Imagine has benefited mainly from public funding to create its initial endowment, build its building, and benefit from it today for the operation of labs and the work of research teams. But, to go even further, hit harder, accelerate research exponentially, give a name to a growing number of diseases and increase treatments, the Institute has to develop cutting-edge programs, attract the best French and international researchers, welcome additional teams and have the best technology.
By 2028, Imagine Institute has the following goals:
- Double the number of children diagnosed, thereby significantly reducing diagnostic uncertainty,
- Double the number of deciphered mechanisms responsible for these pathologies,
- Double the number of clinical trials to eventually double the therapeutic solutions in aid of sick children,
- Continue to enhance the Institute’s exceptional cohorts, sources of discoveries.
This will be possible thanks to you. Join our team, take part in an unparalleled project of excellence, change the lives of children and their families by giving them hope and the new treatments that they are waiting for.
Priority programs to accelerate discoveries
Imagine’s strength is bringing research teams, Reference Centers for Rare Diseases and clinical departments together around patients with genetic diseases to provide them with better care. To accelerate innovation in aid of patients, Imagine has identified priority fields for the next few years.
Meant to protect the body against attacks from bacteria, viruses or your own cells, the immune system can be defective because of a genetic error. Patients with an immunodeficiency suffer from repeated or severe infections. These diseases affect about one child in 5,000; several thousand people live with such a disease in France.
When a genetic defect affects one of the cell lines making up the blood, it can lead to a blood disorder, or hemopathy. Depending on the cell concerned - white blood cells, red blood cells, platelets - these pathologies take very varied forms: more or less severe anemias, mastocytosis (rare disease related to the proliferation of some white blood cells), hemophilia, etc.
More than 150 kidney diseases are associated with the presence of a genetic alteration. Eventually, most of these patients have to have a kidney transplant. To date, there is no treatment.
These diseases cover severe neurological, neuromuscular and neurosensory disorders, and psychiatric disorders. They occur from childhood and affect nearly 3% of children. Only some of these diseases have a specific treatment.
Development and Cardiology
Congenital malformations and developmental disorders are a major public health issue, which affects 2 to 3% of births. They can have a genetic origin, even involving more complex mechanisms. The genetic origin is rarely known and to date, very few treatments are available.
And for each of its programs, Imagine has set the goal to:
Name the disease
Firstly, above all it is about identifying the gene or genes responsible for the disease. Naming the disease is an essential condition to supporting patients and families.
- Continue to enhance the Institute’s exceptional cohorts. Cohorts help to monitor patients, the development of their pathology and symptoms. Thanks to big data technologies, their analysis paves the way for a better understanding of diseases and new therapeutic avenues,
- Continue with the genetic characterization of diseases and discover new diagnostic tools,
- Implement all resources to read and interpret all DNA, including the non-coding parts, which represent 98% of the genome and which have not been well explored to date.
Understand the disease
Once the gene in question has been discovered, researchers then study the disruptions caused. The objective of this is to identify the mechanisms to restore or to compensate for in order to treat the disease.
- Decipher the associated physiopathological mechanisms by developing new experimental models,
- Explore data from the cohorts to accelerate genetic studies, fuel research programs, establish the genotype-phenotype links (connections between the gene and symptoms), and identify groups of patients with a view to clinical trials with suitable therapies.
Of course, the purpose of this research is to develop treatments. Knowledge of deregulated mechanisms can help turn to already known therapies or to develop any new avenues.
- Double the number of clinical trials within the reference centers for rare diseases, to eventually double the therapeutic solutions: today 61 therapeutic solutions are being tested in the labs at Imagine, in the pre-clinical or clinical stage, giving hope that in 2 to 5 years more than 100 rare diseases will benefit from a treatment,
- Find common denominators to treat pools of diseases by targeting common mechanisms,
- Develop gene therapy (a therapeutic strategy consisting of transferring normal genes into the cells of an individual to replace the defective gene and treat the disease) and cell therapy for several hematologic diseases, or forms of dystrophic epidermolysis bullosa,
- Develop new treatments for orphan diseases by targeting physiopathological defects.
Major projects to develop tomorrow’s medicine
A cross-disciplinary priority program dedicated to data science and computer-aided decisions
This newly created program combines the strengths and expertise needed to deploy tomorrow’s medicine. Thanks to the development of bioinformatics tools and artificial intelligence, its objective is to draw attention to clinical and biological data, explore the fields that are still unknown, such as non-coding DNA regions which we now know have a role in the body, analyze pathologies on the level of a single cell to better understand them, and therefore, offer a tailored treatment that can evolve over time.
- Develop new bioinformatics tool and artificial intelligence to decipher genetic diseases,
- Develop software to store and analyze phenotypic data like: “eCohort” a sort of supra database connected to others to easily add and retrieve information: it now connects 102 databases, which corresponds to 200,000 patients,
- Continue with the development of Dr Warehouse: this text data warehouse has 3 uses: clinical research, hypothesis detection or data mining, and translational research, which is the stage between basic research and clinical research. Already featuring 50 million pieces of data concerning 680,000 patients, from 5.6 million documents, the goal is to continue to add to it, and particularly add “Omics”, environmental and sociological data,
- Accelerate sequencing capabilities - reading of DNA - by acquiring at least one other latest generation sequencing device,
- Deploy a single cell platform which allows a transcriptomic, epigenetic and immune analysis on the level of a single cell,
- Simplify the use of databases to accelerate discoveries and develop E-health tools.
Give even more resources to clinical research
To accelerate the availability of discoveries, Imagine Institute is already committed to giving even more resources to clinical research teams - the stage of research where advances start to be applied to patients - and deploys clinical investigation programs. Indeed, it is a key stage in the development of new diagnostic and therapeutic approaches improving patient care.
- Recruit 4 additional people to support the reference centers for rare diseases in the development of clinical trials,
- Establish funds dedicated to the clinical trial process, which is always very expensive,
- Collaborate with the European network dedicated to clinical trials to accelerate the development of new therapeutic molecules,
- Develop new modeling approaches and algorithms based on Big Data in particular, to optimize clinical trials and the “recruitment” of patients.