Cambridge Gene Therapy

Cambridge Gene Therapy was a biotechnology spin-out from the University of Cambridge, founded in 2017 by Professor Timothy Cox. The company was dedicated to developing gene therapies for rare, neurodegenerative lysosomal storage disorders, with a primary focus on GM2 gangliosidosis (including Tay-Sachs and Sandhoff diseases). Its therapeutic approach utilized adeno-associated virus (AAV) vectors to deliver functional human hexosaminidase subunits directly to the brain via convection-enhanced delivery. Although the company secured seed funding from Cambridge Enterprise and obtained orphan drug designations for its clinical program, it has since been dissolved.