Beijing Innocare Pharma Tech Co. Ltd. has gained IND clearance in China to conduct clinical trials of ICP-538, an orally administered molecular glue degrader targeting VAV1, which is a key protein downstream of T-cell and B-cell receptors. ICP-538 is being studied for the treatment of autoimmune diseases, such as inflammatory bowel disease, systemic lupus erythematosus and multiple sclerosis.
Vesalic has characterized a systemic metabolic dysfunction that creates a toxic exosome cargo in amyotrophic lateral sclerosis (ALS) patients, which is carried to the CNS, where it binds to and damages neurons, yielding a novel druggable target against the disease.
TMEM97 regulates microglial activation and neuronal stress pathways linked to neuropathic pain and neuroinflammation in multiple sclerosis (MS). Pharmacological targeting of TMEM97 has shown neuroprotective effects in models of traumatic brain injury, Huntington’s disease and retinal ganglion cell degeneration, and may reduce pain while preserving neuronal function.
Laminins are extracellular matrix (ECM) glycoproteins that preserve the structural and functional integrity of tissues. These heterotrimeric proteins, composed of one α, β and γ chain encoded by different genes, provide mechanical support, facilitate cell adhesion and maintain tissue organization and stability.
Astellas Pharma Inc. recently presented data regarding ASP-2246, a drug candidate comprised of an mRNA encoding the transcription factor NeuroD1 encapsulated in lipid nanoparticles.
Parkinson’s disease (PD) is a neurodegenerative disorder that affects movement, and tremor is one of its signatures. But it is a much more wide-ranging disorder, and patients experience problems with cognitive and emotional processes as well. SCAN, the somato-cognitive action network identified in 2023, could reshape the definition of PD. Treating this circuit can improve outcomes.
Yichang Humanwell Pharmaceutical Co. Ltd. has identified compounds acting as potassium voltage-gated channel subfamily KQT member 2/3 (KCNQ2/3) activators. They are reported to be useful for the treatment of amyotrophic lateral sclerosis, depression, epilepsy, stroke, multiple sclerosis, pain, and Alzheimer’s and Parkinson’s disease, among others.
Researchers from the University of Oxford and the Health Research Institute La Fe (Spain) investigated the potential of multigene RNA-based therapeutics in Alzheimer’s disease, aiming to overcome potential compensatory mechanisms and patient heterogeneity.
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