Furthermore, the team succeeded in editing genes to replicate ornithine transcarbamylase deficiency, a genetic disorder disrupting the urea cycle. This achievement advances methods for modeling ...

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

Christian Plank in 2009. Its lead candidate is ETH-OTCD, for Ornithine transcarbamylase deficiency, a rare urea cycle disorder caused by a single defective enzyme. One of the biggest obstacles for ...

The company is developing ARCT-810 (LUNAR-OTC), a mRNA-based therapeutic candidate, which is in Phase 2 clinical trial for treating ornithine transcarbamylase deficiency; and ARCT-032 (LUNAR-CF), a ...

ornithine transcarbamylase deficiency, phenylketonuria, crigler-najjar syndrome type 1, and cystic fibrosis. It has strategic alliances and collaborations with AstraZeneca; Merck & Co., Inc; Vertex ...

Avalotcagene Ontaparvovec is a gene therapy commercialized by Ultragenyx Pharmaceutical, with a leading Phase III program in Ornithine-Transcarbamylase Deficiency. According ...