MMP3 gene therapy for glaucoma; plus programmable mRNA translation and more

A Trinity College Dublin-led team, including Exhaura Ltd. co-founders, reported in Science Advances that an adeno-associated virus (AAV)9 gene therapy encoding the matrix metalloproteinase MMP3 decreased intraocular pressure by increasing aqueous outflow in donor human eyes and in mouse and non-human primate (NHP) models of glaucoma. The vector efficiently transduced corneal endothelial cells in vitro and in vivo, and persistent expression of MMP3 was well tolerated in the NHP model. Trinity holds an IP portfolio related to the use of MMP3 to treat glaucoma.

Kyoto University researchers shared in Nature Communications a molecular toolkit, dubbed CARTRIDGE, that uses Cas proteins as translational repressors and activators in mammalian cells by inserting guide RNA sequences into the 5’ untranslated regions (UTRs) of target mRNAs. ...