Prevail Therapeutics, a company that focuses on developing gene therapies for neurodegenerative diseases, has announced that a composition of matter patent has been issued for PR001. This therapy is intended to treat neuronopathic Gaucher disease and Parkinson’s disease with GBA1 mutations.
About Gaucher Disease
Gaucher disease is a lysosomal storage disorder. The enzyme beta-glucocerebrosidase has extremely low or no activity, and it leads to the accumulation of a lipid called glucocerebroside. This buildup causes damage to the tissue and organs. The GBA gene is mutated in those who have this disease, and it is responsible for the production of beta-glucocerebrosidase. It is passed down in an autosomal recessive pattern, meaning a child must inherit the mutated version of the gene from both parents. The incidence of the general population is one of every 60,000 people, but 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 is a less common symptom of this type. Types II and III affect the central nervous system (CNS), and they present all of the symptoms of type I. Along with these effects, 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 rapidly 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.
About Parkinson’s Disease
Parkinson’s disease is a progressive disorder that affects the central nervous system (CNS). It is characterized by its effect on movement through five different stages. As the disease progresses, severity increases.
- Stage one is characterized by subtle tremors on one side of the body.
- In stage two symptoms are more noticeable, with tremors and rigidity on both sides of the body.
- Stage three brings loss of balance and slowed movement.
- Stage four makes it impossible for one to live independently.
- Stage five is the most severe, as patients cannot stand or walk. Hallucinations and delusions are common symptoms of this stage.
Parkinson’s disease occurs due to the death of motor neurons, some of which produce dopamine. Dopamine is important in the transmittance of messages to the muscles from the brain, so the loss of dopamine results in the loss of motor functions. Abnormal brain activity occurs when these neurons are lost. Doctors do not know why these motor neurons die, but they do suspect a few factors that play a role, such as genetics, environmental factors like toxins, and Lewy bodies.
About the Patent
The United States Patent and Trademark Office issued Patent No. 10,837,028 on November 7 for the AAV vector used in PR001. This patent extends until October of 2038, but Prevail can apply for extensions or more coverage.
This patent marks a very important step for PR001, which is currently being investigated in a Phase 1/2 trial called PROPEL for patients with Parkinson’s disease with GBA1 mutations. There is also a Phase 1/2 PROVIDE trial, which intends to evaluate the gene therapy as a treatment for Gaucher disease.
Researchers predict that the next analysis of data for the PROPEL trial will take place halfway through 2021, while enrollment for the PROVIDE trial will begin at the end of this year. If all goes well, the next update on this trial will come later in 2021.
The FDA has already granted PR001 the Fast Track designation for the treatment of both disease, the Rare Pediatric Disease designation for neuronopathic Gaucher disease, and the Orphan Drug designation for Gaucher disease. These are all very exciting developments, and Prevail is hopeful that PR001 will see similar success in the future.
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