To address the need for novel therapeutic candidates against Duchenne muscular dystrophy (DMD), investigators at Mitorx Therapeutics Ltd. developed a library of novel small-molecule mitochondriotropic agents.
Galectin-3 (Gal-3) is used as a marker of activated microglia, and thus a protein that drives inflammation. Patients with Alzheimer’s disease (AD) have shown increased levels of Gal-3 in their cerebrospinal fluid (CSF) compared to healthy controls, as well as increased expression in their brain tissue.
Biocytogen Pharmaceuticals (Beijing) Co. Ltd. and Abl Bio Inc. have established a collaboration to develop new bispecific antibody-drug conjugates (bsADCs).
Vaccines that target α-synuclein have a lot of potential for the treatment of Parkinson’s disease (PD) and other synucleinopathies. Tridem Bioscience GmbH & Co. KG has developed the proprietary WISIT vaccine technology.
Leucine-rich repeat kinase 2 (LRRK2), a key drug target, plays a major role in Parkinson’s disease (PD). Mutations in LRRK2, specifically LRRK2 G2019S which increases LRRK2 kinase activity, have been observed in a majority of PD patients.
Ono Pharmaceutical Co. Ltd. has entered into a drug discovery collaboration agreement with the University of Oxford to verify drug discovery seeds and obtain screening compounds for the creation of new medicines.
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease for which there is a 10% rate of familial cases, with the rest being sporadic cases. Both genetic and environmental factors contribute to the etiology of ALS, and more than 120 genes have been reported to be tied to the disease, but few with strong association. Thus, identifying additional genes contributing to ALS will help shed light on the disease and its related therapies.
Deep learning algorithms have enabled the discovery of molecular structures of interest in biomedicine to design treatments against aggressive diseases such as idiopathic pulmonary fibrosis (IPF). Scientists at Insilico Medicine Inc. selected a target for IPF using artificial intelligence (AI), then designed an inhibitor to block it, and tested it in vitro, in vivo, and in clinical trials.
Scientists at Fujifilm Corp. and Osaka University have described piperazine derivatives acting as drug efflux pump inhibitors reported to be useful for the treatment of bacterial infections.
Sudo Biosciences Ltd. has divulged non-receptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of scleroderma, radiographic ankylosing spondylitis, type 1 diabetes, idiopathic pulmonary fibrosis, multiple sclerosis, psoriasis, rheumatoid arthritis, Sjögren’s syndrome and systemic lupus erythematosus, among others.