iECURE announced encouraging early results from its Phase 1/2 OTC-HOPE clinical trial evaluating ECUR-506, a gene editing therapy for ornithine transcarbamylase (OTC) deficiency. The first infant treated at the lowest dose level showed a complete clinical response, achieving successful discontinuation of ammonia scavenging medications and normalization of protein intake after three months. These improvements were sustained throughout the six-month observation period.
The treatment was well-tolerated overall, with the only reported adverse event being asymptomatic transaminitis detected four weeks post-treatment, which resolved within four weeks with immunosuppressive therapy.
The OTC-HOPE trial is actively enrolling patients in the U.S., U.K., Spain, and Australia, with a full data readout expected in the first half of 2026. ECUR-506 is designed to treat neonatal-onset OTC deficiency, a rare genetic disorder that affects over 1,000 newborns worldwide each year.
The dual AAV vector approach, incorporating Precision BioSciences' ARCUS® technology, represents an advanced gene insertion strategy. By precisely targeting the PCSK9 locus for OTC gene insertion, this method aims to achieve sustained therapeutic protein expression—a critical factor for the long-term effectiveness of treatment in OTC deficiency.
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#AAV #genetherapy #ornithinetranscarbamylase #rarediseases #dualAAV
Today, we reported preliminary findings from the first infant dosed in our ongoing OTC-HOPE Phase 1/2 #ClinicalStudy of ECUR-506, our #GeneEditing candidate designed to treat neonatal onset ornithine transcarbamylase (#OTC) deficiency. Treatment with ECUR-506 at the lowest dose level was generally well tolerated in this infant and achieved a complete clinical response, as of 6 months post-exposure, as demonstrated by the removal of ammonia scavenging medicines followed by the absence of hyperammonemic crises and normalization of protein intake.
We are encouraged by the tremendous potential of ECUR-506 and look forward to continuing enrollment in our OTC-HOPE Phase 1/2 trial worldwide, with a full data readout expected in the first half of 2026.
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