Moderna’s mRNA candidate picked for FDA’s Operation Warp Speed for rare diseases

Moderna is joining another Operation Warp Speed initiative — this time, for rare diseases.

The biotech’s mRNA candidate for a protein and lipid metabolism disorder was selected by the Center for Biologics Evaluation and Research to be part of the FDA’s START pilot program, joining five other publicly-named assets.

START will focus on an initial seven candidates, with four selected by CBER and three chosen by the Center for Drug Evaluation and Research. So far, Denali Therapeutics, Larimar Therapeutics, Grace Science, Neurogene and Myrtelle have been named as participants.

The drugmakers will benefit from “rapid” interactions with the FDA to support development of their treatments through the registrational stage. The agency will advise the companies on study design, patient population and statistical methods beyond the usual extent of its standard meetings.

Moderna’s candidate, dubbed mRNA-3705, is in Phase 1/2 development for methylmalonic acidemia (MAA) due to methylmalonic-CoA mutase (MUT) deficiency. The condition leads to a toxic buildup of acids in the body that can trigger multi-organ disease. There are currently no approved treatments, with standard of care including dietary and palliative measures.

mRNA-3705 features mRNA encoding for MUT encapsulated in the company’s lipid nanoparticle. MUT is the mitochondrial enzyme that’s most often missing in people with MMA.

Kyle Holen

Moderna previously described 2024 as its “year of transition,” as it doubled down on diversifying its pipeline to ease its dependence on preventative vaccines. “This selection highlights the promise of Moderna’s innovative mRNA platform beyond vaccines and the potential this novel medicine may have in addressing the serious and unmet medical needs of MMA,” SVP Kyle Holen said in a company release.

Last month, the FDA greenlit Moderna’s RSV vaccine, the third entrant behind GSK and Pfizer. The thumbs-up marked Moderna’s first commercial product outside of the Covid-19 indication. As for its rare disease efforts, the company also has early-stage candidates for glycogen storage disease and propionic acidemia.