Medulloblastoma is one of the most common pediatric brain malignancies. A deeper understanding of the mechanisms underlying tumor cell fate determination is crucial to develop novel, effective and safe therapeutic strategies. With this aim, researchers from the University of Chinese Academy of Sciences and collaborating institutions recently conducted a study integrating whole-genome bisulfite sequencing and bulk RNA sequencing (RNA-seq) data from 189 human medulloblastoma samples.
Sarepta Therapeutics Inc. has filed a clinical trial application (CTA) in New Zealand seeking clearance to initiate a first-in-human trial of SRP-1005 (formerly ARO-HTT). Pending approval, the INSIGHTT trial is anticipated to begin in the second quarter of this year.
Biopharma industry zeal for obesity therapeutics does not appear to be dying down any time soon, with Eli Lilly and Co. entering a $1.3 billion deal with Nimbus Therapeutics LLC for a new small molecule, and Arrowhead Pharmaceuticals Inc. rolling out phase I/IIa data of its RNAi therapeutics used in combination with Lilly’s GLP-1/GIP receptor agonist tirzepatide.
Rona Therapeutics Co. Ltd. has announced the submission of RN-5681 to the Australian Human Research Ethics Committee (HREC), and anticipates dosing to begin in a phase I trial in the first quarter of next year.
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.
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