Novel Oral Therapy for Pompe disease

Novel Oral Therapy for Pompe disease

Posted On: 31/03/2021 Recently Posted!

A new player, MAZE Therapeutics, has joined the growing number of companies with an interest in Pompe disease. This time they are targeting Muscle Glycogen Synthase, the enzyme that causes a very rare muscle form of Glycogen Storage Type 0.

The company claim to reduce the activity level of the enzyme to prevent the harmful build-up of glycogen in muscle cells, without depleting the activity so much as to impair normal muscle function.

Their Substrate Reduction Therapy (SRT) therapy will take the form of an oral pill and they are expecting to enter clinical trials in 2022.

The company are in preliminary discussions with the International Pompe Association, and so we hope to be able to give frequent updates on the progress of this interesting development.

You can read more about the company’s Pompe programme on their website and below is a short extract:

GYS1: our therapeutic target for Pompe disease

We believe a novel approach to treating Pompe disease is halting skeletal and respiratory muscle glycogen synthesis and its subsequent accumulation by inhibiting the action of the gene GYS1 through substrate reduction therapy, or SRT.

High levels of accumulated glycogen are toxic to muscle and our GYS1 inhibitor has been shown to reverses that accumulation in preclinical models