The P-I/II (MGT009) dose-escalation study evaluated botaretigene sparoparvovec in 49 patients aged ≥5yrs. with XLRP associated with RPGR gene
The results showed an acceptable safety profile & efficacy, improvement in retinal sensitivity, visual function & functional vision, and improvement in walk time @26wk. b/w the treated eyes in low & intermediate dose cohorts & untreated eyes in the randomized concurrent control arm at low illumination levels. The safety profile was consistent with prior reports, AE was anticipated & manageable with no dose-limiting events
The company also presented the results from the P-I study of 3 doses of JNJ-1887 for dry AMD with GA which met the 1EPs of safety over 2yr. follow-up period & continual decline in lesion growth over 6mos.
Ref: Janssen | Image: Janssen
