Patient Trials

Expanded Access Policy

PYC Therapeutics is a clinical-stage biopharma company dedicated to the discovery and development of RNA Therapeutics for rare inherited diseases. Our lead program is in Ophthalmology, to treat Retinitis Pigmentosa type 11 with our proprietary molecule VP-001. We have recently received Fast Track Designation for this program from the US FDA. We are committed to conducting clinical trials to evaluate the safety and efficacy of VP-001 to obtain the necessary marketing approvals that allow physicians to prescribe them so patients may have broad access.


In some circumstances, a treating physician may make a request to provide an unapproved therapy to a qualifying individual, which must be approved by the U.S. Food and Drug Administration (FDA) and supported by the pharmaceutical manufacturer. This is called an expanded access program (EAP), sometimes known as “compassionate use,” and refers to a potential pathway for a patient with a serious or life-threatening disease or condition to try an investigational therapy outside of a clinical trial when there is no comparable or satisfactory therapy available.


While we understand that some patients may wish to access our unapproved investigational drugs, our goal is to provide access to our medicines at the appropriate time and in a manner that is most beneficial to the relevant patient population. At this stage of our development, we do not have sufficient evidence of clinical safety and efficacy of our investigational drug, VP-001, to support an expanded access program. Therefore, PYC Therapeutics does not currently offer an expanded access program. We believe enrollment in our ongoing clinical trials is the safest and most effective way of achieving this goal and we encourage all patients and physicians who are interested in accessing our investigational medicines to visit the website to find out about enrolling.


PYC Therapeutics may revise this expanded access policy at any time. Additionally, the posting of this policy by PYC does not serve as a guarantee of access to any specific investigational new drug by any individual patient.
If you have any questions, please reach out to us at

Expressions of Interest

Expressions of Interest

We believe in the importance of collaborating closely with patients, their families, patient advocacy organisations, physicians, researchers and regulatory authorities to help achieve our goals together.

Treating physicians, patients and/or caregivers interested in learning more about PYC Therapeutics’ investigational product currently undergoing clinical studies can find more information at

General information, please contact

Register your interest