PHILADELPHIA, May 06, 2025--(BUSINESS WIRE)--iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion therapies for the treatment of liver disorders ...
(RTTNews) - Precision BioSciences, Inc. (DTIL), a clinical-stage gene editing company, on Thursday, announced positive clinical efficacy data from its partner iECURE in the Phase 1/2 OTC-HOPE study ...
PHILADELPHIA, March 18, 2026--iECURE, Inc., a clinical-stage genome editing company developing variant-agnostic in vivo ...
PHILADELPHIA--(BUSINESS WIRE)--iECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver ...
Clinical trial sites in the United States, United Kingdom, and Australia will evaluate ECUR-506, an investigational gene editing-based therapy, in newborn males with neonatal onset Ornithine ...
ECUR-506 is an investigational gene editing therapy that utilizes an adeno-associated virus to deliver a functional copy of the OTC gene. The Food and Drug Administration (FDA) has granted Fast Track ...
iECURE's ECUR-506 showed promising safety and efficacy in a Phase 1/2 study for neonatal-onset OTC deficiency, with no major safety issues. BMO Capital Markets upgraded Precision BioSciences to ...
First participant dosed achieved complete clinical response per study protocol and data suggests partial restoration of functional OTC enzyme activity in the liver PHILADELPHIA--(BUSINESS WIRE)-- ...
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 ...
OTC deficiency is a rare genetic condition and the most prevalent urea cycle disorder. Newborns with neonatal onset OTC deficiency exhibit hyperammonemia symptoms shortly after birth, including ...
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