Clinical Trials

We are developing PR001, a potentially disease-modifying, single-dose gene therapy, for patients with Parkinson's disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease.
Stage: Phase 1/2 clinical trial
Trial Status: Recruiting
Locations: USA

For more information visit

Type 2 Gaucher Disease
Stage: Phase 1/2 clinical trial
Trial Status: Recruiting
Locations: USA

For more information visit

We are developing PR006, a potentially disease-modifying, single-dose gene therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).
Stage: Phase 1/2 clinical trial
Trial Status: Recruiting
Locations: USA

For more information visit

We are also developing a pipeline of therapies for patients with other neurodegenerative diseases.

Our clinical studies will be the primary route by which patients receive access to our investigational drug(s). We intend to post information related to our ongoing applicable clinical trials on If you have questions about Prevail’s clinical trials, please write to

Our Expanded Access and Compassionate Use Policy

Prevail is committed to developing safe and effective medicines and treatments for patients worldwide.  Before marketing drugs to patients, Prevail designs and conducts clinical trials to generate valid scientific evidence and to protect the rights, safety, and welfare of research participants.

Enrolling in Prevail’s clinical trials is the primary way that patients who seek access to Prevail’s investigational drug(s) may receive those drug(s).  Information about Prevail’s ongoing clinical trials can be found on by searching “Prevail Therapeutics” in the “sponsor” field under the heading “Advanced Search.”

“Expanded access” and “compassionate use” are processes through which a patient and the patient’s physician may seek to obtain an investigational product outside of a clinical trial. These programs only apply to medicines not yet approved by the national drug regulatory authority in the country of the patient’s residence.

Prevail evaluates expanded access and compassionate use requests on an individual basis. The decision-making criteria Prevail applies when considering whether to grant expanded access or compassionate use requests (“Program Requests”) are listed below.  Prevail’s decision to grant or deny a Program Request may be modified or reversed by Prevail at any time, and is subject to Prevail’s continuing evaluation of safety and effectiveness data about the investigational drug, as well as other considerations related to sustainability, inventory, manufacturing, and clinical development programs.  Prevail may revise this policy at any time.

Request Process

Requests for access to an investigational drug on an expanded access or compassionate use basis must be made in writing and signed and submitted by a licensed physician who is in a treatment relationship with the patient for whom the investigational drug is being requested.  These requests should be sent to  If a patient submits a Program Request to Prevail directly, Prevail will respond to the patient asking him or her to have his or her physician submit the request.

Prevail will make reasonable efforts to acknowledge receipt of the Program Request within five (5) business days. All information submitted as part of a request will be maintained in confidence and used solely for the purpose of evaluating a patient’s eligibility for expanded access or compassionate use, as applicable.

Prevail intends to provide a written response to all Program Requests in a timely manner, but the time required may vary on a case-by-case basis due to a variety of factors.  Decisions regarding Program Requests are made in Prevail’s sole discretion, based on the criteria below.

Decision Criteria

When evaluating a Program Request, Prevail will consider whether:

  • The patient has a serious or life-threatening disease or condition, as represented by the requesting physician and otherwise confirmed through medical records;
  • The patient is ineligible or otherwise unable to participate in any clinical trial of the Prevail investigational drug or of any other investigational drug for that patient’s condition;
  • There are alternative treatment options available and/or whether such treatment options have been used;
  • Clinical data are available to assess the potential benefits and risks in providing the investigational drug for treatment, and the potential benefits to the patient justifies the potential risks of the treatment use;
  • The use of the investigational drug will be monitored and controlled appropriately and adequately by a licensed physician who is qualified to administer the investigational drug in a safe manner and who practices at a facility that has the capability, including adequate equipment and personnel, to provide any care for the patient needed in connection with the administration of such drug;
  • With the possible granting of the request for the investigational drug, there would remain sufficient supply of investigational drug for ongoing and planned clinical trials;
  • The granting of the request would affect or interfere with the initiation, conduct, or completion of clinical trials, or otherwise compromise the potential development and/or commercialization of the investigational drug; and
  • The Program Request, and Prevail’s grant of the request, would be compliant with applicable local, state/provincial, and federal/national laws and regulations, as well as industry standards.

Patient Inquiries

Our therapies are not yet available to patients. We are working to bring forward these potential medicines as quickly as possible.

If you have questions about Prevail’s expanded access policy or clinical trials, please write to Prevail will try to acknowledge receipt of your message within five (5) business days of receiving your question, but it may take longer.