Researchers at Indian Institute of Technology (BHU) Varanasi and collaborators have designed inhibitors of cholinesterase based on benzothiazole-phenylpiperizine as a potential treatment for Alzheimer’s disease.
Researchers at Shanghai Jingxin Biopharmaceutical Co. Ltd. and Zhejiang Jingxin Pharmaceutical Co. Ltd. have divulged compounds acting as leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of Parkinson’s disease.
Essential tremor is a movement disorder marked by involuntary, rhythmic shaking in the hands but sometimes affecting the head, voice and other areas. Its exact cause is still unknown. Recent research suggests that dysfunction in the neurotransmitter systems, particularly involving γ-aminobutyric acid (GABA), contributes significantly to the disorder.
Neurocrine Biosciences Inc. has disclosed vesicular monoamine transporter 2 (VMAT2) inhibitors reported to be useful for the treatment of neurological and psychiatric disorders, among others.
Some 1 million people around the world suffer severe neurological problems, such as epilepsy, motor impairment and cognitive dysfunction, because they express insufficient SynGAP1, a GTPase-activating protein that operates postsynaptically. Current therapies can mitigate symptoms but not cure the underlying disease. Researchers at the Allen Institute and collaborators have demonstrated a potential genetic cure for SynGAP1 deficiency.
Tr1x Inc. has obtained IND clearance from the FDA for TRX-319, paving the way for initiation of a phase I/IIa study in progressive multiple sclerosis (MS) early next year. TRX-319 is designed to combine targeted B-cell control with active anti-inflammatory signaling and T-cell regulation, with the goal of restoring immune balance.
Scientists at Indiana University and Purdue Research Foundation have synthesized inositol polyphosphate-5-phosphatase D (SHIP-1; INPP5D) inhibitors reported to be useful for the treatment of mild cognitive impairment, Alzheimer’s disease, vascular cognitive impairment, Lewy body dementia and frontotemporal dementia.
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in the enzyme palmitoyl-protein thioesterase 1 (PPT1) due to biallelic loss-of-function mutations in the gene encoding the lysosomal enzyme, leading to widespread neurological dysfunction and premature death.
Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.
Inhibiting histone deacetylase 6 (HDAC6) has therapeutic potential against several neurodegenerative disorders. A collaboration including researchers from Eikonizo Therapeutics Inc., spanning the U.K., U.S. and France, developed EKZ-438, which has shown strong potential against amyotrophic lateral sclerosis and frontotemporal dementia in preclinical studies.
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