Transcripta Bio Inc. has raised $24 million since its series A to fund IND-enabling studies across its portfolio and support clinical preparation in its autism spectrum disorder and facioscapulohumeral muscular dystrophy programs. The company’s pipeline also includes programs for myotonic dystrophy and Huntington’s disease.
Jiangxi Kerui Pharmaceutical Co. Ltd. has reported new sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of acute pain.
Pancreatic polypeptide (PP) selectively activates NPY4 receptors, a signaling pathway that may become impaired as NPY levels decline during Alzheimer’s disease progression. Researchers from Henan University of Chinese Medicine described P1642-1, a novel PP analogue, in the 5×FAD mouse model of AD.
Mentari Therapeutics Inc. has announced a $200 million private placement to support continued development of its pipeline of targeted biologics for migraine.
Insilico Medicine Cayman Topco has nominated ISM-9528 as a preclinical candidate for pain management. ISM-9528 targets an undisclosed novel mechanism to offer a brain-penetrant, orally available, non-opioid treatment option for pain.
BChE inhibition can restore cholinergic signaling and may limit amyloid aggregation, while Nrf2 activation strengthens antioxidant defenses, suppresses neuroinflammation and protects against ferroptosis. Simultaneous modulation of these pathways is therefore expected to provide broader neuroprotection than single-target approaches and may better address the multifactorial nature of AD.
Estrigenix Therapeutics Inc. has closed the first $2 million tranche of a seed financing to move its selective estrogen receptor-β (ERβ) platform through translational preclinical development.
Conventional transplantation approaches using neural progenitors have emerged as a potential alternative for stroke treatment, but their efficacy is limited by poor survival and insufficient migration and integration in the post-stroke environment.
Researchers from Ractigen Therapeutics Co. Ltd. described the preclinical profile of RAG-17, an siRNA therapy designed to knockdown the expression of SOD1, conjugated to a smart chemistry aided delivery (SCAD) system for enhanced CNS delivery via intrathecal injection.
The Foundation for Angelman Syndrome Therapeutics (FAST) has awarded a 2-year grant to Frances Arnold, PhD, winner of the 2018 Nobel Prize in Chemistry, to develop new molecular tools that could help advance future RNA-targeted therapeutics for Angelman syndrome.
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