While mRNA mostly became known during the pandemic because of its use in COVID-19 vaccines, the technology had been investigated as a therapeutic long before then — and it was hoped to have particular potential for rare diseases.
“I think there’s great potential for treating monogenic diseases” with mRNA therapies, said P.J. Brooks, PhD, acting director of the Division of Rare Diseases Research Innovation at the National Center for Advancing Translational Sciences at the NIH. “That’s what you’re seeing with the propionic acidemia and methylmalonic acidemia trials.”
This month, Vertex announced that the FDA approved its investigational new drug application for an inhaled mRNA therapy for cystic fibrosis. The company, which is developing the treatment in partnership with Moderna, said it plans to start a single ascending dose trial in the coming weeksopens in a new tab or window.
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There are other mRNA therapies for rare diseases in preclinical trials. Moderna and the Institute for Life Changing Medicines are developing an mRNA therapy for Crigler-Najjar syndrome type 1. Moderna is also conducting preclinical work for OTC deficiency and phenylketonuria.
