Inhibiting transient receptor potential melastatin 2 (TRPM2) may mitigate injury arising from ischemic stroke. Researchers at Zhejiang University and its medical school previously reported a small-molecule inhibitor of TRPM2, which also inhibited the human ether-a-go-go-related gene (hERG) channel, implying high risk of cardiotoxicity.
Brenig Therapeutics Inc. has described leucine-rich repeat kinase 2 (LRRK2; dardarin) and (LRRK2; dardarin) (G2019S mutant) inhibitors reported to be useful for the treatment of Parkinson’s disease, among others.
The number of deaths caused by prion diseases reaches about 30,000 annually. Only 5 months pass from the diagnosis of seemingly healthy patients to the fatal outcome of this neurodegenerative condition, and just 1 month until quality of life is completely lost. Removing the brain protein that causes this genetic or infectious disorder could be achieved thanks to new gene-silencing techniques. At a special meeting of the American Society of Gene & Cell Therapy, in “AAV-mediated epigenetic editing for prion disease,” Sonia Vallabh presented not just the data of her research, but the impact of this disease on her family and on herself.
Biohaven Therapeutics Ltd. has synthesized pyruvate kinase M2 (PKM2) activators reported to be useful for the treatment of age-related macular degeneration, amyotrophic lateral sclerosis, multiple sclerosis, retinitis pigmentosa and fibrosis.
Researchers at Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and Suzhou Vigonvita Life Sciences Co. Ltd. have described NMDA receptor antagonists and/or monoamine transporter inhibitors reported to be useful for the treatment of neurological disorders.
Captor Therapeutics Inc. has disclosed molecular glue degraders comprising a cereblon (CRBN) binding moiety acting as serine/threonine-protein kinase Nek7 degradation inducers reported to be useful for the treatment of amyotrophic lateral sclerosis, Still’s disease, Behçet’s disease, familial Mediterranean fever, Alzheimer’s disease, pulmonary arterial hypertension and more.
Gemma Biotherapeutics Inc.’s GB-221, a novel gene therapy for spinal muscular atrophy type 1 (SMA1), has received clinical trial clearance from ANVISA, Brazil’s health regulatory agency.
Changes to a U.S. CDC website regarding autism and vaccines has sparked a backlash from numerous scientific and other groups, placing HHS Secretary Robert F. Kennedy Jr. (RFK) in the spotlight once again for appearing to break promises made earlier this year to secure his nomination.
Vanderbilt University has divulged muscarinic M1 receptor positive allosteric modulators (PAMs) reported to be useful for the treatment of pain, Alzheimer’s disease, schizophrenia, attention deficit hyperactivity disorder (ADHD), tardive dyskinesia, Tourette syndrome, sleep disorders and cognitive disorders, among others.
CSPC Pharmaceutical Group Ltd. has obtained approval from China’s National Medical Products Administration (NMPA) to initiate clinical trials with the company’s selective 5-HT2A receptor agonist, SYH-2056 tablets, for the treatment of depression.
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