Researchers from Exscientia plc presented preclinical data for the novel reversible LSD1 inhibitor EXS-74539, being developed as a monotherapy or in combination with standard of care for the treatment of oncology and hematology indications including acute myeloid leukemia (AML) and small-cell lung cancer.
Duchenne muscular dystrophy (DMD) is a disorder characterized by progressive degeneration of muscles, resulting in muscle loss, mobility limitations and lately, mortal risk. DMD is caused by mutations in the dystrophin gene (DMD) and about 80% of these are suitable for exon skipping, obtaining a functional dystrophin protein.
Left atrial appendage (LAA) occlusion procedures are designed to reduce the risk of thromboembolism in patients who have non-valvular atrial fibrillation, but a study presented at the 2023 Transcatheter Cardiovascular Therapeutics annual meeting in San Francisco demonstrated that about half of patients continue to have LAA leaks – and potentially the risk of dangerous blood clots and stroke – a year after the procedure.
The EU approved the first drug-coated balloon for treatment of in-stent restenosis nearly a decade ago, but U.S. physicians continue to have their hands tied in treating the fairly common problem. Results from Boston Scientific Corp.’s investigational device exemption trial for its Agent paclitaxel-coated balloon presented in San Francisco at Transcatheter Cardiovascular Therapeutics could finally put the tool in the hands of cardiologists. The device demonstrated a nearly 50% reduction in the risk of target lesion revascularization and target vessel myocardial infarction compared to conventional balloon angioplasty.
Researchers from Boehringer Ingelheim Pharma GmbH & Co. KG presented the discovery and preclinical characterization of BI-3706674, a potent and orally available small-molecule inhibitor of the KRAS oncogene, targeting both KRAS-mutant and KRAS wild-type amplified (WTamp) cancers.
Researchers from Università degli Studi di Foggia presented data from a study that aimed to investigate different circulating microRNAs (miRNAs) as possible biomarkers for the diagnosis and prognosis of multiple sclerosis (MS).
Fabry disease is a rare X-linked lysosomal storage disorder where a deficiency in α-galactosidase A (GLA) results in the pathological accumulation of globotriaosylceramide (Gb3 or GL-3) and other glycosphingolipids in vascular endothelial cells, nerve cells, cardiomyocytes and renal cells.
Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT protein, and a recent study conducted by researchers from Huidagene Therapeutics Co. Ltd. aimed to assess the potential of high-fidelity Cas12Max (hfCas12Max)-based gene editing therapy as a novel treatment for HD.
At the 30th Annual Congress of the European Society for Gene and Cell Therapy in Brussels this week, researchers presented both preclinical and clinical strategies for applying gene therapy to a functional HIV cure. At a Wednesday session on Infectious Diseases & Vaccines, Alessio Nahmad, of Tabby Therapeutics Ltd., described using B cells edited to express broadly neutralizing antibodies (bnAbs) 3BNC117 to deliver high titers of antibodies in mice.
The Congress of the European Society of Medical Oncology (ESMO) held in Madrid from Oct. 20 to 24, 2023, could well add the letter W to its acronym after the four sessions on gynecological cancer that could represent a milestone in the lives of all women who suffer from cervical, ovarian, or endometrial cancer.
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