Rocket Presents Results of RP-L201 in the P-II Trial for the Treatment of Severe Leukocyte Adhesion Deficiency-I at ASGCT 2022
Shots:
- The P-II trial evaluates the safety & efficacy of RP-L201 in patients with sev. LAD-I
- At the data cut-off of Mar 2022, the results showed a sustained CD18 restoration & expression on ≥10% of neutrophils in all 9 patients aged 5mos. to 9yrs. @ 3 to 24mos. of follow-up after RP-L201 infusion, 100% OS @1yr. post-infusion via Kaplan, reduction in the rate of all-cause hospitalizations & sev. inf., resolution of LAD-I-related skin rash & restoration of wound repair capabilities along with sustained phenotypic correction
- The safety profile was favorable in all patients with no RP-L201-related serious AEs. The company is expected to file an application in H1’23, following discussions with health authorities
Ref: Businesswire | Image: Rocket
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