Moderna donates ultra-rare disease therapy to nonprofit founded by late Takeda R&D chief

close-up of a microbiological pipette in the genetic laboratory
To help find a treatment for the ultra-rare disease, Moderna is offering mRNA-3351 for no upfront fees and no downstream payments. (science photo/Shutterstock)

Moderna is donating an ultra-rare disease mRNA therapeutic to the Institute for Life Changing Medicines (ILCM), the nonprofit founded by the late Tachi Yamada, M.D., to find therapies for devastating genetic diseases.

The new collaboration will see Moderna offer up an experimental treatment called mRNA-3351 for Crigler-Najjar syndrome Type 1, or CN-1. The genetically inherited disorder is extremely rare, with only about 70,000 known cases in the world. The condition is caused by a mutation in the UGT1A1 gene that prevents bilirubin from being broken down by the body, leading to jaundice and damage to the brain, muscles and nerves.

Patients with CN-1 are treated with 12-hour phototherapy sessions throughout life or with a liver transplant.

To help find a treatment for the ultra-rare disease, Moderna is offering mRNA-3351 for no upfront fees and no downstream payments. The institute will handle clinical development with plans to start clinical trials in 2022.

Moderna’s therapy encodes the UGT1A1 gene that is mutated in CN-1 to restore the missing or dysfunctional proteins that cause the condition. MRNA-3351, which uses the same formulation as the biotech’s chikungunya virus antibody, has already been granted a rare pediatric disease designation from the FDA.

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ILCM is a nonprofit that finds and develops medicines to treat small patient populations that would not be considered commercially viable for the traditional pharmaceutical industry. Among ILCM’s founders is Yamada, the life sciences investor and former R&D chief at Takeda who died in August.

MRNA-3351 will slide into a pipeline that includes gene therapies for Lesch-Nyhan syndrome and AADC deficiency.

Patients with Lesch-Nyhan syndrome experience kidney impairment, gout and self-mutilating behavior. ILCM’s therapy is currently in preclinical research. AADC deficiency causes a disruption in production of neurotransmitters, leading to problems with the nervous system including difficulty with movement, blood pressure control and heart rate. The nonprofit’s gene therapy for the condition is currently in phase 3 testing and ready to file for registration with the FDA.