Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease. Read More
Chronic obstructive pulmonary disease (COPD) is a prevalent and heterogeneous respiratory disorder with limited effective treatments. IL-33 and IL-4Rα are key mediators of airway inflammation in COPD and hence represent potential therapeutic targets. Read More
ALX Oncology Holdings Inc. is set to begin phase I studies around the middle of this year with ALX-2004, an antibody-drug conjugate (ADC) for the treatment of EGFR-expressing solid tumors, following IND clearance last month. Read More
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). Read More
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. Read More
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). Read More
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. Read More
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.
Consejo Superior de Investigaciones Científicas (CSIC) has synthesized antiviral compounds reported to be useful for the treatment of coronavirus acute respiratory syndrome. Read More
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. Read More
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. Read More
Merck Sharp & Dohme LLC has identified receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, inflammatory bowel disease, psoriasis, rheumatoid arthritis, multiple sclerosis, stroke, Alzheimer’s disease and Parkinson’s disease, among others. Read More
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. Read More
Esperion Therapeutics Inc. has described ATP citrate lyase (ACLY) inhibitors reported to be useful for the treatment of cancer, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), type 2 diabetes, chronic kidney disease, autoimmune disease and inflammatory disorders. Read More
