PHILADELPHIA, May 06, 2025--iECURE to Present Initial OTC-HOPE Clinical Data of ECUR-506 in Ornithine Transcarbamylase ...

Gene editing company iECURE has said data from its Phase I/II ornithine transcarbamylase (OTC) deficiency trial could suggest partial restoration of functional OTC. New results from the company ...

N-acetylglutamate synthetase deficiency; OTC, ornithine transcarbamylase deficiency; PCD, pyruvate carboxylase deficiency; PA, propionic acidemia; THAN, transient hyperammonemia of the newborn ...

self-amplifying mRNA vaccine for COVID-19 and an mRNA replacement therapy for ornithine transcarbamylase (OTC) deficiency (Fig. 1). Preclinical programs include wholly owned mRNA therapeutics for ...

ornithine transcarbamylase, or argininosuccinic acid synthetase, including neonatal-onset deficiency and late-onset disease with a history of hyperammonemic encephalopathy. <20kg: use powder.

Community members are invited to join in a day of fun, games, and giving back at the Brave the Odds Bean Bag Tournament and Bingo Bash this Sunday, May 4, from 1 to 4 p.m. at Squealers in Tea.The ...

The OTC-HOPE study is a Phase 1/2 first-in-human clinical trial of ECUR-506 in baby boys with genetically confirmed neonatal onset OTC deficiency and has been cleared to evaluate ascending dose ...

ECURE, Inc., a gene editing company focused on the development of mutation-agnostic in vivo gene insertion therapies for the treatment of liver disorders with significant unmet need, today announced ...