Patient Trials

Patient Trials

Retinitis Pigmentosa Type 11 (RP11)


VP-001 Phase 1 trial


Trial Status: Recruiting
Location: United States

Contact For Participation:

PYC is conducting a phase 1 open-label study of RP11 Patients who have a genetic mutation in the PRPF31 gene. The primary objective of the study is to assess the safety and tolerability of VP-001. Patients who participated in the RP11 Natural History Study are eligible to participate in this trial.

If you are interested in learning more about this clinical trial, visit for a list of active sites and contact the site’s study coordinator for more information.


Retinitis Pigmentosa Type 11 (RP11) Natural History Study


Trial Status:  Enrolling
Location: United States, Western Australia

Contact For Participation:

PYC is conducting a study to characterise the natural history the systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, or RP11.

This is a non-interventional trial with assessments that will be completed to measure and evaluate structural and functional visual changes including those impacting patient quality of life associated with this inherited retinal condition and observing how these changes evolve over time.

Results of this 4 year study will be utilised in conjunction of the VP-001 clinical trials to asses efficacy. If you are interested in learning more about this clinical trial, visit for a list of active sites and contact the site’s study coordinator for more information.

We believe in the importance of collaborating closely with patients, their families, patient advocacy organizations, 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


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