Imagine your heart's electrical system has a typo in its instruction manual, causing it to beat irregularly. Instead of just treating the symptoms, this work acts like a molecular eraser and pencil to fix those typos in the DNA. By correcting the root cause, the heart muscle can regain its strength and function properly.
Developing cures for rare heart diseases is currently too risky and expensive due to a lack of accurate disease models and poor understanding of how these mutations work.
Preclinical proof-of-concept for gene therapies targeting PLN and PKP2 mutations, including engineered heart tissues and in vitro disease models.
If you are a gene therapy developer dealing with the high risk of rare disease drug discovery — this project developed a parallel testing approach for PLN and PKP2 mutations that provides preclinical proof-of-concept. This reduces the time and cost of identifying effective genetic corrections.
If you are a cardiac clinic network dealing with the lifelong burden of treating inherited cardiomyopathy — this project developed a potential curative treatment. This could shift the business model from chronic symptom management to a one-time curative intervention.
If you are a CRO dealing with a lack of relevant disease models for heart failure — this project developed engineered heart tissues and in vitro models. These tools allow for faster and more accurate testing of new cardiac drugs.
Based on available project data, there is no specific pricing or cost information provided; the project focuses on the preclinical development phase.
The project is currently focused on providing in vitro and in vivo preclinical proof-of-concept. Industrial scaling details are not mentioned in the current data.
The consortium aims to maximize exploitation of the technology and is preparing for orphan drug designation, though specific licensing terms are not listed.
Partners with regulatory expertise are preparing for the immediate start of clinical trials and applying for orphan drug designation upon project completion.
The project runs from 2023-05-01 to 2027-04-30, with clinical trial preparation occurring toward the end of this period.
The consortium is heavily weighted toward research and academia, with 5 universities and 3 research institutes. With only 1 industry partner (9% ratio), the project is primarily driven by scientific discovery and preclinical validation. However, the inclusion of regulatory and clinical trial experts suggests a clear path toward commercialization and patient application.
Contact the Stichting Netherlands Heart Institute regarding gene therapy for ACM.
Contact us to connect with the GEREMY consortium for licensing preclinical cardiac models.
