Recordati Enters into Collaboration with Moderna to Advance mRNA Therapy for Propionic Acidemia
Friday, January 30, 2026
Recordati has announced a strategic collaboration and licence agreement with Moderna to develop and commercialise mRNA-3927 globally for the treatment of propionic acidemia, a rare inherited metabolic disorder.
Propionic acidemia is estimated to affect between one in 100,000 and one in 150,000 people worldwide. The disorder results from genetic variants affecting the PCCA or PCCB genes, leading to enzyme deficiency and repeated, life-threatening metabolic events. At present, no approved therapies directly address the underlying cause of the disease.
mRNA-3927 consists of two messenger RNAs encoding functional PCCA and PCCB subunits. It is administered intravenously and is intended to enable the body to produce functional PCC enzymes. Data from early-stage clinical studies suggest potential clinical benefit with generally manageable side effects.
mRNA-3927 is an investigational, post proof-of-concept therapy designed to restore activity of the propionyl-CoA carboxylase (PCC) enzyme in patients with propionic acidemia. The condition is caused by defects in mitochondrial enzymes, leading to the build-up of toxic metabolites. It typically appears in early childhood and can progress to severe complications, including neurological and cardiac damage, with high mortality rates.
Current treatments mainly manage symptoms and, in severe cases, may involve liver transplantation. If approved, mRNA-3927 could become the first disease-modifying treatment available for this condition.
Under the agreement, Moderna will continue to lead the clinical development of the therapy in collaboration with Recordati, while Recordati will take responsibility for worldwide commercialisation if the product receives regulatory approval.
The therapy is a targeted treatment currently in clinical development. Interim clinical findings published recently indicated early signs of clinical improvement. mRNA-3927 is now being assessed in a potential registrational study aimed at reducing the risk of metabolic decompensation events. Patient enrolment for the study has been completed, and key data are expected by the end of 2026.
As part of the agreement, Recordati will make an upfront payment of USD 50 million to Moderna, along with up to USD 110 million in additional near-term development and regulatory milestone payments. Moderna may also receive future commercial and sales-based milestone payments, as well as tiered royalties on net sales. Recordati does not anticipate a significant impact on its EBITDA before a potential market launch.
The transaction remains subject to customary closing conditions, including U.S. antitrust approval, which is expected within approximately 30 days of filing.
Recordati is an international pharmaceutical group with activities spanning specialty care, primary care and rare diseases. Its operations cover clinical development, manufacturing and commercialisation across multiple regions worldwide.
Source: globenewswire.com
