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.
Rocket Pharmaceuticals, Inc. is committed to advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. After careful and deliberate review of the considerations governing expanded access, we have made the determination that our clinical trials are the most appropriate way to access our investigational gene therapy products at present. For this reason, we do not currently offer an expanded access program (EAP). As a commitment to patients who might potentially benefit from our investigational products, we will continue to review and re-evaluate this policy and will provide ongoing updates on our website as additional clinical study results become available.