MeiraGTx Holdings plc has provided an update on its late-stage clinical pipeline and manufacturing capabilities in genetic medicine. The company highlighted four pivotal-stage programs, including treatments for Parkinson's disease, radiation-induced xerostomia, AIPL1 retinal dystrophy in partnership with Eli Lilly, and X-linked retinitis pigmentosa. MeiraGTx also reported a diverse preclinical pipeline targeting ALS, obesity, Stargardt's disease, and wet age-related macular degeneration, with a broad ophthalmology pipeline in development. The presentation outlined MeiraGTx's in-house cGMP manufacturing infrastructure, which includes two viral vector manufacturing facilities, in-house plasmid production, and a commercially licensed quality control facility. The company emphasized its comprehensive vector engineering technologies, which leverage rational design, high-throughput screening, and AI for novel promoter discovery and gene expression optimization. Additionally, MeiraGTx showcased its proprietary riboswitch platform for titratable gene expression control using oral small molecule inducers. Upcoming regulatory filings for AIPL1 congenital blindness and X-linked retinitis pigmentosa (RPGR) are expected based on ongoing studies. You can access the full presentation through the link below.
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