Project
ARTiDe will achieve a breakthrough in T1D therapy and provide a validated blueprint to develop engineered Tregs for other clinical indications.
The ambitious and innovative program of ARTiDe has been designed so that the overall goal, an antigen-specific Treg product that will be ready for a phase I clinical trial available at the end of the project.
Impacts
The scientific and clinical communities will make an effective use of the pre-clinical validation of new immunotherapies for high burden diseases or disorders with unmet medical needs.
- The scientific and clinical communities will have access to new knowledge allowing for a better understanding of the mode of action for high burden diseases or disorders as well as protocols for the next generation of immunotherapies
- Health care professionals will have access to and use new evidence-based safety and efficacy guidelines for immunotherapies
- Premature mortality from non-communicable diseases will be reduce by one third by 2030
- Health care systems will benefit from strengthened research and innovation expertise, human capacities, and know-how for combating communicable and non-communicable diseases
- Patients and citizens will be knowledgeable of disease threats, involved and empowered to make and shape decisions for their health, and better adhere to knowledge-bases disease management strategies and policies
Health burden of diseases in the EU and worldwide will be reduced by patients receiving new effective, cost-efficient and affordable treatment.
- ARTiDe aims to provide a cure (instead of a symptomatic treatment) by acting early on in recent-onset T1D patients, especially children, who still have enough islets at the time of diagnosis, and by restoring specifically the physiological mechanism that normally protect us from T1D.
- The initial manufacturing cost for an individual personalized TCR-engineered Treg product as proposed by ARTiDe has been estimated at 100-150 k€ per patient in an academic setting, based on the experience of PolTREG and Miltenyi with such therapy. This price tag must be compared to current costs for conventional T1D treatment. The direct medical expenditures for T1D (insulin treatment, hospitalization costs, etc.) are around 14,000 USD per patient per annum in the USA
Diseases for which T cell therapies and genetic engineering are of therapeutic interest will also benefit from ARTiDe.
- ARTiDe knowledge (on the mechanisms underpinning the rupture of immunological tolerance) will be of general relevance in the fields of autoimmunity as well as other Immune-mediated inflammatory diseases (IMIDs) (E.G. around 80 different diseases like thyroiditis, multiple sclerosis, rheumatoid arthritis, and inflammatory bowel disease, organ and bone marrow transplantations, allergies and cancers).
- These fields will benefit from ARTiDe strategies to identify and characterize Tregs and optimal TCR as well as ARTiDe technology pipelines to rationally select and integrate these into GMP cell manufacturing processes.
Patients information
Why is pre-clinical research so important?
Immune-mediated inflammatory diseases (IMID) affect 5-10% of the population worldwide. They often cannot be cured and patients experience considerable morbidity and mortality. A prototypic IMID is type I diabetes (T1D), one of the most frequent diseases in children. Its treatment consists of insulin replacement therapy and is purely symptomatic. T1D is caused by an aberrant T cell response against pancreatic β cells. The most desirable treatment would thus be an antigen-specific immunotherapy that controls the disease-driving cells without causing toxicity or impairing protective immunity against pathogens and cancer. We know that such physiological functions are normally provided by antigen specific Tregs in healthy individuals. However, today, autoantigen-specific Tregs providing physiological protection from disease are poorly characterized. In addition, the preclinical models for testing humanT Cell Receptors (TCR) engineered Tregs are lacking. Finally, there is no Good Manufacturing Practices (GMP) compliant process to produce TCR-engineered Tregs.