Recordati has entered into a global collaboration and licensing agreement with Moderna to develop and commercialize mRNA-3927, an investigational mRNA-based therapy for the treatment of propionic acidemia (PA), a rare and life-threatening inherited metabolic disorder. The partnership brings together Moderna’s expertise in mRNA technology and Recordati’s long-standing experience in rare metabolic diseases and global commercialization.
Under the terms of the agreement, Moderna will continue to lead the development of mRNA-3927, working in collaboration with Recordati. If the therapy receives regulatory approval, Recordati will assume responsibility for worldwide commercialization. The companies said the collaboration is designed to accelerate the development of what could become the first disease-modifying treatment available for patients with propionic acidemia.
Propionic acidemia is caused by defects in the propionyl-CoA carboxylase (PCC) enzyme, leading to the accumulation of toxic metabolites in the body. The condition often presents in early childhood and can progress to severe complications, including neurological and cardiac damage, and is associated with high morbidity and mortality. Current treatment options are largely symptomatic, focusing on dietary management and supportive care, with liver transplantation sometimes considered in severe cases.
mRNA-3927 is designed to restore PCC enzyme activity by delivering functional genetic instructions through mRNA, addressing the underlying cause of the disease rather than just its symptoms. The therapy has already passed proof-of-concept and is in advanced clinical development. Interim clinical data published recently in the journal Nature showed early signs of clinical improvement, supporting its potential as a disease-modifying approach.
The investigational therapy is currently being evaluated in a potential registrational clinical study aimed at reducing the risk of metabolic decompensation events, a serious and common complication in patients with propionic acidemia. The study has completed patient enrollment, with a key data readout expected by the end of 2026.
Financially, Recordati will pay Moderna an upfront $50 million, with the possibility of up to an additional $110 million in near-term development and regulatory milestone payments. Moderna is also eligible for further commercial and sales-based milestones, as well as tiered royalties on annual net sales if the product is approved. Recordati said it does not expect the deal to have a significant impact on its EBITDA prior to a potential product launch. The transaction remains subject to customary closing conditions, including U.S. antitrust clearance.
Recordati Chief Executive Officer Rob Koremans said propionic acidemia represents a significant unmet medical need due to the lack of disease-modifying therapies. He added that the collaboration strengthens Recordati’s development portfolio and builds on its heritage in metabolic disorders. Moderna CEO Stéphane Bancel said the partnership aligns with the company’s mission to apply mRNA technology beyond vaccines and into rare diseases, highlighting Recordati’s commercial expertise as a key asset in bringing mRNA-3927 to patients worldwide.