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Our Programs

Our Pipeline

We are developing potentially disease-modifying AAV9-based gene therapies for the treatment of genetically defined neurodegenerative diseases.

Programs Indication Approach
Stage of Development
Discovery
Preclinical
Phase 1/2
Pivotal
PR001 (LY3884961) Parkinson's disease with GBA1 mutations (PD‑GBA) GBA1 Gene Transfer
in phase 1/2
Type 2 Gaucher disease (GD2) GBA1 Gene Transfer
in phase 1/2
Type 1 Gaucher disease (GD1) GBA1 Gene Transfer
in phase 1/2
PR006 (LY3884963) Frontotemporal dementia with GRN mutations (FTD‑GRN) GRN Gene Transfer
in phase 1/2
Additional Programs Neurodegenerative disorders Undisclosed
in preclinical phase
Additional Programs Neurodevelopmental and neurodegenerative disorders Undisclosed
in discovery phase

PR001

PR001 is being developed as a potentially disease-modifying, single-dose gene therapy for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and Gaucher disease Types 1 and 2 (GD1, GD2).

PD-GBA and Gaucher disease are driven by mutations in the same gene, called GBA1. This gene contains the instructions for making the lysosomal enzyme beta-glucocerebrosidase, or GCase, which is needed for the disposal and recycling of glycolipids — a type of cellular component that is known to accumulate with aging. PD-GBA patients have mutations in at least one chromosomal copy of GBA1, while Gaucher disease patients have mutations in both chromosomal copies. Without enough GCase, glycolipids accumulate, causing lysosomal dysfunction and aggregation of the protein α-Synuclein in cells, which we believe leads to the inflammation and neurodegeneration behind PD-GBA and neuronopathic Gaucher disease.

PR001 utilizes the well-studied viral vector AAV9 to deliver a healthy copy of the GBA1 gene and is currently being studied in three Phase 1/2 clinical trials.

Our PROPEL trial is a Phase 1/2 clinical trial of PR001 for the treatment of patients with PD-GBA.
Our PROVIDE trial is a Phase 1/2 clinical trial of PR001 for the treatment of Type 2 Gaucher disease (GD2).
Our PROCEED trial is a Phase 1/2 clinical trial of PR001 for the treatment of Type 1 Gaucher disease (GD1).

At Prevail, we focus on two methods of drug administration, based on the disease type: Intra-cisterna magna and intravenous infusion (IV).

Parkinson’s Disease / Gaucher Disease Type 2 (GD2)

In our PROPEL and PROVIDE clinical trials, PR001 is administered by a one-time injection into an area above the spinal canal, where the brain and spinal cord meet, called the cisterna magna to treat the brain and therefore, the neurodegenerative manifestations of PD-GBA and GD2. The cisterna magna injection is a direct, non-surgical technique that has been used safely in humans for a century.

Gaucher Disease Type 1 (GD1)

In our PROCEED clinical trial, PR001 is administered as a slow, single-dose IV infusion through a venous catheter into a peripheral vein to treat the peripheral manifestations of GD1.

Gaucher Disease Type 1 Pre-Treatment vs Treated

The U.S. FDA has granted Fast Track designation for PR001 for the treatment of PD-GBA and for the treatment of GD2. It has also granted Orphan Drug designation for PR001 for the treatment of patients with Type 1 and Type 2 Gaucher disease, and Rare Pediatric Disease designation for the treatment of GD2.

PR006

PR006 is being developed as a potentially disease-modifying, single-dose gene therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

FTD-GRN is a rapidly progressing neurodegenerative disease caused by a lack of progranulin, a protein that is found both outside of brain cells and inside the cells, in the lysosomes. Healthy levels of progranulin are necessary for cellular processes such as lysosomal function, neuronal survival and normal activity of the microglia, a type of brain-based immune cell. In FTD-GRN patients, mutations in the gene GRN cause the body to produce insufficient progranulin. Without enough of the enzyme, the lysosomes cannot effectively degrade or recycle proteins. This leads to inflammation and neurodegeneration.

PR006 is designed to slow or stop disease progression in FTD-GRN patients by increasing progranulin levels via delivery of a healthy GRN gene into the central nervous system (CNS). Like PR001, PR006 is administered by injection into the cisterna magna, using the well-studied viral vector AAV9.

The U.S. FDA has granted Orphan Drug designation for the treatment of FTD and Fast Track designation for the treatment of FTD-GRN. The European Commission has granted orphan designation for PR006 for the treatment of FTD.

PR006 is currently being studied in our PROCLAIM trial, a Phase 1/2 clinical trial of PR006 for the treatment of patients with FTD-GRN.

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