An Innovative Hematopoietic Stem Cell Gene Therapy Approach Benefits CLN1 Disease in the Mouse Model
Scientists provided evidence of the efficacy and feasibility of a novel approach to treat CLN1 disease and possibly other neurodegenerative conditions, paving the way for its future clinical application.
[89bio, Inc.] 89bio, Inc. announced positive topline data from the Phase IIb ENLIVEN trial evaluating treatment with pegozafermin in patients with NASH. In the study, both the 44mg Q2W and 30mg QW doses met, with high statistical significance.
[Communications Biology] The expression of myocardial infarction-associated transcript (MIAT) was up-regulated during liver fibrosis. Silencing MIAT led to the suppression of hepatic stellate cell proliferation and collagen expression.