Clinical Trials:
Leukocyte Adhesion
Deficiency-I (LAD-I)
Deficiency-I (LAD-I)

RP-L201 is being developed for severe Leukocyte Adhesion Deficiency Type I (LAD-I), a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant (HSCT).
RP-L201 is an investigational gene therapy that contains autologous (patient-derived) hematopoietic stem cells (HSCs) that have been genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene.
The open-label, single-arm, global Phase 1/2 registration-enabling clinical trial of RP-L201 (ClinicalTrials.gov ID: NCT03812263) assessed the safety and efficacy of RP-L201 in pediatric patients with severe LAD-I. The primary efficacy end point of the phase 2 study was survival without allogeneic HSCT (HSCT-free survival) at least one year after RP-L201 infusion and at two years of age among the patients who were younger than one year of age at enrollment.
Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@nullrocketpharma.com.
For more details about the trial, visit clinicaltrials.gov.
To read our Expanded Access statement, click here.