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Patients

We are committed to ensuring patients and families remain at the heart of our work as we strive to advance potential one-time gene therapies for people living with neurodegenerative diseases.

Recognizing the critical need to include patient and caregiver voices throughout the drug development process, we seek to understand the unique needs of each disease community we serve, collaborate with advocacy leaders around shared goals and initiatives, and empower decision-making through information sharing and community education.

Parkinson’s Disease (PD)

Parkinson’s disease is a severe and progressive neurodegenerative disorder that affects more than seven million people worldwide and up to one million people in the United States. Although Parkinson’s disease has historically been categorized as a movement disorder, people living with PD can experience a range of non-motor symptoms, including psychosis, dementia and cognitive impairment.

PD with GBA1 mutations (PD-GBA)

Genetic studies have identified dozens of genes linked to Parkinson’s disease. Mutations in the GBA1 gene are now known to be the single largest genetic risk factor for developing Parkinson’s disease, causing a subtype of the disorder known as PD-GBA. It is estimated that as many as seven to ten percent of people living with Parkinson’s disease worldwide, and some 90,000 patients in the United States, carry at least one GBA1 mutation. Click here .

Clinical trial: PROPEL PR001

We are developing PR001, a potentially disease-modifying, single-dose gene therapy for people living with Parkinson’s disease with GBA1 mutations (PD-GBA). Click here to learn more about Prevail’s research in PD-GBA. For more information visit ClinicalTrials.gov

Gaucher Disease

Gaucher disease is a lysosomal storage disorder caused by variations in the GBA1 gene. These genetic variants lead to dysfunctional glucocerebrosidase enzyme (GCase), a lysosomal enzyme that helps break down and recycle certain fatty substances in the cell. Without functional GCase, the fatty substances accumulate in the lysosome rather than being recycled. The lysosomes swell in size, transforming healthy cells into namesake Gaucher cells which clog organs, reducing their function and resulting in a wide range of symptoms throughout the body.

Gaucher disease has three subtypes (Type 1, Type 2 and Type 3) classified by variation in the severity of symptoms, age of onset, and the presence or lack of symptoms that affect the brain.

Type 1 Gaucher disease (GD1)

Type 1 Gaucher disease (GD1) has a wide variety of symptoms, such as spleen and liver enlargement, low blood counts, issues with bruising, bleeding, bone damage, bone pain and severe fatigue. People living with GD1 are at a higher risk of developing Parkinson’s disease because of the genetic connection between both diseases.

Clinical trial: PROCEED PR001

We are developing LY3884961 (formerly PR001), a potentially disease-modifying, single-dose gene therapy for people living with Type 1 Gaucher disease. Click here to learn more about Prevail’s research in Gaucher disease. For more information visit ClinicalTrials.gov.

Type 2 Gaucher disease (GD2)

Type 2 Gaucher disease (GD2) is the most severe form of Gaucher disease and primarily affects children within the first two years of life. Type 2 and Type 3 Gaucher disease are also known as neuronopathic Gaucher disease (nGD) because these forms mainly involve symptoms that affect the brain and central nervous system. GD2 brings about rapid, progressive and irreversible damage to the brain, which is usually noticeable within the first six months of life. Sadly, children diagnosed with GD2 have a shortened lifespan, as the disease is typically fatal by the age of two.

Children with GD2 not only have issues related to the brain, causing symptoms such as seizures, difficulty feeding, difficulty breathing, visual problems and increased muscle tone (known as spasticity), but in some cases may also experience symptoms that affect various parts of the body. These symptoms can include an enlarged spleen and liver along with blood abnormalities.

GD2 can present along a spectrum and each case may be different. Unfortunately, there are currently no approved treatments to address the neurological symptoms associated with GD2. Support and resources are available for affected children and their families. If you suspect your child may have GD2 or if you’ve already received a diagnosis, reach out to your healthcare team for guidance and assistance.

Clinical trial: PROVIDE PR001

We are developing LY3884961 (formerly PR001), a potentially disease-modifying, single-dose gene therapy for children and infants diagnosed with Type 2 Gaucher disease. Click here to learn more about Prevail’s research in neuronopathic Gaucher disease (nGD). For more information visit ClinicalTrials.gov

Frontotemporal Dementia (FTD)

Frontotemporal dementia is the second most common cause of dementia in people under the age of 60 (after Alzheimer’s disease), affecting 50,000 to 60,000 patients in the U.S. and 80,000 to 110,000 in the European Union. FTD is characterized by the progressive degeneration of the frontal and temporal lobes of the brain, which control decision-making, behavior, emotion and language.

FTD with GRN Mutations

Several forms of FTD are known to be caused by genetic mutations. For example, mutations in the GRN gene lead to lower levels of progranulin, an important protein needed for normal functioning of various cells. This reduction in progranulin leads to the neuroinflammation and neurodegeneration observed in FTD-GRN, considered the most aggressive and rapidly progressive form of FTD. Click here for more information.

Clinical trial: PROCLAIM PR006

We are developing PR006, a potentially disease-modifying, single-dose gene therapy for people living with frontotemporal dementia with GRN mutations. Click here to learn more about Prevail’s research in FTD-GRN. For more information visit ClinicalTrials.gov

To learn more about how clinical trials work and general considerations for participation, visit:

Check out these advocacy groups for helpful information and resources:

If you or a loved one is affected by Parkinson’s disease, Gaucher disease, frontotemporal dementia, or another genetic or rare condition, you may wish to connect with one or more of the patient advocacy groups listed below for additional information and resources.

For questions about Prevail’s research or programs, please contact prevail.patients@lilly.com.

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