Hematology
Teams
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Olivier Hermine
>> Research Lab
Molecular mechanisms of hematological disorders and therapeutic implications -
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Mariane de Montalembert
>> Reference Center for rare diseases
Reference centre for sickle cell disease, thalassemia and other red cell disord…
Associated with the French Healthcare Network MCGRE and the European Reference Network (ERN) EuroBloodnet -
Annie Harroche
>> Reference Center for rare diseases
Reference Center for Constitutional Hemorrhagic Diseases
Associated with the French Healthcare Network MHEMO and the European Reference Network (ERN) EuroBloodnet -
Olivier Hermine
>> Reference Center for rare diseases
Reference Center for Mastocytosis (CEREMAST)
Associated with the French Healthcare Network MARIH, the European Reference Network (ERN) EuroBloodnet and European Competence Network on Mastocytosis -
Alain Fischer
>> Reference Center for rare diseases
Reference Center for primary immunodeficiencies (CEREDIH)
Associated with the French Healthcare Network MARIH and the European Reference Network (ERN) RITA -
Imagine's Offers
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ThalassemiaGenotype Phenotype
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Cohorts
Pediatric and adult
30 / 30
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Observational trials
NCT01911871: multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations (SUFEMYO)
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Patient samples
Blood
Urine
Saliva
Red blood cells
CD34+ hematopoietic stem progenitor cells
Targets-
In vitro models
Human erythroid progenitor cell line
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Specific targets
b-globin
Therapeutics-
Patents
WO/2015/079031: Methods and pharmaceutical compositions for the treatment of beta-thalassemias
WO/2020/053224: methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies
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Specific therapeutic approaches
Lentiviral gene therapy
Small molecules
Clinical trials-
NCT
NCT02151526: phase I/II study evaluating the safety and efficacy of lentiGlobin BB305 drug product in β-Thalassemia Major and Sickle Cell Disease
NCT01571635: phase II study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta( β)- Thalassemia
NCT02604433: phase III efficacy and safety study of Luspatercept (ACE-536) versus placebo in adults who require regular red blood cell transfusions due to Beta (β) Thalassemia (BELIEVE)
Sickle Cell Disease (SCD)Genotype Phenotype-
Cohorts
ESCORT-HU: pediatric and adult
>1000
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Observational trials
NCT01911871: multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations (SUFEMYO)
NCT02909283: Sickle Cell Anemia and cerebral microcirculation : multimodal exploration (DREAM²)
NCT02516579: European Sickle Cell Disease Cohort-Hydroxyurea (ESCORT-HU)
NCT03786549: impact of a pediatric-adult care transition program on the health status of patients with Sickle Cell Disease - a randomized controlled trial
IDEAL: Identification of early predictors in sickle cell anemia to allow personalized medicine
Mortality study of pediatric sickle cell disease from 2000 to 2015 in France
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Patient samples
Blood
Urine
Saliva
Red blood cells
CD34+ hematopoietic stem progenitor cells
TargetsTherapeutics-
Patents
WO2018/220211: Viral vectors combining gene therapy and genome editing approaches for gene therapy of genetic disorders
WO2018/220210: Recombinant lentiviral vector for stem cell-based gene therapy of sickle cell disorder
WO/2020/053224: Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies
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Protocols/Technics
Protocols for lentiviral transduction of hematopoietic stem cells for ex vivo gene therapy of SCD
Protocols for genome editing of hematopoietic stem cells for ex vivo gene therapy of SCD
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Specific therapeutic approaches
Lentiviral gene therapy
Clinical trials-
NCT
NCT03964792: safety and efficacy of gene therapy of the Sickle Cell Disease by transplantation of an autologous CD34+ enriched cell fraction that contains CD34+ cells transduced ex vivo with the GLOBE1 lentiviral vector expressing the βAS3 globin gene in patients with Sickle Cell Disease (DREPAGLOBE)
NCT02212535: phase I/II assessment of tolerance of mobilizing peripheral hematopoietic stem cells by Plerixafor in Sickle Cell Patients (DrepaMob)
NCT02151526: phase I/II study evaluating the safety and efficacy of lentiGlobin BB305 drug product in β-Thalassemia Major and Sickle Cell Disease
NCT02813850: phase III oxygen therapy and pregnancy in Sickle Cell Disease (DRO2G)
Pyruvate Kinase Deficiency (PKD)Genotype Phenotype-
Cohorts
Pediatric
Less than 10
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TNGS / WES / WGS*
PIC-Registry 1
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Patient samples
Blood
Urine
Saliva
TargetsTherapeuticsClinical trialsG6P DeficiencyGenotype Phenotype-
Cohorts
Pediatric and adult
80 / 20
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Patient samples
Blood
Urine
Saliva
TargetsTherapeuticsClinical trialsRed blood cell membrane anomaliesGenotype Phenotype-
Cohorts
Pediatric
35
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Patient samples
