Leukocyte Adhesion Deficiency-I (LAD)
RP-L201 is being developed for LAD-I, a rare genetic immune disorder that predisposes patients to recurrent and fatal infections, often resulting in death within the first two years of life.
The Phase 2 portion of the clinical trial for RP-L201 is currently underway. The Phase 1/2 trial aims to enroll 9 patients is designed to assess the therapeutic safety and efficacy in pediatric patients with severe LAD-I. The primary endpoint in the Phase 2 study is overall survival.