Sialidosis is a lysosomal storage disease caused by mutations in the NEU1 gene, which encodes sialidase neuraminidase 1. These mutations lead to enzyme deficiency and subsequently accumulation of oligosaccharides and sialylated glycopeptides in tissues and body fluids, which in turn lead to cell and organ dysfunction. There are no approved therapies. Three different AAV9 vectors encoding NEU1 were developed and tested by UMass Chan Medical School researchers in the preclinical setting in mice.
The targeted delivery of optimized stem cells directly into injured tissues has been used to maximize efficacy and minimize systemic exposure. Still, despite hundreds of clinical trials evaluating mesenchymal stem cell (MSC) therapy as a treatment, clinical efficacy remains highly variable. Investigators at Case Western Reserve University have developed an optimized combination of cytokines and growth factors applied to MSCs (HXB-319).
NGGT (Suzhou) Biotechnology Co. Ltd. has presented preclinical data on an AAV vector approach that expresses human PAH, rAAV8-PAH, also known as NGGT-002. NGGT-002 has liver tropism and it was codon-optimized for expressing PAH in the liver.
Researchers from Healios K.K. presented preclinical data for HLCN-061, a novel gene-engineered human induced pluripotent stem cell (iPSC)-derived NK cell product being developed for the treatment of solid tumors.
Renal medullary carcinoma (RMC) is characterized by the complete loss of the SMARCB1 tumor suppressor, and it predominantly affects individuals with sickle cell trait (SCT), characterized by increased sickling of red blood cells in the renal medulla. It has been previously demonstrated that RMC tumors show a hypoxia signature, and in a recent study, researchers from MD Anderson Cancer Center aimed to investigate the connection between SMARCB1 loss and hypoxia under the setting of SCT.
Amplo Biotechnology Inc. has been awarded a fast track phase I/II Small Business Technology Transfer (STTR) grant from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health (NIH) to fund further development of AMP-201, an AAV-ColQ gene therapy designed to address congenital myasthenic syndrome caused by collagen Q (ColQ) deficiency.
Nectin cell adhesion molecule 4 (Nectin-4) overexpression in different tumors correlates with cancer progression and poor prognosis in many human malignancies. The first Nectin-4-targeting antibody, enfortumab vedotin (EV), has limited efficacy and frequent adverse effects in several solid tumors. Researchers at Mabwell (Shanghai) Bioscience Co. Ltd. and colleagues have now reported on the generation and characterization of 9MW-2821, an antibody-drug conjugate consisting of an antibody targeting Nectin-4 conjugated to monomethyl auristatin E (MMAE).
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