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.
Liaoning Haisco Pharmaceutical Co. Ltd. has identified crystalline salts of known cholesterol side-chain cleavage enzyme, mitochondrial (CYP11A1) inhibitors reported to be useful for the treatment of cancer.
Farsight Medical Technology (Shanghai) Co. Ltd. has synthesized cyclosporin derivatives acting as peptidyl-prolyl cis-trans isomerase F, mitochondrial (PPIF; cyclophilin D) and/or peptidyl-prolyl cis-trans isomerase A (PPIA; cyclophilin A; CYPA) inhibitors reported to be useful for the treatment of asthma, cancer, multiorgan failure, viral infections, rheumatoid arthritis, renal disorders, neurological disorders and cardiovascular disorders, among others.
Shanghai Fosun Pharmaceutical Industry Development Co. Ltd. has disclosed kinesin-like protein KIF18A inhibitors reported to be useful for the treatment of cancer.
Acute lung injury (ALI) is a severe condition marked by uncontrolled lung inflammation and tissue damage, often leading to respiratory failure. Excessive immune activation recruits neutrophils that release pro-inflammatory cytokines, including IL‑6 and TNF‑α, while generating reactive oxygen species (ROS) that damage DNA and trigger cell death. Thus, controlling inflammation and limiting ROS are key strategies to prevent ALI.
Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.
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