Crigler-Najjar Syndrome Type 1 is an ultra-rare disease where treatment relies on a liver transplant or full-day phototherapy sessions.
Moderna said this week it is donating a new mRNA therapy (mRNA-3351) to a nonprofit to develop as a treatment for Crigler-Najjar syndrome Type 1 (CN-1), an ultra-rare disease.
Under the terms of the agreement, Moderna will license mRNA-3351 to the Institute for Life Changing Medicines (ILCM) with no up-front fees and without any downstream payments.
The aim of the collaboration is to make an mRNA therapy for the treatment of CN-1 available at no cost to patients. It is estimated that there are only 70 to 100 known cases of CN-1 in the world.
The ILCM, which will be responsible for the clinical development of mRNA-3351, plans to start trials next year.
CN-1 is an ultra-rare genetically inherited disorder caused by mutation in the UGT1A1 gene in which bilirubin cannot be broken down. The syndrome occurs when the protein that normally converts bilirubin into a form that can be easily removed from the body does not work properly. Without this enzyme, bilirubin can build up in the body and lead to jaundice and damage of the brain, muscles, and nerves. The symptoms become apparent shortly after birth and can be life threatening.
Current standard of care is either phototherapy treatments for up to 12 hours a day for life or a liver transplant.
The ILCM says it "identifies, develops, and promotes access to life-changing medicines that are designed to treat small populations of patients and are therefore not considered commercially viable." Its other focus areas are aromatic l-amino acid decarboxylase deficiency and Lesch-Nyhan syndrome.
“At Moderna, we believe that mRNA therapies have the potential to profoundly impact rare disease patients and their families. Ultra-rare diseases are always a challenge for our industry given the very small number of patients who could benefit from the medicine,” Stéphane Bancel, Moderna’s CEO, said in a statement. “We decided that rather than charge a high price for the medicine candidate, which is not aligned with our values, we would rather give it away for free.”