Despite the title of the Sunday, June 4 lead-off presentation at the American Society of Clinical Oncology (ASCO) meeting in Chicago, there was little room left for doubt about the increasingly important place of artificial intelligence (AI) in drug development. The program, Artificial Intelligence for Drug Development: Fad or Future, ultimately pointed to a positive future, with the only faddish part being discarded approaches that no longer work.
α-Synucleinopathies constitute a set of neurological disorders including Parkinson’s disease (PD), dementia with Lewy bodies, multiple systems atrophy (MSA), and other rare disorders. The development of positron emission tomography (PET) tracers for imaging α-synuclein aggregates is essential for performing efficient and accurate diagnosis, tracking disease progression and monitoring efficacy of potential therapies.
Researchers from Atux Iskay LLC assessed whether reactivation of protein phosphatase 2A (PP2A) using a small-molecule PP2A activator, ATUX-1215, could counter TGF-β signaling in preclinical models of idiopathic pulmonary fibrosis (IPF).
Charcot-Marie-Tooth disease (CMT) is a group of neuropathies characterized by sensory and motor dysfunction that progress with aging. It is considered that about 60% of the axonal forms of the disease, such as CMT2, remain genetically undiagnosed.
Bietti’s crystalline corneoretinal dystrophy (BCD) is an autosomal recessive inherited disease caused by mutations in the cytochrome P450 (CYP) family 4 subfamily V member 2 (CYP4V2) gene, which encodes a polyunsaturated fatty acid (PUFA) hydroxylase dominantly expressed in retinal pigment epithelium (RPE) cells.
Mutations in the RPGRIP1 gene are associated with rare retinal dystrophies and most commonly with Leber congenital amaurosis (LCA) type 6, which is characterized by vision loss, among other symptoms.
Glioblastoma multiforme (GBM) is an aggressive brain cancer with poor prognosis and survival. TNF-related apoptosis-inducing ligand (TRAIL) is a protein that induces apoptosis in cancer cells by binding death receptors type 4 and 5. Researchers at the University of North Carolina explored using hiNeuroS-TRAIL combined with the ClpP activator TR-107 as a potential treatment.
At the recent ATS meeting, Tavanta Therapeutics Inc. introduced TAVT-135, a cell-penetrating peptide (CPP) conjugate that acts as a chloride ion (Cl-) transporter. The product is currently being investigated for cystic fibrosis (CF), regardless of CF transmembrane conductance regulatory (CFTR) gene mutation status.