Using C-X-C chemokine receptor type 4 (CXCR4) antagonists as cell mobilization agents has resulted in some FDA approved agents, such as Plerixafor, for hematopoietic stem cell transplantation and neutropenia. Oral cell mobilizers could result in using them in conditions such as sickle-cell disease (SCD) and chronic neutropenia. Emory University has developed and presented data for their CXCR4 antagonist EMU-116.
Amira Therapeutics SL has announced new preclinical findings for its lead compound AMI-463, which is being developed primarily for rhabdomyosarcoma, the most common soft tissue sarcoma in children. AMI-463 is a first-in-class inhibitor that blocks the cell adhesion molecule (CAM)-related down-regulated by oncogenes (CDON).
Cartography Biosciences Inc. has received IND approval from the FDA for CBI-1214, a T-cell engager being developed for the treatment of colorectal cancer.
Researchers from Chimerix, now part of Jazz Pharmaceuticals, presented preclinical data on ONC-206, a compound that functions as both an agonist of the mitochondrial protease Caseinolytic peptidase P (CLPP) and an antagonist of the G protein-coupled receptor DRD2, in models of triple-negative breast cancer (TNBC).
Werewolf Therapeutics Inc. has announced that its priorities for the coming year include a focus on the company’s Inducer T-cell engager platform, where preclinical studies have demonstrated robust silencing and reduction of off-tumor toxicity.
Viatris Inc. has announced FDA clearance of its IND application for MR-146, an Enriched Tear Film (ETF) AAV gene therapy candidate for the treatment of neurotrophic keratopathy. The company plans to initiate a phase I/II trial in patients with neurotrophic keratopathy in the first half of next year.
During aging, hematopoietic stem cells (HSCs) undergo functional decline affecting their ability to regenerate the hematopoietic system and support lymphoid cell production. This functional decline contributes to some aging-related diseases. Moreover, aging is associated with biomechanical changes in HSCs, including alterations in nuclear envelope tension and nuclear mechanical integrity and mechanotransduction. However, it remains unclear whether aging of somatic stem cells can be prevented by targeting changes in nuclear mechanosignaling.
Researchers at the University of Sydney have uncovered a mechanism that may explain why glioblastoma returns after treatment, and the world-first discovery offers new clues for future therapies. Glioblastoma is one of the deadliest brain cancers, accounting for about half of all brain tumors, with a median survival rate of just 15 months. Despite surgery and chemotherapy, more than 1,250 clinical trials over the past 20 years have struggled to improve survival rates.
Alicorn Pharmaceutical Co. Ltd. has described protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer.
Meta Pharmaceuticals Inc. and Shenzhen Moyuan Pharmaceuticals Co. Ltd. have divulged lactate dehydrogenase A (LDHA) inhibitors reported to be useful for the treatment of autoimmune disorders.
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