Shenzhen Icarbonx Intelligent Peptide Pharmaceutical Technology Co. Ltd. has disclosed peptides targeting IQ motif and SEC7 domain-containing protein 1 (IQSEC1; BRAG2) reported to be useful for the treatment of stroke, major depression, substance abuse and dependence, and Alzheimer’s disease.
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.
Otsuka Pharmaceutical Co. Ltd. filed an NDA with the U.S. FDA for centanafadine (formerly EB-1020), a new nonstimulant therapy for treating attention-deficit hyperactivity disorder (ADHD) in children, adolescents and adults. Centanafadine is a norepinephrine, dopamine and serotonin reuptake inhibito formulated as a once-daily extended-release capsule.
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.
Nervgen Pharma Corp. rolled out expanded findings from the Connect phase Ib/IIa study with NVG-291 in spinal cord injury patients showing that the 35-amino acid peptide derived from the intracellular wedge domain of phosphatase sigma provided durable functional gains that continued at week 16 and after.
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