Precision BioSciences, Inc. announced on January 9, 2025, that its partner iECURE reported positive clinical efficacy and safety data from the first patient dosed with ECUR-506 in the Phase 1/2 OTC-HOPE study. ECUR-506 utilizes a PCSK9-specific ARCUS nuclease, licensed from Precision, to insert a functional copy of the OTC gene.
Treatment with ECUR-506 resulted in a complete clinical response from three months post-exposure to the end of the six-month study period. The infant no longer required ammonia scavenger medication and increased mean daily protein intake to age-appropriate levels, with mean ammonia levels remaining within normal limits.
ECUR-506 was generally well tolerated, with no significant clinical safety concerns apart from asymptomatic transaminitis at four weeks, which resolved with immunosuppressive therapy. These results provide initial clinical validation for the ARCUS platform's gene insertion capabilities, increasing confidence in its therapeutic potential for Precision's wholly-owned programs.
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