Transformative Gene Regulation Technology
The delivery of biologics directly to cells and tissues where they are needed using gene therapy promises to revolutionize medicine. One critical piece of this therapeutic strategy will be the ability to modulate the levels of transduced proteins – tuning them on or off, up or down – to maximize therapeutic effect. MeiraGTx has developed a suite of genetic switches that allow the regulation of expression of any linked gene therapeutic. This new technology will enable the correct levels of any transduced protein to be maintained in patients by the simple oral administration of a specific small molecule. In this way, we are harnessing the power of next generation gene therapies through precision dosing.
Groundbreaking Research on the Underlying Causes of Neurodegenerative Disease
The endosomal network in the cell is critical to the processing of proteins and has emerged as a point of convergence for research in neurodegenerative diseases such as Alzheimer’s disease (AD) and Parkinson’s disease (PD). In seminal observations, endosomal dysfunction was observed to precede the deposition of amyloid and the formation of intracellular tau tangles, the hallmark proteopathies of AD pathology. A central regulator of endosomal protein trafficking is a multi-protein assembly called the retromer complex. In key model-guided screens of gene expression in the AD brain, and in follow up experiments, the retromer complex has been shown to play a pivotal role in AD pathology and emerged as an important target for therapeutic intervention in the disease. MeiraGTx and our academic partners are pursuing proof-of-concept experiments to design gene therapy-based approaches to modulating retromer complex activity to treat neurodegenerative diseases such as Alzheimer’s disease and Parkinson’s disease.