Clinical Trials:

Fanconi Anemia

FA Clinical Trials Resources

RP-L102 is being developed for FA, a rare DNA repair disorder characterized by bone marrow failure, a predisposition to cancer and often death before the age of 40.

An open-label, global registrational clinical trial of RP-L102 for the treatment of FA has completed enrollment. The trial aims to enroll 12 patients total from the U.S. and EU. The primary endpoint of the trial, which has been agreed upon by the FDA and EMA is mitomycin-C (MMC) resistance, a measure of bone marrow cell correction.

For more information about FA, visit our Patients and Community page or email us at

For more details about the trial, visit

To read our Expanded Access statement, click here.

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