Moderna to develop mRNA therapeutic for very rare disease

By Jane Byrne

- Last updated on GMT

© GettyImages/niphon
© GettyImages/niphon

Related tags mRNA therapeutics Moderna Rare disease

Moderna and the nonprofit Institute for Life Changing Medicines (ILCM) are collaborating to develop a new messenger RNA (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, said the US biotech.

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.


mRNA-3351 encodes for the human UGT1A1 and is designed to restore the missing or dysfunctional proteins that causes CN-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.

The treatment has been granted rare pediatric disease designation by the US Food and Drug Administration (FDA).

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.

Current 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.

James M Wilson, cofounder and CSO, 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.”


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