Ornithine-transcarbamylase deficiency is a genetic disorder which causes the accumulation of excess ammonia in the blood. Ammonia is the residual product of metabolism of protein, which is toxic in high concentration. Lack of energy, unwilling to eat, poorly controlled body temperature or breathing rate, pyloric stenosis, splenomegaly, cerebral edema, hepatic failure, and aminoaciduria are the observed symptoms of the disease. The complications associated with ornithine-transcarbamylase deficiency are intellectual disability, developmental delays, liver damage, skin lesions, and brittle hair. Ornithine-transcarbamylase deficiency generally occurs due to mutation in OTC gene which helps in synthesis of enzyme, ornithine transcarbamylase. Dimension Therapeutics, Inc. is in the process of developing scAAV8OTC gene therapy for the treatment of ornithine transcarbamylase deficiency. Translate Bio, Inc. is in the process of developing OTC drug candidate for the treatment of ornithine transcarbamylase deficiency. Some of the companies having the pipeline of ornithine transcarbamylase deficiency include Translate Bio, Inc., Dimension Therapeutics, Inc., and others.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licensing, grants, technology, and others.