Rare Daily Staff

Moderna and the nonprofit Institute for Life Changing Medicines entered a collaboration to develop a new mRNA therapeutic for Crigler-Najjar syndrome type 1, an ultra-rare disease with the goal of making a therapy 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. It is estimated that there are only approximately 70-100 known cases of CN-1 in the world.

Current standard of care treatment relies on phototherapy of up to 12 hours a day throughout life. The only definitive treatment is liver transplant, which is associated with its own set of side effects and risk of death.

Moderna’s mRNA-3351 encodes for the human UGT1A1 and is designed to restore the missing or dysfunctional proteins that cause Crigler-Najjar syndrome type 1. The proposed therapeutic uses the same proprietary LNP formulation as Moderna’s antibody against chikungunya virus (mRNA-1944), propionic acidemia (mRNA-3927), and methylmalonic acidemia (mRNA-3704) programs. mRNA-3351 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration.

“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. “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.”

The Institute for Life Changing Medicines was set up to identify, develop, and promote access to life changing medicines that are designed to treat small populations of patients and are therefore not considered commercially viable. The organization’s pipeline of next generation medicines includes cell and gene therapies for the treatment of rare diseases.

“Children with this disease can be stigmatized for being severely jaundiced while living in fear of experiencing a life-threatening episode of kernicterus,” said James Wilson, co-founder and chief scientific officer of the Institute and director of the Orphan Disease Center, Perelman School of Medicine, University of Pennsylvania. “The mRNA therapeutic platform of Moderna has the potential to not only treat but to prevent these lethal metabolic crises. This partnership with Moderna could serve as a model for developing life changing medicines for those living with rare diseases when traditional business models for drug development fall short.”

Photo: James Wilson, co-founder of the Institute for Life Changing Medicines and Co-founder and chief scientific officer of the Institute and director of the Orphan Disease Center, Perelman School of Medicine, University of Pennsylvania

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