Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have discovered GTPase KRAS mutant inhibitors, particularly G12V, G12D and G12C mutant inhibitors. They are reported to be useful for the treatment of cancer.
Children’s Medical Center Corp. has synthesized ceramide glucosyltransferase (glucosylceramide synthase; GLCT-1) inhibitors with potential for use in the treatment of cancer, neurological disorders, infections and more.
GM1 gangliosidosis is a lysosomal storage disease caused by mutations in the human GLB1 gene, encoding the ubiquitous lysosomal β-galactosidase. GM1 causes a rapidly progressing neurodegeneration, which can be lethal in the first 2 years of life in the most severe cases.
Glucocerebrosidase (GCase), encoded by the gene GBA1, is a ubiquitous lysosomal enzyme that breaks down lipid substrates, glucosylceramide (GL-1) and glucosylsphingosine (Lyso-GL1), into glucose and ceramide. Loss-of-function mutations in GBA1 reduce GCase activity, resulting in lipid accumulation within lysosomes and subsequent lysosomal dysfunction.
Cbio A/S has received European regulatory clearance to begin a first-in-human trial of novoleucel, the company’s next-generation T-cell therapy, in late-stage cervical cancer. The phase I/IIa study will enroll patients with persistent or recurrent cervical cancer at Karolinska University Hospital.
Valanx Biotech Gmbh has secured €3 million (US$4 million) in funding to advance its lead antibody-drug conjugate (ADC) program. The financing will enable preclinical development of VLX-ADC-001.
Baseimmune Ltd. has announced its strategic expansion into fibrosis, with a new fibrosis-focused pipeline led by a program for idiopathic pulmonary fibrosis (IPF).
Researchers from Sab Biotherapeutics Inc. presented data generated in the development and validation of a pharmacokinetic (PK) assay for SAB-142 and the evaluation of its toxicology and immunologic effects in a juvenile cynomolgus monkey model.
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