MeiraGTx (MGTX) announced the Company will exhibit five posters at the European Society of Gene and Cell Therapy, ESGCT, 2024 Annual Congress, which is being held from October 22-25, 2024, in Rome, Italy. Title: “Evolution of a high-producing, modular upstream platform for AAV manufacturing.” The poster shows the evolution of MeiraGTx’s upstream manufacturing platform, which was optimized and modulated through the choice of transfection reagents, adeno-associated virus production enhancers, and transfection parameters, in fed-batch and perfusion culture mode. Title: “AAV-based evaluation of novel in silico promoters to drive expression in rod photoreceptors.” Using an AI-assisted promoter engineering approach, ten novel promoter sequences were initially designed, cloned into an adeno-associated virus backbone carrying eGFP and finally packaged into AAV5 or AAV7m8. Title: “Identification of highly potent and tissue-specific promoters with massively parallel screening.” Here, the company developed a massively parallel reporter assay to screen a synthetic library of over 240,000 promoters that are 182 bp long. Title: “AAV-mediated gene therapy attenuates loss of vision in a mouse model of Bardet-Biedl-Syndrome 10.” In this study, researchers set out to optimize and identify an AAV vector carrying the human BBS10 gene providing sustained efficacy and a good safety profile for clinical translation. Title: “Riboswitch-regulated gene and cell therapy.” Here, the company reports that by linking our synthetic aptamer to alternative splicing gene expression platform, researchers have created a robust, synthetic mammalian riboswitch cassette that regulates gene expression tightly and dynamically in response to small-molecule inducers
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