EDITSCD – Assessing efficacy and safety of genome EDITing approaches for Sickle Cell Disease

Project Acronym EDITSCD
Funding Program HORIZON EUROPE
Budget € 6,001,250.00
Coordinator Institut Imagine (FR)
Starting-ending date 1 September 2022 – 31 August 2027

In the EDISCD project, the consortium will study Sickle Cell Disease (SCD) hematopoietic stem/progenitor cells (HSPC) pathophysiology and improve and benchmark the efficacy and safety of different genome editing approaches for SCD. The disease is one of the most prevalent monogenic diseases in Europe. A single amino acid substitution in the beta-globin chain of the adult hemoglobin (Hb) drives red blood cell sickling and multi-organ damage.

EDITSCD consortium

The project involves a multidisciplinary team of scientists with complementary, synergic expertise in SCD pathophysiology and gene therapy of SCD (IMA) and β-thalassemia (OSR), HSC biology and bone marrow niche in β-hemoglobinopathies (OSR), development and improvement of genome editing tools (UKLFR-1/2, ETHZ, AZ and INSERM) and improvement and analysis of the specificity of genome editing tools (BIU, UKLFR-2, INSERM, AZ, ETHZ).  Shared expertise amongst the different partners will simplify the exchange of information, protocols and reagents.

EATRIS role in the project

EATRIS will support researchers in developing their biomedical discoveries into novel translational tools and interventions and will provide innovation management support to the project (WP6). By leading WP6 – Innovation Management (IM), the IM Team will analyse the feasibility of further development of specific interventions arising from the platform, by performing an early translational assessment of the described approach. This Platform will be evaluated by reviewing the future development steps of the platform required for the SCD indication in addition to other indications where genome editing offers a potential therapeutic strategy, from the viewpoint of intellectual property analysis, unmet medical need assessment including clinician second opinions, and preclinical and clinical developments steps required. EATRIS will work closely with all WP partners to ensure that the EDITSCD approaches to genome editing are sustainable, aligning with the expectations of all internal and external stakeholders in the Gene Therapy field.