Imagine the heart as a pump that can't fix its own leaks once the muscle cells are gone. This project uses a special genetic 'instruction manual' (mRNA) delivered in tiny fat bubbles to tell the remaining heart cells to start dividing and multiplying. It's like giving the heart a restart button to regrow its own healthy tissue instead of relying on external transplants.
Heart failure causes massive loss of cardiomyocytes that cannot spontaneously regenerate in humans. Current treatments manage symptoms but fail to address the root cause: the lack of new contractile heart cells.
A lead bicistronic mRNA product (CPY) and a roadmap for a GMP production line for LNP encapsulation.
If you are a drug developer dealing with the failure of traditional cell therapies to engraft in the heart — this project developed a bicistronic mRNA (CPY) that triggers endogenous cardiomyocyte proliferation. This allows for heart repair without the need for exogenous cell transplantation.
If you are a device company dealing with the need for precise drug delivery in the myocardium — this project developed a roadmap for catheter-based endomyocardial injection of mRNA-LNPs. This creates a high-value use case for specialized delivery catheters in pig and human models.
If you are a manufacturer dealing with the complexity of LNP encapsulation — this project developed a GMP production line for mRNA-LNP encapsulation. This provides a blueprint for scaling the production of cardiac-specific genetic medicines.
Based on available project data, the specific cost per dose or price point is not mentioned; however, the project received an EU contribution of EUR 5,010,638 for development.
Yes, the project objective explicitly includes the construction of a GMP production line for the mRNA-LNP encapsulation technology to generate pilot batches.
The final work package of the project is specifically responsible for intellectual property management and the exploitation of REGeRNA results.
The project runs from 2022-09-01 to 2027-08-31 and is framed to lead to a phase 1 trial at completion.
Delivery is planned via lipid nanoparticles (LNP) through either direct myocardial administration or systemic delivery using cardiac-specific receptors, including catheter-based endomyocardial injection.
The consortium is well-balanced for translational research, consisting of 6 partners across 5 countries. With a 33% industry ratio (2 industrial partners), the project bridges the gap between academic discovery (3 universities, 1 research institute) and commercial viability, specifically targeting GMP production and clinical trial readiness.
Contact the Institut National de la Santé et de la Recherche Médicale (INSERM) in France.
Contact us to explore licensing opportunities for the CPY mRNA construct or GMP LNP production protocols.
