Moderna has agreed to license the mRNA-3351 to ILCM free of cost, involving no upfront fees, and any downstream payments


Moderna, ILCM partner to develop CN-1 treatment. (Credit: Furiosa-L from Pixabay.)

Moderna has teamed up with the non-profit Institute for Life Changing Medicines (ILCM) to develop mRNA-3351, its new mRNA therapeutic for Crigler-Najjar Syndrome Type 1 (CN-1).

The collaboration is aimed at developing an mRNA therapy for the treatment of ultra-rare disease CN-1 and offering the medication to patients at no cost.

Under the terms of the agreement, Moderna will license the mRNA-3351 to ILCM free of cost, involving no upfront fees, and any downstream payments.

ILCM has agreed to carry out the clinical development of mRNA-3351, with plans to commence clinical trials of mRNA-3351 from next year.

CN-1 is an ultra-rare genetically inherited disorder, caused by the mutation in the UGT1A1 gene, with only around 70-100 known cases in the world.

The syndrome occurs when an enzyme that converts liver secretion bilirubin into a form that can be easily removed from the body, fails to work properly.

It results in the accumulation of bilirubin in the body and may lead to jaundice and damage to the brain, muscles and nerves, which can be life-threatening.

Moderna CEO Stéphane Bancel said: “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.

“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.”

The current standard of care treatment is limited to phototherapy, and the only definitive treatment is a liver transplant, which is linked to side effects and risk of death.

The biotechnology company said that its mRNA-3351 encodes for the human UGT1A1 and is designed to restore the missing or dysfunctional proteins that cause the disease.

mRNA-3351 leverages the same LNP formulation used in Moderna’s antibody against chikungunya virus, propionic acidemia, and methylmalonic acidemia programmes.

The US Food and Drug Administration (FDA) has granted Rare Paediatric Disease designation for mRNA-3351

ILCM co-founder and CEO Alex Karnal said: “We are energised to collaborate with Moderna to advance an mRNA for treating Crigler-Najjar.

“At the Institute, we believe that a healthy life is a basic human right, and we are fighting to improve the lives of all people suffering from less common but still devastating diseases.

“We are grateful for Moderna’s support of our novel non-profit business model by providing our first medicine aimed at transforming the lives of people suffering from Crigler-Najjar syndrome around the world.”