Moderna and ILCM Announce Collaboration to Develop mRNA Therapeutic for CN-1

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Moderna announced a collaboration to develop a new mRNA therapeutic for Crigler-Najjar Syndrome Type 1 at no cost to patients.

Moderna announced in a Sept. 7, 2021, press release a collaboration to develop a new mRNA therapeutic (mRNA-3351) for Crigler-Najjar Syndrome Type 1 (CN-1). 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 agreement, Moderna will license mRNA-3351 to ILCM with no upfront fees and without any downstream payments. ILCM will oversee the clinical development of mRNA-3351, including initiating clinical studies of mRNA-3351 in 2022.

It is estimated that there are only approximately 70-100 known cases of CN-1 in the world. Current standard of care treatments for CN-1 rely on phototherapy treatments of up to 12 hours a day throughout life. The only definitive treatment is a liver transplant.


“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, CEO of Moderna, in a company press release. “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 material free of charge. mRNA-3351 is a therapeutic candidate for Crigler-Najjar Syndrome Type 1 and our goal is to bring new hope for patients and their families.”

Source: Moderna