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Moderna and the Institute for Life Changing Medicines Announce a New Collaboration to Develop an mRNA Therapeutic for Ultra-Rare Disease, Crigler-Najjar Syndrome Type 1
Moderna to provide investigational mRNA CN-1 therapy free of charge CAMBRIDGE, Mass. & STAMFORD, Conn.--(BUSINESS WIRE)-- Moderna, Inc., (Nasdaq: MRNA) a
About this update from Moderna, Inc.
[{"type":"text","content":"\nModerna to provide investigational mRNA CN-1 therapy free of charge\n\n CAMBRIDGE, Mass. & STAMFORD, Conn.--(BUSINESS WIRE)--\nModerna, 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.\n\nUnder 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.\n\nCrigler-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.\n\nCurrent standard of care treatments rely on phototherapy treatments of up to 12 hours a day throughout life. The only definitive treatment is liver transplant that is associated with its own set of side effects and risk of death.\n\n“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,” said Stéphane Bancel, Chief Executive Officer of Moderna. “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. In this innovative partnership, the Institute for Life Changing Medicines will not pay Moderna an upfront fee or any downstream payments. Moderna will also provide the mRNA-3351 mat...