Method for treatment of glycogen storage disease type III by AAV vector containing a bacterial glycogen debranching enzyme

Value Proposition

Glycogen storage disease (GSDIII, Cori Disease) is due to a genetic mutation in the glycogen debranching enzyme (GDE), and can cause uncontrolled glycogen buildup in liver, muscles, and other body compartments. There is currently no treatment for GSDIII other than diet modulation.

Technology

This invention is a novel method for treatment of GSDIII using an AAV vector to carry a smaller bacterial GDE in lieu of the large human GDE.

Other Applications

This method can potentially utilize GDEs from various different bacterial strains.

Advantages

This invention could provide the first effective GSDIII therapy.

This invention utilizes an AAV vector, which has shown efficacy for other gene therapies.

This invention further uses a smaller bacterial gene rather than a larger human gene whose size could provide challenges in treatment delivery.