Imagine your blood cells are like tiny balloons that collapse and get stuck in your veins. This project looks for the best way to 'reprogram' the body's master blood cells to either fix the mistake or switch on a backup system from when we were babies. It's like comparing three different types of high-tech biological erasers to see which one cleans the genetic error most safely.
Current gene therapies for Sickle Cell Disease face safety risks and unpredictable results because the patient's own stem cells are already damaged by oxidative stress and mutations.
A comparative analysis and set of best-practice protocols for three genome editing methods (CRISPR/Cas9 nuclease, CRISPR/Cas9 HDR, and Base Editing) to treat SCD.
If you are a gene therapy developer dealing with safety concerns regarding off-target mutations — this project developed a comparison of three genome editing strategies that improves genome integrity. This allows for the selection of the safest method for clinical transition.
If you are a drug manufacturer dealing with the high mutational burden in Sickle Cell Disease patients — this project developed tools to evaluate how editing affects stem cell properties. This helps in designing therapies that don't fail due to pre-existing cell dysfunction.
If you are a CRO dealing with a lack of standardized protocols for HSPC editing — this project developed best practice tools and protocols for genome editing-based therapies. This reduces the risk of failure during the transition from lab to patient.
Based on available project data, the specific cost per patient or treatment is not provided; only the EU contribution of EUR 6,532,000 for the research phase is listed.
Based on available project data, the project focuses on evaluating efficacy and safety in stem cells, which is a prerequisite for industrial scaling, but specific scaling metrics are not mentioned.
Based on available project data, the consortium has developed a base editing approach to generate HPFH mutations, but specific patent or licensing terms are not disclosed.
The project runs from 2022-09-01 to 2027-08-31, suggesting that final safety and efficacy data will be available by late 2027.
The project focuses on the transplantation of autologous, genetically modified hematopoietic stem/progenitor cells, which aligns with current clinical transplantation workflows.
The consortium is research-heavy with 7 academic/research partners and 2 industrial partners (22% industry ratio). Spanning 8 countries, it combines deep academic expertise in genetics (Imagine Institut) with industrial capacity, suggesting a focus on high-risk, high-reward validation before commercialization.
Contact Imagine Institut des Maladies Genetiques Necker Enfants Malades Fondation
Contact us to explore licensing opportunities for the base editing protocols developed in EDITSCD.
