Researchers from Imperial College London and collaborators further investigated C/EBPβ as a target for MASLD and evaluated the efficacy of a C/EBPβ-targeted siRNA approach to reverse metabolic dysfunction and restore liver function in high-fat-diet-induced steatosis, which can be precursor to MASH and hepatocellular carcinoma.
Effective targeted therapies against aggressive breast cancer subtypes, such as triple-negative breast cancer (TNBC), are still lacking. Developing therapeutics targeting nonenzymatic, intracellular proteins with causal roles in TNBC progression remains a significant challenge.
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.
Dexoligo Therapeutics has presented data for their liver-targeted siRNA DXO-1801 for the potential treatment of inflammation-driven anemia, a serious complication of chronic diseases such as chronic kidney disease, myelofibrosis or advanced solid tumors characterized by elevated pro-inflammatory cytokines where the availability for iron for erythropoiesis is limited due to increased hepcidin levels.
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.
Sanegenebio has obtained clinical trial clearance in China for SGB-7342, an siRNA medicine targeting inhibin β E (INHBE) for the treatment of obesity. A phase I trial is scheduled to begin early 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.
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).
