IECURE, INC. | MedPath

iECURE's Gene Insertion Therapy ECUR-506 Shows Complete Clinical Response in OTC Deficiency Trial

4 months ago

• iECURE's ECUR-506, an in vivo gene insertion therapy, demonstrated a complete clinical response in an infant with neonatal-onset Ornithine Transcarbamylase (OTC) deficiency. • The Phase 1/2 OTC-HOPE study showed that ECUR-506 was generally well-tolerated, with no significant clinical safety concerns, offering potential lasting benefits. • Treatment allowed discontinuation of ammonia scavenger medication and increased protein intake to age-appropriate levels, maintaining normal ammonia levels for six months. • Precision BioSciences' ARCUS gene editing platform, utilized in ECUR-506, shows promise for treating genetic diseases, boosting confidence in its therapeutic potential.