Recordati and Moderna have partnered to develop and commercialize mRNA-3927, a new therapy aimed at treating propionic acidemia, potentially transforming treatment options for this rare metabolic disorder.

Target Information

Recordati has entered into a strategic collaboration with Moderna to develop and commercialize mRNA-3927, an investigational therapy aimed at treating propionic acidemia (PA), a rare inherited metabolic disorder. This collaboration seeks to leverage Moderna's cutting-edge mRNA technology alongside Recordati's established infrastructure in rare disease commercialization. Under the agreement, Moderna will take the lead in the development of mRNA-3927, while Recordati will manage global commercialization efforts if the product receives regulatory approval.

mRNA-3927 aims to restore the activity of the propionyl-CoA carboxylase (PCC) enzyme, which is crucial for patients suffering from propionic acidemia. This disorder is characterized by the accumulation of toxic metabolites due to faulty mitochondrial enzymes, leading to severe neurological and cardiac conditions. Currently, treatment options are largely symptomatic, and have the potential to require interventions such as liver transplants. If mRNA-3927 gains approval, it could represent the first medically transformative treatment available for PA.

Industry Overview

In recent years, the pharmaceutical industry has seen significant advancements in the field of gene and mRNA therapies, particularly in addressing rare diseases. These disorders, often overlooked in the past, are gaining increased attention due to their devastating effects and the growi