Moderna, Inc., (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines and the nonprofit Institute for Life Changing Medicines (ILCM), today announced a new collaboration to develop a new mRNA therapeutic (mRNA-3351) for Crigler-Najjar Syndrome Type 1 (CN-1), an ultra-rare disease. It is estimated that there are only approximately 70-100 known cases of CN-1 in the world. The goal of the collaboration is to make an mRNA therapy for the treatment of CN-1 available at no cost to patients.
Under the terms of the agreement, Moderna will license mRNA-3351 to ILCM with no upfront fees, and without any downstream payments. ILCM will be responsible for the clinical development of mRNA-3351. ILCM plans to initiate clinical studies of mRNA-3351 in 2022.
Crigler-Najjar Syndrome Type 1 (CN-1) is an ultra-rare genetically inherited disorder caused by the mutation in the UGT1A1 gene in which bilirubin, a substance made by the liver 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 to the brain, muscles and nerves. The symptoms become apparent shortly after birth and can be life-threatening.
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