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Advancing gene therapy

At Prevail, we are developing gene therapies designed to slow or stop the underlying causes of neurodegenerative and rare diseases

Learn more about our clinical research
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We are developing gene therapies that aim to target the genetic root causes of neurodegenerative and rare diseases

Our goal is to help those living with diseases that have limited treatment options beyond symptom management.

Two people in lab coats and gloves walking down a hallway while reviewing documents.

Gene therapy is a medical treatment that uses genetic material to treat or prevent diseases caused by faulty genes. By addressing the root cause, it has the potential to change how certain diseases are managed

Learn more about our clinical researcH

Definitions

β-glucocerebrosidase (GCase)

An enzyme in the body. It helps break down fatty substances. These substances are recycled inside lysosomes, which are the recycling centers of cells. If GCase doesn’t work properly or is missing, fat builds up in lysosomes. This buildup happens in places like the spleen, liver, and bone marrow. It can cause health problems, such as Gaucher disease.

Cerebrospinal fluid (CSF)

A clear fluid that surrounds and cushions the brain and spinal cord, protecting them and helping transport nutrients and waste.

Cisterna magna

An area at the base of the skull where the brain and the spinal cord meet. This space is filled with cerebrospinal fluid and can be used by doctors to deliver treatments directly to the brain and spinal cord.

Clinical trials

Research studies that test new treatments or procedures in people to make sure they are safe and work well.

Dopamine transporter single-photon emission computed tomography (DaT-SPECT)

A scan that is used to diagnose neurodegenerative Parkinson’s disease from other types of Parkinson’s disease. For example, Parkinson’s disease caused by medicines or reduced blood supply to the brain.

Electrocardiogram (ECG)

A test that checks the heart’s rhythm and electrical activity using small sensors attached to the skin.

Enzyme replacement therapy (ERT)

A treatment that gives the body enzymes it’s missing or that aren’t working properly. For Gaucher disease, ERT provides a replacement for the GCase enzyme through an infusion. This helps reduce the buildup of fat in the body and manage symptoms.

Frontotemporal dementia (FTD)

A type of dementia that gets worse over time. It damages the brain’s frontal and temporal lobes. These areas control personality, behavior, language, and movement, which means FTD can lead to significant changes in how a person acts, communicates, and moves.

FTD with GRN genetic variants

FTD is a type of dementia that damages the brain’s frontal and temporal lobes, causing changes in personality, behavior, language, and movement.

About 5–10% of FTD cases are linked to genetic variants in the GRN gene. The GRN gene provides instructions for making progranulin, a protein needed to keep brain cells healthy. Genetic variants in GRN lead to lower levels of progranulin, causing brain cell damage and faster disease progression. People with these genetic variants often experience more severe and rapidly worsening symptoms.

GBA1 genetic variant

The GBA1 gene gives cells the instructions to make the GCase enzyme. This enzyme helps break down fatty substances inside lysosomes, which are the cell’s recycling centers. A GBA1 genetic variant is a change in this gene. This change can stop the GCase enzyme from working properly. When this happens, fatty substances build up in the body. This buildup can cause Gaucher disease or increase the risk of Parkinson’s disease.

Gaucher disease (GD)

A rare condition passed down through families, caused by changes (genetic variants) in the GBA1 gene. These changes affect a specific enzyme called GCase, which is needed to break down certain fatty substances. When GCase doesn’t work properly, these substances build up in lysosomes, the recycling centers of cells. Over time, this buildup can affect organs like the spleen, liver, and bone marrow, causing symptoms such as fatigue, bone pain, and organ swelling.

Gene therapy

A type of treatment that uses genetic material, such as DNA or RNA, to fix or replace faulty genes in the body. This treats or prevents certain diseases by enabling the genes to work as they should.

Genetic variant

A genetic variant is a change in a gene that can affect how it works. Changes in genes are mostly harmless. But, in some cases, they can make the body work differently and increase the risk of certain diseases. Genetic variants were previously called ‘mutations’.

Immunosuppressants

Medicines that lower the activity of the immune system. In gene therapy, they are used to reduce the chances of the immune system reacting to the inactivated virus that delivers the gene therapy to cells.

Informed Consent Form (ICF)

A document that provides important details about a clinical trial. It explains the potential risks and what is required before, during, and after the clinical trial. It also outlines the rights of participants. Participants sign the form to show they understand the clinical trial and agree to take part.

Infusion

A way of delivering fluids, including medicine, directly into the bloodstream using a needle or small tube.

Intravenous / intravenously (IV)

A method of giving medicine or fluids directly into a vein.

Lumbar puncture

A medical procedure where a small sample of cerebrospinal fluid is taken from the lower back using a needle. It’s sometimes called a spinal tap.

Lysosomes

Small parts of cells that act as recycling centers. They break down and recycle waste materials, including fatty substances, to keep the cells healthy. When lysosomes don’t work properly, these substances can build up and cause health problems, such as in Gaucher disease.

Magnetic resonance imaging (MRI)

A medical scan that uses powerful magnets and radio waves to create detailed images of the inside of the body, like the brain or spine.

Parkinson’s disease (PD)

A brain condition that affects movement. It happens when certain brain cells that produce a chemical called dopamine stop working properly. Without enough dopamine, the brain cannot send proper signals to the body. This can cause symptoms like tremors, stiffness, slow movements, and trouble with balance.

Parkinson’s disease with GBA1 genetic variants

Parkinson’s disease is a brain condition that affects movement, caused by the loss of brain cells that produce dopamine. Without enough dopamine, symptoms like tremors, stiffness, and slow movements can occur.

Some cases of Parkinson’s are linked to genetic variants in a gene called GBA1, the most common genetic risk factor for the condition. These genetic variants increase the risk of developing Parkinson’s and can lead to earlier and faster-progressing symptoms.

Peripheral vein

A vein found in areas like the arms, hands, legs, or feet. These veins are closer to the surface of the body and are commonly used for giving medications or drawing blood.

Placebo-controlled

In a clinical trial where there are two groups of participants, one group receives the active treatment or intervention, while the other group receives an inactive treatment or intervention (the placebo). The placebo is administered in the same way as the active treatment but does not contain any medicine.

Small interfering RNA (siRNA)

A type of therapy that uses small pieces of RNA, a natural molecule in cells, to block specific genes from making harmful proteins. siRNA works by ‘silencing’ genes that may cause or worsen certain diseases. This therapy is different from traditional gene therapy, as it focuses on turning off harmful gene activity rather than replacing or fixing genes.

Substrate reduction therapy (SRT)

An oral treatment (tablet) used for Gaucher disease. It works by partially stopping the body from making fatty substances. These substances are usually broken down by an enzyme called GCase. When GCase doesn’t work properly, these substances build up in lysosomes, the recycling centers of cells. SRT helps reduce this buildup and manage symptoms caused by Gaucher disease, such as organ swelling, fatigue, and bone pain.

Type 1 Gaucher disease

Type 1 Gaucher disease is the most common form of Gaucher disease, a rare inherited condition caused by genetic variants in the GBA1 gene. These genetic variants affect an enzyme called GCase, leading to a buildup of fatty substances in lysosomes, the recycling centers of cells.

Type 1 Gaucher disease usually begins during adolescence but can start at any age. Symptoms include bone pain, fractures, enlarged organs, bruising, and fatigue. Unlike other types, Type 1 does not typically affect the brain or spinal cord.
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