Moderna to develop mRNA therapeutic for ultra-rare disease

Moderna and non-profit firm Institute for Life Changing Medicines have partnered to develop mRNA-3351 for Crigler-Najjar Syndrome Type 1.

Under the terms of the agreement, messenger RNA (mRNA) biopharma company Moderna will licence mRNA-3351 to the Institute for Life Changing Medicines (ILCM) with no upfront fees or downstream payments.

ILCM will be responsible for clinically developing mRNA-3351 and anticipates clinical studies to begin in 2022.

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mRNA-3351 uses the same lipid nano particle (LNP) formulation as the firm’s antibody against chikungunya virus mRNA-1944 and is designed to create the missing or dysfunctional proteins that causes Crigler-Najjar Syndrome Type 1 (CN-1).

CN-1 is an extremely rare genetically inherited illness that is caused by mutation in the UGT1A1 gene. The disorder occurs when bilirubin, a substance made by the liver, is unable to be broken down and what normally changes bilirubin into a form that can be removed from the body does not function properly. The absence of this enzyme can cause bilirubin to build up and potentially lead to damage to the brain, muscles, nerves, and jaundice.

There are only 70-100 identified cases of CN-1 in the world and patients rely on phototherapy treatments, which can take up to 12 hours a day. The only conclusive treatment option is a liver transplant, which of course comes with risks.

“Ultra-rare diseases are always a challenge for the industry given the very small number of patients who could benefit from the medicine,” a spokesperson for Moderna told us.

“Moderna believes that with the potential of its science comes a responsibility to the many patients our technology could help, regardless of whether they have a disease shared by millions, or one that is unique to them alone. As ILCM co-founder and chief scientific officer James Wilson suggested, this partnership 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.”

mRNA-3351 has been given Rare Pediatric Disease designation by the US Food and Drug Administration (FDA.)