Infantile Malignant Osteopetrosis
RP-L401 is currently being developed for IMO, a severe bone disorder that affects children and prevents normal bone growth. The bone deficiencies cause bone marrow failure, vision and hearing loss, and other complications and frequently results in death during the early years of life.
RP-L401 is currently being evaluated in a non-randomized, open-label Phase 1 clinical trial which will enroll two pediatric patients, one month of age or older. The trial is designed to assess safety and tolerability of RP-L401 as well as preliminary efficacy.