Consejo Superior de Investigaciones Científicas (CSIC) has synthesized antiviral compounds reported to be useful for the treatment of coronavirus acute respiratory syndrome.
C4 Therapeutics Inc. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN) E3 ubiquitin ligase-binding moiety covalently linked to a Raf kinase B (V600E mutant)-targeting moiety through a linker. They are reported to be useful for the treatment of cancer.
Glioblastoma (GBM) is an aggressive cancer from the CNS usually characterized by a very bad prognosis. It is known that around 30%-35% of patients with GBM develop epilepsy as a comorbidity of the disease.
Being able to detect and monitor the aggregation of α-synuclein in situ could lead to more objective, earlier diagnosis of Parkinson’s disease as well as allow real-time monitoring of whether patients are responding to treatment.
Down syndrome (DS) is the most prevalent genetic cause of Alzheimer’s disease (AD). Previous evidence suggests that increased dosage of the amyloid precursor protein (APP) gene plays a crucial role in AD in individuals with Down syndrome (DS-AD), making APP expression a crucial therapeutic target.
Currently available therapeutic strategies for alcohol use disorder (AUD) have limited efficacy, with many patients not responding to treatment. The dopaminergic system, involved in motivation and the reinforcing effects of drugs, has been proposed as an interesting target in AUD.
Tumor necrosis factor-α (TNF-α) is a key cytokine involved in the pathogenesis of ulcerative colitis (UC), a chronic inflammatory bowel disease, and has emerged as a promising therapeutic target.
Huidagene Therapeutics Co. Ltd. has presented data for HG-303, a new CRISPR-hfCas12Max-based therapeutic approach that knocks down ATXN2 expression for the treatment of amyotrophic lateral sclerosis (ALS).
N4 Pharma plc has successfully completed the first in vivo study of orally delivered Nuvec using its lead program N4 101, an orally administered anti-inflammatory treatment for inflammatory bowel disease (IBD).