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
