Audentes is exploring opportunities to outlicense the Crigler Najjar program to continue to advance this important gene therapy. If you have been involved in the LUSTRO or VALENS clinical studies, please contact your clinical trial site for more information.
About Crigler-Najjar Syndrome
Crigler-Najjar Syndrome (CN) is a rare, congenital autosomal recessive monogenic disease characterized by severely high levels of bilirubin in the blood and risk of irreversible neurological damage and death. CN is estimated to affect approximately one in 1,000,000 newborns. Infants with CN develop severe jaundice shortly after birth resulting in rapid presentation and diagnosis. CN is caused by mutations in the gene encoding the UGT1A1 (uridine-diphosphate (UDP)-glucuronosyltransferase (UGT) 1A1) enzyme resulting in an inability to convert unconjugated bilirubin to a water-soluble form that can be excreted from the body.
Clinical diagnosis is confirmed via genetic testing of the UGT1A1 gene. The current standard of care for Crigler-Najjar Syndrome is aggressive phototherapy for more than ten to 12 hours per day using intense LED or fluorescent light focused on the bare skin, while the eyes are shielded. Phototherapy speeds bilirubin decomposition and excretion, lowering serum bilirubin levels. Phototherapy wanes in effectiveness beginning around age four due to thickening of the skin and a reduction in surface area to body mass ratio, and a liver transplant may be required for survival.
For more information about Crigler-Najjar Syndrome, please visit the links below.
Community Support & Resources
Where can you find support from the Crigler Najjar community?
Below are organizations dedicated to helping the Crigler Najjar patient community around the world. Each of them offers something unique.