At the recent International Society for Stem Cell Research meeting, Vita Therapeutics Inc. presented the first preclinical results on VTA-100, a cell therapy consisting of induced pluripotent stem cell (iPSC)-derived satellite cells, for the treatment of limb-girdle muscular dystrophy 2A/R1.
Granulosa cell tumors (GCT) are a rare subtype of ovarian cancers, characterized by a slow clinical progression and high rates of late recurrence. With limited treatment options available apart from invasive surgery, targeted therapies would be key. In previous research, scientists at Hudson Institute of Medical Research in Australia demonstrated that targeting X-linked inhibitor of apoptosis protein (XIAP) using small-molecule inhibitors (called Smac-mimetics [SM]) in combination with other compounds could work as a therapeutic approach for GCT.
It has been previously demonstrated that in zebrafish, the activity of UXS1, a gene that encodes UDP-glucuronate decarboxylase 1, is essential for production and organization of skeletal extracellular matrix. Now, researchers from the University of Oslo and affiliated organizations have identified a novel pathogenic variant in UXS1.
It has been previously demonstrated that the Bruton tyrosine kinase (BTK) pathway plays a key role promoting cell adhesion and chemotaxis, and that increased adhesion and aberrant chemotaxis contribute to pathogenesis of myelofibrosis (MF). At the recent EHA meeting, researchers from Telios Pharma Inc. presented preclinical data for TL-895, a novel BTK inhibitor currently in clinical development for the treatment of patients with chronic lymphocytic leukemia and MF.
Researchers from the University of Lausanne and affiliated organizations recently presented data from studies that aimed to identify novel genes associated with neurodevelopmental disease (NDD).
Primary lymphedema (PL) is a chronic condition that results from abnormal development or functioning of the lymphatic system caused by gene mutations. Researchers from St George's University of London and colleagues reported the identification of novel variants in the ERG gene during their study performed through whole genome analysis of PL cases included in the 100,000 Genomes Project.
Along with preliminary first-in-human data, investigators from F-star Therapeutics Inc. recently presented preclinical results for the tetravalent bispecific antibody FS-222, which targets PD-L1 and CD137 and is being developed for the treatment of advanced treatment-refractory solid tumors.
Bruton tyrosine kinase (BTK) is involved in cell proliferation and survival in B-cell malignancies. Both covalent and noncovalent BTK inhibitors are in place, but their clinical benefit may be limited due to acquired BTK mutations.
New research shows base and prime editing can correct some forms of phenylketonuria (PKU) in mice and human cell lines, raising the prospect that this gene-editing approach could allow children born with the inherited metabolic disorder to have a treatment that would avoid the need for dietary restrictions and medication.
Colorectal cancer (CRC) is one of the digestive tract cancers with the highest prevalence. Cancer immunotherapy for CRC brings substantial benefit to patients, but the development of resistance mechanisms and relapsing processes along with immune escape call for new treatment options.
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