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PHILADELPHIA, PA — iECURE’s experimental gene-editing therapy reduced the annualized rate of life-threatening metabolic crises by 74% in the low-dose cohort of an early-stage trial in infants with severe ornithine …
iECURE Gene Therapy Cuts Severe Metabolic Crises in Trial Read More PHILADELPHIA, PA — iECURE said new data from its ongoing OTC-HOPE clinical trial showed sustained clinical benefit in the first infant treated with its experimental gene-editing therapy ECUR-506 and statistically …
iECURE Reports Early Clinical Gains in OTC Gene Therapy Trial Read More PHILADELPHIA, PA — iECURE, Inc. reported preliminary clinical data showing its experimental gene-editing therapy reduced life-threatening metabolic crises in infants with severe ornithine transcarbamylase deficiency, offering early evidence of efficacy …
iECURE Gene Therapy Data Show Fewer Metabolic Crises Read More PHILADELPHIA, PA — iECURE announced that new clinical data from its ongoing OTC-HOPE trial evaluating ECUR-506 in neonatal-onset ornithine transcarbamylase deficiency will be presented this month at two medical conferences.
iECURE Reports Early OTC Gene Therapy Trial Results Read More PHILADELPHIA, PA — iECURE said the U.S. Food and Drug Administration has selected ECUR-506, the company’s investigational gene insertion therapy for neonatal-onset ornithine transcarbamylase deficiency, for the agency’s Chemistry, Manufacturing, …
FDA Selects iECURE Therapy for Manufacturing Readiness Pilot Program Read More PHILADELPHIA, PA — A rare-disease gene therapy developed in Philadelphia has cleared a major regulatory hurdle, positioning it on an accelerated path toward approval in the United States and the …
FDA Fast-Track Boost Lifts iECURE Gene Therapy Toward Hope for Newborns Read More PHILADELPHIA, PA — iECURE, Inc. will present new clinical data on its experimental gene therapy candidate, ECUR-506, at two international medical meetings in Kyoto, Japan, this September. The therapy is …
iECURE to Present New Data on Gene Therapy for Rare Metabolic Disorder at Global Conferences Read More PHILADELPHIA, PA — iECURE, Inc. recently unveiled encouraging early data from its ongoing OTC-HOPE Phase 1/2 study at the 2025 ACMG Annual Clinical Genetics Meeting. The study, which evaluates the …
iECURE Highlights Promising Results from OTC-HOPE Study at 2025 ACMG Genetics Meeting Read More PHILADELPHIA, PA — iECURE, Inc. has announced that early data from its Phase 1/2 OTC-HOPE clinical trial, evaluating the gene-editing candidate ECUR-506 for neonatal onset ornithine transcarbamylase (OTC) deficiency, will be …
iECURE to Present Promising Initial Data from Gene Editing Trial for Neonatal OTC Deficiency Read More