Rgenta Therapeutics Inc. has announced preclinical data on RGT-0474060, an oral PMS1 inhibitor for Huntington’s disease. RGT-0474060 is an orally bioavailable small molecule designed to inhibit PMS1 RNA and protein expression, to modulate splicing of CAG repeat expansion known to cause Huntington’s disease.
The effects of aging pose an additional challenge for people with HIV due to the neurological and psychological consequences that persist despite antiretroviral therapy. At the Conference on Retroviruses and Opportunistic Infections (CROI) held Feb. 22-25, 2026, in Denver, the scientific community examined how the virus affects the brain, how the reservoir is established in the CNS, and which genetic, immunological or treatment-related factors influence cognitive health.
Slate Medicines Inc. has launched with $130 million in series A financing and a focus on advancing next-generation therapeutics for migraine and other headache disorders.
Porosome Therapeutics Inc. has announced the development of novel first-in-class porosome-targeting small molecules and blood-brain barrier-traversing peptide drugs developed using the company’s Porosome.AI platform to treat various secretory disorders, such as Alzheimer’s disease and type 2 diabetes.
Newco Vesalic Ltd. has formed to take forward research indicating extracellular vesicles secreted by skeletal muscle cells carry toxic payloads that are key drivers of motor neuron diseases, including amyotrophic lateral sclerosis. The discovery of this process, which is largely external to the brain and the central nervous system, has opened up new targeting possibilities, and Vesalic is now working on in vivo studies to demonstrate preclinical proof of concept.
Angelini Pharma SpA and Quiver Bioscience Inc. have entered into a collaboration and licensing agreement to advance novel therapeutics for genetic epilepsies. Under the multiyear collaboration, Angelini Pharma will leverage Quiver’s technology platform to gain deeper understanding of a broad set of developmental and epileptic encephalopathies.
Brise Pharmaceutical (Shanghai) Co. Ltd. has discovered carbocyclic or heterocyclic derivatives acting as transient receptor potential cation channel subfamily M member 3 (TRPM3) antagonists.
Work at Emcure Pharmaceuticals Ltd. and Peptris Technologies has led to the identification of new fused bicyclic heterocyclic derivatives acting as histone deacetylase (HDAC) inhibitors.
Lario Therapeutics Ltd. has received a total of $2.4 million in grant funding from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and Wellcome to support the continued expansion of its neuronal calcium channel drug discovery platform. The funding provides validation of Lario’s work on selective small-molecule inhibitors of voltage-gated neuronal calcium channels.
Shanghai SIMR Biotech Co. Ltd. has prepared and tested new GABA-A receptor subunit α5 (GABRA5) inverse agonists. They are reported to be useful for the treatment of pain, stroke and Alzheimer’s disease.
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