Prevail Therapeutics recently submitted an Investigational New Drug application for their gene therapy called PR001. The FDA has recently notified them that this application is active, meaning that Prevail Therapeutics can begin to conduct their clinical trial. They are planning Phase 1/2, which they plan to begin in the first half of 2020. This therapy is meant to treat neuronopathic Gaucher disease (nGD), which is a lysosomal storage disorder. Prevail Therapeutics believes that “PR001 has tremendous potential to help patients.”
About Gaucher Disease
Gaucher disease is a lysosomal storage disorder. It occurs when the enzyme beta-glucocerebrosidase has extremely low or no activity, which leads to the accumulation of a lipid called glucocerebroside. This accumulation causes damage to the tissue and organs. The GBA gene, which is responsible for the production of beta-glucocerebrosidase, is mutated in those who have this disease. It is passed down in an autosomal recessive pattern, meaning a child must inherit the mutated version of the gene from both parents. While it is very rare in the general population, at one of every 60,000 people, it is more common within the Ashkenazi Jewish population.
Gaucher disease comes in multiple different types, which vary in symptoms and severity. Type I is characterized by an enlarged liver or spleen, anemia, easy bruising, and various bone issues like pain, fracturing, and arthritis. Lung disease may also be a symptom of this type, but it is not as common. Types II and III affect the central nervous system, and they present all of the symptoms of type I. Along with these symptoms, those with types II and III may also experience unusual eye movements, seizures, and brain damage. Type II is the most severe of the three, as it progresses quickly and is fatal. While many symptoms are present in all types, it is important to remember that different individuals present various symptoms with differing severity.
This disease is diagnosed with enzyme assay through a blood test or genetic testing. While both of these things can diagnose someone with Gaucher disease, the combination of the two are necessary to diagnose the specific type. Once one has been diagnosed, treatments vary depending on the type of Gaucher disease. For type II there are no treatments, but symptoms can be managed. Enzyme replacement therapy or oral medications exist for those with types I and III.
About PR001 and the Study
PR001 is a disease-modifying AAV-based gene therapy for the treatment of Type II Gaucher disease. Prevail Therapeutics specializes in treatments of this type, and they specifically focus on neurodegenerative diseases. The company has already submitted data to the FDA about safety of this therapy, which has led to the active status of their Investigational New Drug application. Based on their previous findings, Prevail Therapeutics will continue into Phase 1/2 with a higher dosage than originally planned.
Enrollment is expected to begin in the first half of 2020. Along with Phase 1/2 for the treatment of Type II Gaucher disease, Prevail Therapeutics also expects to begin a Phase 1/2 for the treatment of Type III in the second half of 2020. The company states that there is an unmet need for treatments of Gaucher disease, and that they hope their therapy will be able to better the lives of those with this disease.
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