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α-Gal A enzyme/alpha-galactosidase A enzyme

An enzyme involved in breaking down complex molecules to simpler molecules within the body, notably so they can be eliminated. α-Gal A helps prevent a fatty substance called Gb3 (globotriaosylceramide) from building up in the tissues, which causes disease in vital organs.

Classical Fabry disease

People with classical Fabry disease do not produce any α-Gal A enzyme or very little enzyme. The disease can significantly impact the normal functioning of many vital organs in the body, mainly the heart, kidneys and skin.

Clinical study

Medical research studies involving people. Researchers test possible new drugs in the laboratory to begin with and then, if they look promising, they are carefully tested in people in clinical studies.

Discontinuation/study discontinuation/termination

If you decide to leave the clinical study, or if you are withdrawn from it.

Efficacy

The ability of the medicine to achieve the intended result.

Enzyme

A substance that acts as a catalyst (stimulus) in living organisms, regulating the rate at which chemical reactions happen.

Enzyme replacement therapy (ERT)

A medical treatment which replaces an enzyme that is deficient or absent in the body. It is a treatment option for people with Fabry disease.

Fabry disease

A debilitating rare genetic disorder that can significantly impact the normal functioning of many vital organs in the body, mainly the heart and kidneys.

Gb3 (globotriaosylceramide)

A fatty substance that, in Fabry disease, can build up in the tissues of the body when the α-Gal A enzyme is in low quantities in the blood, causing disease in vital organs.

Gene therapy

The treatment of disease by delivery of a new gene into a patient’s cells to replace an incorrect or damaged gene. Most often, gene therapy works by introducing a corrected copy of a defective gene into the patient’s cells, without removing or modifying DNA. The goal of gene therapy is to treat, or potentially cure, a genetic disease by adding back a normal copy of the gene involved for the disease.

Genetic testing

The study of a person’s DNA in order to identify genetic differences or susceptibility to particular diseases or abnormalities.

GLA gene

The GLA gene is responsible for providing the body with instructions for cells to produce an enzyme called alpha-galactosidase A (α-Gal A).

Infusion

A medicine administered directly into the vein with a needle or catheter.

Non-classical Fabry disease

People with non-classical Fabry disease, also referred to as late-onset or atypical Fabry disease, have low levels of α-Gal A enzyme, and may have a more variable disease course than those with classical Fabry disease. They are generally less severely affected and disease may be limited to a single organ.

Phase I/II clinical study

A study that tests the safety, side effects and best dose of a new treatment. Phase I/II clinical trials can also test how well a certain type of disease responds to a new treatment.

Prednisone

A steroid that can be used to decrease the body’s immune response and decrease inflammation.

Progressive disease

Sometimes also called a chronic disease. The symptoms may gradually worsen over time as the disease progresses. For example, in Fabry disease the symptoms may get worse over time as more and more of the body’s cells and organs are affected by the build-up of the fatty substance Gb3. The symptoms of many chronic diseases can be improved with various treatments.

Quality of life

The standard of health, comfort and happiness felt.

Safety (clinical study)

How safe an investigational agent is, and what side effects it may cause.

Sangamo

A pioneering company focusing on advancing genomic medicine to change how diseases are treated, helping patients look forward to a better future. Sangamo’s research efforts in Fabry disease will concentrate on investigating the ability of gene therapy to treat this disease, where there is a clinical need.

Screening

The process to identify whether a person is suitable to take part in a clinical study.

Side effect [treatment]

An undesirable effect caused by a treatment.

ST-920

ST-920 is an investigational gene therapy designed to deliver a healthy GLA gene to the liver. The liver should then be able to produce α-Gal A and secrete it via the blood stream to the rest of the body.

Tolerability

The degree to which side effects of a treatment can be tolerated (accepted) by the participant in a clinical study.