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.
Takeda Pharmaceutical Co. Ltd. has synthesized heterocyclic compounds acting as orexin OX2 receptor (OX2R; HCRTR2) agonists reported to be useful for the treatment of narcolepsy, among others.
Scineuro Pharmaceuticals Holdings Ltd. has received a $5 million research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to accelerate the preclinical development of the company’s novel LRRK2-targeted antisense oligonucleotide (ASO) program, SNP-614, for Parkinson’s disease.
Revir Therapeutics Inc. has announced progress in its HTT-PMS1 genetic medicine program for Huntington’s disease with the identification of a lead compound and the award of a $4.6 million grant from the California Institute for Regenerative Medicine (CIRM).
A significant share of the risk and heritability of attention-deficit hyperactivity disorder (ADHD) is explained by rare genetic variants. A study led by scientists from Aarhus University in Denmark has uncovered their weight in this condition and identified three variants that will help to better understand their role, the risk of developing it, or its comorbidities, in contrast with the common and more frequent variants associated with ADHD.
Jiangsu Hengrui Pharmaceuticals Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have divulged aromatic compounds acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain.
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