Crigler-Najjar syndrome is a severe condition, characterized by the high concentration of bilirubin (a toxic substance) in blood. Bilirubin is produced by liver due to the breakdown of red blood cells. The toxic form of bilirubin (unconjugated bilirubin), is converted into nontoxic form (conjugated bilirubin). Patients suffering with Crigler-Najjar syndrome have a high level of unconjugated form of bilirubin in blood. Due to its high concentration, kernicterus, a form of brain damage can occur. Kernicterus can further lead to intellectual disability, choreoathetosis, and hearing problems. Audentes Therapeutics Inc. is in the process of developing AT342 as a gene therapy for the treatment of Crigler-Najjar syndrome. Also, Genethon, involved in the designing of gene therapy products for rare diseases is also in the process of developing an in-vivo gene therapy/AAV for the treatment of Crigler-Najjar syndrome. Other than this International Stem Cell Corporation is also involved in Crigler-Najjar syndrome pipeline.
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.