Moderna is an American pharmaceutical and biotechnology company that focuses on developing vaccines using mRNA technology. Most of us know the company for the Covid-19 vaccine, but it was actually founded back in 2010. Yesterday, the company and the non-profit Institute for Life-Changing Medicines (ILCM) announced their collaboration to create a new therapy for the Crigler-Najjar Syndrome Type 1 (CN-1). The purpose of the union is to provide free of charge therapy (mRNA-3351), under ILCM license, for those who suffer from CN-1. This means that ILCM will initiate clinical development and trials by next year.
What is CN-1 Syndrome?
According to the U.S National Library of Medicine, the Crigler-Najjar syndrome is a severe condition that affects 1 in 1 million newborns. Those suffering from it will have high bilirubin levels in the blood that the liver cannot process. Also known as hyperbilirubinemia. Type -1 is the most severe, and many people die from it during childhood. The condition is caused by a mutation known as UGT1A1, and symptoms can become visible after birth.
What are current treatments, and what will the new collaboration offer?
The current treatments use phototherapy, and sometimes those who suffer from the condition need to spend up to 12 hours a day receiving it. Others have tried a more definite treatment, which is a liver transplant.
The union between Moderna and ILCM seeks to offer CN-1 patients a new mRNA therapy, and it will be given free of charge to anyone who needs it. mRNA technology can be used to develop treatments for severe illnesses and prevent lethal metabolical crises. The mRNA-3351 treatment will use human UGT1A1 and prevent the syndrome. It will work similarly to the already developed mRNA therapies against the chikungunya virus, methylmalonic academia and propionic academia.