Help Us Explore New Ways to Regenerate Hope in Parkinson’s Research

If you or a loved one has Parkinson’s disease, continue reading to learn more about the REGENERATE-PD Phase 2 study of an investigational* gene therapy, currently enrolling eligible participants.

The REGENERATE-PD study is sponsored by AskBio

*Investigational means that the therapy has not been approved for use and is still being studied to further evaluate its safety and efficacy.

Nicole, living with Parkinson's disease

Nicole, living with Parkinson's disease

Nicole, living with Parkinson's disease

About the REGENERATE-PD Study

The REGENERATE-PD Phase 2 study aims to better understand how an investigational, potential one-time treatment may help stabilize or improve symptoms of Parkinson’s disease and positively impact daily living.

FAST FACT 1

This is a clinical study for the treatment of Parkinson’s disease.

FAST FACT 2

This is a study of an investigational gene therapy called ametefgene parvec (previously known as AB-1005) that is administered only once. Ametefgene parvec (say am-uh-tef-jean par-veck) is being evaluated to see if it works to stabilize or improve symptoms in Parkinson’s disease.

FAST FACT 3

Ametefgene parvec is called “investigational” because it is still being studied and is not yet approved for use in any country.

Who may be able to join?

Here’s a brief eligibility checklist to get started:

  • Adults (aged 45–75 years old)

  • Diagnosed with Parkinson’s disease between 4 and 10 years ago

  • Experience about 2 ½ hours of OFF time each day

  • Never had brain surgery

  • Never had cell therapy or gene therapy

If you can check all these boxes, then we’d love to talk to you to share more information about the study. There are also other criteria. We would welcome the opportunity to discuss with you to confirm if you may be able to participate.

Help Us Explore New Ways to Regenerate Hope in Parkinson’s Research

How is ametefgene parvec designed to work?

The study will evaluate the investigational gene therapy called ametefgene parvec. This one-time administration of gene therapy may help support dopamine-producing neurons in the brain and have a long-lasting benefit for patients living with Parkinson’s disease.

Here is why we believe it may work:

Dopamine is a chemical in the brain that is important for movement, motivation, and learning.
Some of the core symptoms of Parkinson’s disease occur because the neurons that are supposed to create dopamine become damaged and can’t function well. Without these neurons making enough dopamine, people with Parkinson’s disease have symptoms that include shaking, slow movement, muscle stiffness, difficulty balancing, and slurred speech.
What if these dopamine-producing neurons could be restored? That is the goal of ametefgene parvec. Instead of supplementing with dopamine, this gene therapy may provide a way to continuously support growth and survival of the neurons in the brain that naturally produce dopamine. When these neurons are working normally they should make the dopamine that is needed.
If the survival and function of dopamine-producing neurons in the brain is successful, symptoms of PD in patients may stabilize or improve.
Ametefgene parvec is being studied as a one-time gene therapy treatment that aims to target dopamine-producing neurons and to understand if this may be effective and last for a period of time.
AskBio, Faces of Hope, Nicole LaBolle

Nicole, living with Parkinson's disease

AskBio, Faces of Hope, Nicole LaBolle

Nicole, living with Parkinson's diseaseNicole, living with Parkinson's diseaseNicole, living with Parkinson's disease

AskBio, Faces of Hope, Nicole LaBolle

Nicole, living with Parkinson's diseaseNicole, living with Parkinson's diseaseNicole, living with Parkinson's disease

Get Started

We’re here to answer your questions—even if you’re not yet sure if this study is right for you.

What Participation Might Look Like

We are grateful to every person who considers participating in clinical studies. Without willing participants, we cannot do the important research needed to bring new treatments to patients now and in the future. If you are interested in taking part, the study team will guide you in every step, answer your questions, and make sure you feel comfortable throughout your journey.

Participation involves uncertainty, some procedures (including surgery) involve risks. Participation in this study may not provide direct benefit.

If you choose to participate, here is an overview of what you can expect. Click each card to learn more.

Baseline Evaluations

Baseline evaluation: 2–3 months

We will carefully evaluate your current health and medical history to confirm your eligibility to participate in the study. This includes some baseline exams, blood testing, questionnaires, and brain imaging.

Random Assignment

Random Assignment

2 of 3 participants will be randomly assigned to receive ametefgene parvec during surgery. 1 of 3 study participants will be randomly assigned to a control surgery group and will not receive the investigational gene therapy.

Surgery

Hospital: 1–3 days

The surgery will be performed in the hospital by a specialized brain surgeon. You will be hospitalized to monitor your recovery after surgery. Most participants are able to go home the day after surgery. Check the FAQs for more information on possible risks associated with surgery.

Follow-up

Follow-up: 18–48 months

Follow-up clinic visits will be scheduled every 3 to 6 months.  These visits will track your progress, review any medical concerns, complete health assessments, and perform neurological examinations, blood tests, and collect brain imaging to assess your Parkinson’s disease symptoms and potential impact of the investigational gene therapy. At the end of this phase, you’ll find out if you were in the group that received the gene therapy or the control surgery group.
After completion of the follow-up, participants who received ametefgene parvec may be eligible to continue with a longer follow-up study. For participants in the control group, they may have the option to enter a new study to receive ametefgene parvec.
Appointments with the study team and for your surgery may require travel. Travel arrangements will be made for you and your care partner and all study-related costs will be covered.

For Care Partners

We know the importance of care partners for people living with Parkinson’s disease. We invite and encourage your care partner to play an active role in supporting you through this clinical study, if you choose to participate. Care partners are welcome to join you at your appointments throughout the study, and we’ll cover their travel costs as well as yours.

Paul, living with Parkinson’s disease

What is gene therapy?

