Ascletis Pharma Inc. has selected ASC-35, a once-monthly, subcutaneously administered GLP-1 receptor (GLP-1R)/GIP receptor (GIPR) dual peptide agonist, as a clinical development candidate. Ascletis expects to submit an IND application to the FDA for ASC-35 for the treatment of obesity in the second quarter of next year.
Lysosomal homeostasis is crucial to the metabolism of certain proteins and lipids that would otherwise accumulate, thus leading to cellular stress and pathology. This is common in diseases such as Parkinson’s disease and Gaucher disease. Researchers set out to find a brain-penetrant small-molecule agonist of the lysosomal channel TRPML1, also known as mucolipin-1.
Researchers at the Institute for Bioengineering of Catalonia (IBEC) and collaborators have introduced a novel therapeutic strategy for Alzheimer’s disease (AD) that leverages the multivalency of supramolecular nanomedicines to reprogram blood-brain barrier (BBB) function, facilitating efficient amyloid-β (Aβ) clearance and restoring cognitive function in animal models.
Myrobalan Therapeutics Nanjing Co. Ltd. has divulged uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-like) antagonists reported to be useful for the treatment of multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease.
Sichuan Kelun Pharmaceutical Research Institute Co. Ltd. and Suzhou Kelun Pharmaceutical Co. Ltd. have identified carboxylic acid ester compounds reported to be useful for anesthesia.
Beijing Chempion Biotechnology Co. Ltd. has synthesized antibody-drug conjugates comprising antibodies targeting folate receptor α (FOLR1; FR-α) covalently linked to a cytotoxic drug through a linker reported to be useful for the treatment of cancer.
Beijing Avistone Pharmaceuticals Biotechnology Co. Ltd. has disclosed spiro/bridged ring compounds acting as HER2 (erbB2) (exon 20 insertion [Ex20Ins] [Ala775_Gly776insTyrValMetAla mutant]) inhibitors reported to be useful for the treatment of atherosclerosis, cancer and pulmonary fibrosis.
Parkinson’s disease (PD) is a progressive disease characterized by loss of dopaminergic neurons in the substantia nigra, which lead to motor symptoms. Aromatic-L-amino-acid decarboxylase (AADC) converts levodopa into dopamine and glial cell line-derived neurotrophic factor (GDNF) promotes the survival, growth and regeneration of dopaminergic neurons. VGN-R09b is an adeno-associated viral vector (AAV)-based AADC and GDNF combination gene therapy that is delivered to the striatum for the treatment of PD.
Polo-like kinase 4 (PLK4), which regulates centriole duplication and mitotic progression, is overexpressed in several cancers, including neuroblastoma as well as breast, lung and colorectal cancers, making it an attractive target. Several PLK4 inhibitors have been developed, but only one has entered clinical trials, which involve patients with acute myeloid or chronic myelomonocytic leukemia.