The therapeutic validity of RNA editing tools in vivo remains largely unknown in disease models both in terms of RNA editing efficacy and therapeutic improvement of disease-related symptoms. In recent work, scientists from the Institute of Neuroscience at the Chinese Academy of Sciences and colleagues evaluated RNA correction therapy in a mouse model that recapitulates the phenotype of human dominant-inherited deafness. Read More
An inhibitor of sodium/glucose cotransporter 2 (SGLT2), luseogliflozin, was found to have protective effect in a model of acute ischemic stroke in research by Kyushu University scientists and their collaborators. Read More
Pasithea Therapeutics announced positive results from a preclinical proof-of-concept study of PAS-002, the company's DNA tolerizing vaccine construct encoding GlialCAM, a molecule that has been recently identified in the brain's white matter, which is the portion of the brain attacked in multiple sclerosis (MS). Read More
As the resident innate immune cells of the brain, microglia are emerging as key drivers of neurological diseases, but as yet there is no systematic way of exploring their potential as drug targets. Read More
Scripps Research Institute and Vova Ida Therapeutics have divulged novel compounds that may inhibit NAD consumption and/or increase NAD synthesis, reported to be useful for neurodegenerative, degenerative and metabolic disorders, including amyotrophic lateral sclerosis, diabetes, nonalcoholic fatty liver disease (NAFLD), Parkinson's disease and renal, metabolic and retinal diseases, among other disorders. Read More
Seagen has described new antibody-drug conjugates (ADCs) comprising antibodies covalently bound to stimulator of interferon genes protein (STING; TMEM173) agonists through a linker and reported to be useful for the treatment of cancer. Read More
E-Scape Bio has identified indazoles acting as leucine-rich repeat kinase 2 (LRRK2; dardarin) and/or LRRK2 (G2019S mutant) inhibitors reported to be useful for the treatment of Parkinson's dementia, multiple system atrophy, Lewy body dementia, Parkinson's disease and Alzheimer's disease. Read More
Reata Pharmaceuticals has described ursolic acid derivatives acting as nitric oxide (NO) production inhibitors and/or nuclear factor erythroid 2-related factor 2 (NFE2-related factor 2; NFE2L2; NRF2) activators reported to be useful for the treatment of cancer and inflammatory disorders, among other disorders. Read More
Beckley Psytech has synthesized new tryptamine analogues acting as 5-HT2A and/or 5-HT2B and/or 5-HT2C receptor agonists reported to be useful for the treatment of depression. Read More
Researchers presented data from a study that aimed to investigate the mechanism by which extracellular vesicle (EV)-associated miRNAs are involved in the activation of autophagy and the development of chemoradiation resistance in advanced non-small cell lung cancer (NSCLC). Read More
Ceapro announced that it has entered into an additional research project expanding on the ongoing collaboration with McMaster University to develop an inhalable immuno-therapeutic/-prophylactic for COVID-19-induced lung fibrosis. Read More
Athira Pharma has presented data on the development and evaluation of novel orally bioavailable, blood-brain-barrier-penetrant small-molecule hepatocyte growth factor (HGF)/mesenchymal-epithelial transition factor (MET) positive modulators for the potential treatment of cognitive impairment. Read More
Cullgen announced that the Chinese National Medical Products Administration (NMPA) has allowed the IND application for CG-001419, a first-in-class, selective, potent oral targeted protein tyrosine receptor kinase (TRK) degrader for the treatment of neurotrophic tyrosine receptor kinase (NTRK) fusion-positive cancers, which have been identified in numerous solid tumors including non-small cell lung, breast and pancreatic cancers. Read More
Homology Medicines announced the details of its optimized, in vivo, one-time gene therapy product candidate, HMI-204, being developed for the treatment of metachromatic leukodystrophy (MLD). Read More
CGeneTech announced that the ALK inhibitor CGT-9475, which was independently developed by Shengshi Tyco, has been approved by the FDA to enter clinical trials aiming to evaluate the candidate's ability to overcome the issues of drug resistance and brain metastasis in the treatment of lung cancer. Read More
