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.
Shandong Quanzhong Biomedical Technology Co. Ltd. has synthesized monoamine transmitter reuptake inhibitors reported to be useful for the treatment of neurological disorders, depression, autism, pain, substance abuse and dependency, epilepsy, osteoporosis and metabolic diseases, among others.
Ventoux Biosciences Inc. has initiated a research program to explore the therapeutic potential of lead compound, VEN-201, in translational preclinical models of spinal cord injury.
Schizophrenia is a complex psychiatric disorder marked by positive, negative and cognitive symptoms, many of which are poorly addressed by current dopamine-blocking antipsychotics. Preclinical studies show that muscarinic acetylcholine M4 receptor positive allosteric modulators (M4 PAMs) can reduce hyperdopaminergic activity and improve both positive and negative symptom-related behaviors, suggesting they may offer enhanced antipsychotic efficacy with improved tolerability.
Aperture Therapeutics Inc. has nominated APRTX-001 as a development candidate, with the program now advancing through IND-enabling studies. APRTX-001 is a CD33-targeting antisense oligonucleotide (ASO) designed for the treatment of frontotemporal dementia and amyotrophic lateral sclerosis, with potential for indication expansion into Alzheimer’s disease.
Could GLP-1 receptor agonists (GLP-1RAs), already used in obesity and diabetes, be repurposed as drugs to slow aging? Hong Kong, one of the places in the world with the highest human longevity, is also home to a scientific study on the effects of GLP-1RAs. For the first time, scientists at the Chinese University of Hong Kong (CUHK) have assessed their pharmacological potential in later life using a multiomics preclinical approach.
Yichang Humanwell Pharmaceutical Co. Ltd. has disclosed morphinan derivatives acting as κ-opioid receptor agonists reported to be useful for the treatment of pain and pruritus.