Moderna Inc (MRNA)

[axelvento]

Moderna has early clinical evidence that its mRNA technology can treat a rare disease by replacing intracellular proteins. Participants in the phase 1/2 trial experienced a 66% overall relative risk reduction in the frequency of life-threatening events, encouraging the biotech to keep studying the candidate.

The phase 1/2 trial marks the first time a biotech has reported clinical data on an mRNA therapeutic for intracellular protein replacement. In the study, children with propionic acidemia (PA), a rare inherited metabolic disorder, received multiple doses of a lipid nanoparticle-encapsulated dual mRNA therapy.

Moderna’s therapy, mRNA-3927, encodes two subunit proteins, PCCA and PCCB, that form the enzyme that is deficient in patients with PA. By triggering production of the subunit proteins, Moderna could fix the root cause of the disease and prevent the buildup of harmful compounds that cause PA symptoms.

https://www.fiercebiotech.com/biotech/moderna-looking-past-vaccines-guides-first-mrna-protein-replacement-therapy-past-early