Understanding AAV gene therapy

Genes are the blueprints of the human body, telling the body’s cells how to make proteins function. Gene therapy can target faulty genes to correct, increase, or reduce the production of specific proteins responsible for disease. To do this, genes need a safe way to be carried into the body. We use an exciting technology called adeno-associated virus (AAV), which allows the healthy genetic material to be introduced into living tissue. There is no virus being put into the body in this process. AAV gene therapy is important in the treatment of many diseases.

Ametefgene parvec

Ametefgene parvec uses AAV gene therapy to deliver a copy of a human gene to brain cells, providing instructions to make GDNF. GDNF is a protein that supports the health and survival of brain cells that are dying in people living with Parkinson’s disease. This increases a protein that is already in the body and does not affect existing DNA.
Click the image above to watch the video.

Our hope is that ametefgene parvec may provide a long-term treatment solution for people with Parkinson’s disease.

Help Us Explore New Ways to Regenerate Hope in Parkinson’s Research

Paul, living with Parkinson’s disease

Research Sites

The REGENERATE-PD study is recruiting in the United States, the United Kingdom, Poland, and Germany.

Travel costs to the study location for you and your care partner, study treatment, medications, and all procedures required by the study will be provided to you at no cost.

Help Us Explore New Ways to Regenerate Hope in Parkinson’s Research

Frequently Asked Questions (FAQs)

The treatment being studied in REGENERATE‑PD is investigational and has not been approved for use. Its safety and effectiveness have not yet been fully established. Participation in this study may not provide direct benefit.

Gene therapy is a medical technique that involves delivering genetic material directly into a person’s cells to treat or prevent disease.

Dopamine is a chemical messenger that plays an important role in movement, motivation, and learning. It’s produced by specific nerve cells in the brain. However, in people with Parkinson’s disease, the nerve cells that produce dopamine become damaged or may die, making it harder for the brain to send signals to the body. This disruption causes the symptoms of Parkinson’s disease, which include shaking, slow movement, muscle stiffness, difficulty balancing, and slurred speech.

Ametegene parvec is intended to help support the survival and development of these cells so that they may continue to produce dopamine. This gene therapy instructs the cells to make a growth factor, GDNF, that may help these important brain cells function normally and allow dopamine to be made. Once the gene therapy is in the brain, it may be able to stimulate regrowth of dopamine-producing cells and improve a person’s symptoms of Parkinson’s disease. This is why ametefgene parvec only needs to be administered once.

Yes, it has been tested in 11 Parkinson’s patients in a Phase I clinical trial, designed to evaluate safety only, where it was found to be well-tolerated. We learn new information from every study. Based on past studies, we have now learned how to deliver ametefgene parvec directly to the brain.

The treatment being studied in REGENERATE‑PD has not been approved for use and is still being studied to further evaluate its safety and efficacy.

Ametefgene parvec will be injected once via brain surgery, under general anesthesia. Your study neurosurgeon will use an advanced surgical method to administer ametefgene parvec directly into the putamen, a part of the brain known to play a key role in Parkinson’s disease. Before this happens, the study team will explain the surgery and what you should know.

The treatment being studied in REGENERATE‑PD has not been approved for use and is still being studied to further evaluate its safety and efficacy.

As with many studies, some people who take part will be randomly placed into a control group and will not receive ametefgene parvec. A control group gives the researchers something to compare the investigational gene therapy with to evaluate whether it works.

In the REGENERATE-PD study, if you are part of the control group, you will still have surgery, but the neurosurgeon will only make small cuts to your scalp so that you look the same as the people who were randomly assigned to get ametefgene parvec during the surgery. If you undergo control surgery, you will not be given any gene therapy during this study.

Initially, neither you nor your study team will know if you received ametefgene parvec during the surgery, but you will find out later in the study. If you receive the control surgery, you may be able to receive ametefgene parvec at a later time.

You will continue to take your Parkinson’s medications and see your regular doctor as well as the study team during the study.

All investigational medications and therapies carry some risk. They are being evaluated to see if they may be effective and safe for use in routine clinical practice. That’s why, before any clinical study is conducted, it must first be reviewed by independent experts to review the risks of the study and ensure participants are aware of potential risks prior to starting the study. That is also why we take the time to thoroughly check your medical history and perform baseline health assessments to be sure the most appropriate patients enroll in the study.

Before you decide to participate, the study team will take time to explain the study and provide information about the known risks that may be involved. The study team will be able to answer any questions you have.

REGENERATE‑PD is an investigational Phase 2 study. The treatment being studied is not approved; and participation may not provide direct benefit.

We have an experienced surgical team and over 1,000 people have been safely and effectively treated using similar methods of drug delivery to the brain, which allows safe infusion of many types of drugs directly into the brain in a very precise and consistent manner that is tailored to each individual person’s anatomy. There are, however, risks to surgery. Less serious risks that are more likely to occur (in 1% to 10% of study participants) include bleeding at the incision site, bruising, skin infection, facial swelling, headache, pain or numbness at the incision site, and allergic or other reactions to medications given as part of surgical anesthesia—upset stomach, headache, or feeling tired. The less serious risks that are less likely include stuffy nose, nausea, infection of the surgical wounds, and minor bleeding or swelling in the brain along the path of the infusion catheter causing minimal or temporary symptoms such as difficulty swallowing, hoarseness, or weakness in the arms or legs. There are additional rare but more serious risks associated with the surgical procedure. All risks will be reviewed with your study team in detail in advance.

Travel costs to the study location for you and your care partner, study treatment, and all procedures required by the study will be provided to you at no cost.

You will only begin the steps of the study once you have had the chance to thoroughly discuss the study details and have all your questions addressed. This is the process of informed consent. Even after you give your consent and even after you start any part of the study, you may change your mind. You just need to inform the study team who will handle this together with you.