Insilico Medicine Cayman Topco and Hygtia Therapeutics Co. Ltd. have entered into an exclusive license and co-development collaboration to advance ISM-8969 worldwide. ISM-8969 is an orally available, brain penetrant NLRP3 inhibitor for CNS disorders.
Sundance Biosciences Inc. has discovered non-receptor tyrosine-protein kinase TYK2 inhibitors potentially useful for the treatment of multiple sclerosis.
Leigh syndrome is a fatal pediatric neurodegenerative disorder caused by mitochondrial dysfunction, most often due to defects in the mitochondrial respiratory chain. The Ndufs4 knockout (Ndufs4 KO) mouse is an established model of the disease, as loss of the NDUFS4 subunit leads to complex I (CI) deficiency and reproduces the neurological decline and pathology seen in affected children. Researchers from The Children’s Hospital of Philadelphia Research Institute and collaborators described how NV-354, a water-soluble prodrug of succinate, may mitigate this mitochondrial dysfunction.
Zincure Corp. has discovered matrix metalloproteinase-9 (MMP-9, gelatinase B) inhibitors reported to be useful for the treatment of epilepsy, stroke, multiple sclerosis, hypoglycemic encephalopathy and traumatic brain injury.
Brain-derived tau, a protein that is exclusive to the brain and detectable in the blood, could serve as an indicator of brain damage after an ischemic stroke. The analysis of this special form of tau has revealed a relationship between high levels of the protein and extensive brain injury, a higher risk of complications, and poorer outcomes.
A new systematic review and meta-analysis of studies looking at a possible association between Tylenol (acetaminophen) use in pregnancy and neurodevelopmental disorders in children concludes there is no evidence of any meaningful risk. The study was carried out by researchers in the U.K., Italy, Sweden and Norway, in response to U.S. Secretary of Health and Human Services Robert Kennedy saying last September that there is a link. Unless medically necessary, it is “irresponsible” to take Tylenol during pregnancy, Kennedy said.
As the momentum and interest in brain-computer interface (BCI) technologies continue to accelerate, Merge Labs has emerged with an investment from OpenAI in its seed round. Merge Labs says it plans to develop next-generation BCIs capable of interacting with the brain at high bandwidth, integrating with advanced AI, and doing so in ways that are safe and accessible to everyone.
Latigo Biotherapeutics Inc. has synthesized sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers. They are reported to be useful for the treatment of pain, cough and pruritus.
Alexion Pharmaceuticals Inc. has reported complement C1s subcomponent (C1S) inhibitors. They are described as potentially useful for the treatment of amyotrophic lateral sclerosis, rheumatoid arthritis, lupus nephritis and more.
Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited neuromuscular disorder affecting 9.7-82.3 patients per 100,000 individuals. Over 100 genes with all patterns of inheritance have been linked to CMT. These genes encode proteins involved in nerve-specific processes, such as axonal transport, myelination and synaptic transmission, and in general housekeeping pathways. However, the reason underlying why defects in such ubiquitous proteins predominantly affect peripheral nerves remains unclear.
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