Project description
Innovative therapeutics platform targets rare genetic diseases
There are between 6 000 and 8 000 distinct rare diseases in Europe. Although the diseases are rare, the patients are many. Over 30 million Europeans are living with rare diseases. Treatment is currently complicated due to the lack of commercial interest in developing affordable genetic therapies for a limited number of patients. The ERC-funded IMPACT project aims to develop an innovative therapeutics platform with the potential to deliver therapies for many diseases with slight adaptations to protocols. Furthermore, the project will consider regulatory, manufacturing and financial facets. The platform will be optimised using a gene correction strategy for a specific rare disease developed in a previous ERC starting grant.
Objective
Rare genetic diseases affect over 30 million people in Europe. Although individually rare and very diverse, they are all caused by genetic mutations. Recent progress in genetic technologies now holds the promise to correct the root cause of these diseases. However, it remains difficult to develop these innovative technologies into affordable therapies for these diseases that only affect few patients, limiting commercial interest.
IMPACT aims to overcome the translational gap between scientific gene editing and in human gene correction therapies that will be affordable and readily available for patients with rare genetic diseases.
To overcome this gap, we will use a multifaceted approach to set up the infrastructure for a gene correction therapy for Methylmalonic Acidemia (MMA) by elucidating and addressing the essential (1) regulatory, (2) manufacturing, and (3) financial requirements. This approach involves preclinical testing of the therapy in a perfused ex vivo human liver, clarifying the intellectual property strategy, and involving experts, stakeholders, and end users to define the regulatory, manufacturing, and financial strategies required for academic development and clinical implementation. As such, IMPACT aims to valorise the innovative gene correction strategy developed with the ERC Starting Grant to deliver prime editing tools as mRNA encapsulated in lipid nanoparticles to correct the disease-causing mutation in the liver of patients with MMA.
With IMPACT, we do not only aim to develop gene correction therapies in our academic hospital for MMA. Through our efforts in generating the infrastructure for a therapeutic platform approach with only slight adaptations to target different mutations, we ultimately aim to develop therapies for the many patients with different genetic diseases.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques.
- natural sciencesbiological sciencesgeneticsmutation
- engineering and technologynanotechnologynano-materials
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Programme(s)
- HORIZON.1.1 - European Research Council (ERC) Main Programme
Funding Scheme
HORIZON-ERC-POC - HORIZON ERC Proof of Concept GrantsHost institution
3584 CX Utrecht
Netherlands