microscope with slide
Cardiovascular

Corsera Health seeks clinical trial clearance for COR-1004

Oct. 28, 2025
No Comments
Corsera Health Inc. has filed a clinical trial notification (CTN) seeking to initiate a phase I trial of COR-1004 in New Zealand. COR-1004 is a novel investigational subcutaneously administered siRNA targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) to reduce LDL cholesterol (LDL-C).
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Red blood cells in an artery with cholesterol

Kalexo enters dyslipidemia fray with preclinical siRNA candidate

Sep. 23, 2025
By Marian (YoonJee) Chu
Mabwell Bioscience Co. Ltd. and Aditum Bio Management Co. LLC announced, in after-market hours Sept. 17, an agreement to forge a new company called Kalexo Bio Inc. and load the biotech with a preclinical dyslipidemia asset via a potential $1 billion global license deal.
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