Clinical Trials:

Pyruvate Kinase Deficiency (PKD)

PKD Clinical Trials Resources

RP-L301 is currently being developed for PKD, a rare, genetic blood disorder characterized by excessive rupture of red blood cells, resulting in frequent, chronic anemia that may be severe or life-threatening for some patients.

A single arm, open-label, global Phase 2 trial to evaluate the efficacy and safety of RP-L301 for patients with PKD is being initiated. The trial is expected to enroll 10 patients between the ages of 8 and 55 years of age with a confirmed PKLR gene mutation and documented anemia who have undergone a prior splenectomy at the following sites:

Interested in learning more?
Visit our Patients & Caregivers page or email us at

For more details about the trial, visit

To read our Expanded Access statement, click here.

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