IMPlementation of Affordable gene Correction Therapies

Introduction

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.

Project Aim

IMPACT aims to overcome the translational gap between scientific gene editing and human gene correction therapies that will be affordable and readily available for patients with rare genetic diseases.

Approach

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 requirements
2. Manufacturing requirements
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.

Innovation

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.

Long-term Goals

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.