The Bethesda biotech Gain Therapeutics, armed with supercomputer firepower, aims to disrupt Parkinson’s disease treatment. Their GCase regulator, named GT-02287, completely restored motor function in a recent animal study. GCase, short for glucocerebrosidase, targets both lysosomal and mitochondrial dysfunction. In Parkinson’s, the failure of cellular powerhouses (mitochondria) and recycling centers (lysosomes) disrupts essential functions. This…
Allosteric drugs: A differentiated small molecule approach
Over the last decade, drugs that bind to allosteric sites have emerged as an attractive approach in small molecule drug design, offering new possibilities for targeting various diseases, including conditions historically known as “undruggable”. Unlike traditional small molecules that bind to a target protein’s endogenous orthosteric or active site, allosteric small molecules selectively bind and…