A 6.5-month-old boy with the rare inherited urea cycle disorder ornithine transcarbamylase (OTC) deficiency has responded positively in a targeted in vivo gene editing trial, in which a correct copy ...

paving the way for a Phase I/II clinical trial of ECUR-506 to treat Ornithine Transcarbamylase (OTC) deficiency. Known as the OTC-HOPE study, the first-in-human trial will enrol newborn boys aged ...

generated a complete clinical response in a Phase 1/2 trial for Ornithine Transcarbamylase (OTC) deficiency, a rare metabolic disorder. A therapeutic option that can address the accumulation of ...

The Recombinant DNA Advisory Committee (RAC), the National Institutes of Health office responsible for public oversight of gene therapy, met on 8–10 December to examine the clinical trial in ...

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

Arcturus’ pipeline includes RNA therapeutic candidates to potentially treat ornithine transcarbamylase (OTC) deficiency and cystic fibrosis (CF), along with its partnered mRNA vaccine programs ...