Blood
Urine
Saliva
TargetsTherapeuticsClinical trialsCongenital dyserytrhopoiesisGenotype Phenotype-
Cohorts
Pediatric
2
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Patient samples
Blood
Urine
Saliva
TargetsTherapeuticsClinical trials -
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MastocytosisGenotype Phenotype
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Cohorts
Pediatric and adult
>500
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Observational trials
NCT02441166: diagnostic value of bone marrow tryptase in systemic Mastocytosis (EvaTryMS)
Prospective study on pediatric mastocytosis: long term follow up of 53 patients with whole sequencing of KIT
Multicenter retrospective French study on 53 patients with mastocytosis among elderly patients
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Patient samples
Blood
Targets-
Specific targets
tyrosine kinase activity of KIT
TherapeuticsClinical trials-
NCT
NCT00814073: phase III Masitinib in severe indolent or smoldering systemic Mastocytosis (AB06006)
NCT03401060: phase III interest of Denosumab treatment in osteoporosis associated to systemic Mastocytosis (DenosuMast)
NCT01266369: phase II Masitinib in patients with mastocytosis with handicap and bearing the D816V mutation
NCT00831974: phase II efficacy of AB1010 in patients with systemic indolent Mastocytosis
NCT02478957: phase II treatment of indolent systemic Mastocytosis with PA101
Myelodysplastic syndrome (MDS)Genotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trialsAcute Myeloid Leukemia (AML)Genotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trialsAcute Lymphocytic Leukemia (ALL)Genotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trialsChronic Myeloid Leukemia (CML)Genotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trialsChronic Lymphocytic Leukemia (CLL)Genotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trialsMast cell leukemiaGenotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trialsHTLV-1-associated adult T-cell leukemia/lymphomaGenotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trialsMantle cell lymphomaGenotype Phenotype-
Cohorts
Please, contact us
TargetsTherapeuticsClinical trials -
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Hemophilia AGenotype Phenotype
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Cohorts
Pediatric and adult
250
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Patient samples
Blood
Targets-
Specific targets
FVIII
TherapeuticsClinical trials-
NCT
NCT01712438: phase III human cell line-derived recombinant Factor VIII (human-cl-rhFVIII) in previously untreated patients
NCT02615691: phase III safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 Years of age with severe Hemophilia A
NCT02847637: phase III clinical trial to evaluate prophylactic Emicizumab versus no prophylaxis in Hemophilia A participants without Inhibitors (HAVEN 3)
NCT03588299: phase I/II open-label safety and dose-finding study of BAY2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human Factor VIII, in adults with severe Hemophilia A
NCT03587116: phase II six month lead-in study to evaluate prospective efficacy and safety data of current FIX prophylaxis replacement therapy in adult Hemophilia B subjects (FIX:C≤2%) or current FVIII prophylaxis replacement therapy in adult Hemophilia A subjects (FIX:C≤1%)
NCT03093480: phase IV study to evaluate efficacy of rFVIIIFc for immune tolerance induction (ITI) in severe Hemophilia A participants with inhibitors undergoing the first ITI treatment (verITI-8 study)
NCT02795767: phase III study of Emicizumab administered subcutaneously (SC) in pediatric participants with Hemophilia A and Factor VIII (FVIII) inhibitors (HAVEN 2)
NCT00947193: phase II study of Ataluren (PTC124®) in Hemophilia A and B
NCT04158648: phase III study to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of Emicizumab in participants with mild or moderate Hemophilia A without FVIII inhibitors (HAVEN 6)
Hemophilia BGenotype Phenotype-
Cohorts
Pediatric
40
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Patient samples
Blood
Targets-
Specific targets
FIX
TherapeuticsClinical trials-
NCT
NCT01496274: phase II/III safety and efficacy study of a recombinant Fusion protein linking coagulation Factor IX with albumin (rIX-FP) in patients with Hemophilia B
NCT03587116: phase III six month lead-in study to evaluate prospective efficacy and safety data of current FIX prophylaxis replacement therapy in adult Hemophilia B subjects (FIX:C≤2%) or current FVIII prophylaxis replacement therapy in adult Hemophilia A Subjects (FIX:C≤1%)
NCT02053792: phase III safety and efficacy extension study of a recombinant fusion protein linking coagulation Factor IX with albumin (rIX-FP) in patients with Hemophilia B
NCT00947193: phase II study of Ataluren (PTC124®) in Hemophilia A and B
Von Willebrand disease (VWD)Genotype PhenotypeTargetsTherapeuticsClinical trials-
NCT
NCT02932618: phase III BAX 111 rVWF in pediatrics
NCT04106908: effectiveness and tolerability of Eqwilate in real-life conditions
Constitutional ThrombocytopeniaGenotype PhenotypeTargetsTherapeuticsClinical trialsThrombotic disease of hematologic originGenotype PhenotypeTargetsTherapeuticsClinical trials -
Contacts
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Nicolas DOULET
Business development manager
- nicolas.doulet@institutimagine.org
- +33(0)1 42 75 42 55
