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.
Peptidream Inc. has announced a preclinical development milestone in its collaboration with Alnylam Pharmaceuticals Inc. under their siRNA conjugate discovery collaboration.
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.
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).
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.
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.
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.
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.
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).
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.