Sineugene Therapeutics Co. Ltd. has obtained IND clearance from the FDA for SNUG-01, a first-in-class tripartite motif protein 72 (TRIM72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). A phase I/IIa trial will evaluate SNUG-01 in adults with ALS.
Asthma affects about 300 million people worldwide. Inflammation in asthma may drive irreversible airway remodeling due to subepithelial fibrosis, extracellular matrix degradation and increased smooth muscle cell mass levels, among others. In vitro and in vivo assays were performed to test MutT homolog 1 (MTH1) as a therapeutic target in allergic airway inflammation.
Neuronos Ltd., a subsidiary of Beyond Air Inc.’s, has secured an initial $2 million in equity financing from private investors as part of a larger funding round. This investment will accelerate the preclinical development of Neuronos’s small-molecule drug for autism that leverages the regulation of nitric oxide (NO) levels in the brain. The company’s small-molecule therapy is based on research from Hebrew University.
Hillstar Bio has successfully closed a $67 million series A financing round to support its development of next-generation precision immunology therapies for autoimmune diseases. The company’s approach selectively targets and depletes pathogenic immune cells with the potential to reset the immune system.
Prostaglandins induce the regeneration of muscle in rodents and humans through the prostaglandin E2 receptor EP4 subtype receptor, but this therapeutic pathway's potential is limited due to systemic tolerability. Researchers from Mesentech Inc. recently presented new results on their prostaglandin E2 receptor EP4 subtype receptor agonist irodanoprost trying to address this limitation issue.
Focal segmental glomerulosclerosis (FSGS) is a kidney disease that leads to renal failure, and it affects individuals from different ancestries, the highest prevalence being among African and African American populations. DNA samples from 726 patients with FSGS were obtained and DNA sequencing was performed in the search of mutations tied to FSGS compared to a large pool of control populations.
The three-dimensional analysis of cell types and their locations by spatial transcriptomics provides key information of their interactions within tissues or organs. Based on this technology, scientists at the Wellcome Sanger Institute have developed an AI tool called Nichecompass, which shows a comprehensive view of the cancer microenvironments, the different cells, their locations, and how they communicate with each other through different molecules inside the tumor. This AI could process data in an hour and compare samples before and after a treatment.
Additional early-stage research and drug discovery news in brief, from: Maia Biotechnology, Precision Biosciences, Poxel, Telomir Pharmaceuticals, Traws Pharma.
Hangzhou Bio Sincerity Pharmaceutical Technology Co. Ltd. has identified new benzazepinone derivatives acting as receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of cancer.
Work at Artivila (Shenzhen) Innovation Center Ltd. has led to the discovery of new leucine-rich repeat kinase 2 (LRRK2; Dardarin) inhibitors reported to be useful for the treatment of cancer, leprosy, tuberculosis, colitis, inflammatory bowel disease, Alzheimer’s, Parkinson’s and Huntington’s disease.
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