Gene therapy is a rapidly growing field of scientific research in which healthy genes are introduced into a patient’s cells to treat or prevent their genetic disease.

What is gene therapy?

What is gene therapy?

Genes are inherited from your parents, they act as a set of instructions for many functions in the cells of the body. However, some people are born with a gene that does not work properly (called a mutation) which can lead to conditions such as Fabry disease.

Gene therapy is an experimental technique that uses genes to treat or prevent disease. There are already a few gene therapies that have been approved to treat diseases. In the future, this technique may allow doctors to treat more disorders by inserting a gene into a patient’s cells instead of using drugs or surgery.

Researchers are testing several approaches to gene therapy to fight disease:

  • Replacing a gene that is not normal (mutated) with a healthy copy
  • Making a mutated gene inactive
  • Introducing a new gene into the body to help fight a disease

What is ST-920 and how does it work?

In Fabry disease, the gene called galactosidase alpha (GLA), that provides instructions for making an enzyme called alpha-galactosidase A (α-Gal A), does not function correctly.

When α-Gal A enzyme is produced in low quantities, a fatty substance called Gb3 builds up in the tissues, causing disease in vital organs.

What is ST-920 and how does it work?

ST-920 is designed to deliver a healthy copy of the GLA gene to the liver. The liver should then be able to produce the α-Gal A enzyme and secrete it via the blood stream to the rest of the body. It is hoped that a potential increase in the α-Gal A enzyme in the blood could reduce the need for use of current treatments for Fabry disease.