LeukoPredict: an in vitro system predicting and monitoring the therapeutic response of patients in the cure of Chronic Myeloid Leukemia (CML). (website)
MYOCURE – Development of an innovative gene therapy platform to cure rare hereditary muscle disorders (website)
NORMOPERF – Normothermic perfusion devices for renal and hepatic preservation and viability assessment (website)
RAM is a revolutionary magneto-optical technology platform, outperforming current solutions in accuracy, cost, and time to diagnosis, when assessing human blood cells for Malaria presence (website)
RCC-TEST – A breakthrough in Renal Cell Carcinoma: Personalized RCC-Test (website)
SCIDNET – DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID) (website)
TAT-CF – Novel therapeutic approaches for the treatment of cystic fibrosis based on small molecule transmembrane anion transporters (website)
theEoE – A novel drug candidate for the treatment of Eosinophilic Esophagitis – an innovative solution for a significant unmet medical need (website)
UM Cure 2020 – New therapies for uveal melanoma (website)
VISION-DMD – Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy (website)
VSV-EBOPLUS – SYSTEMS ANALYSIS OF ADULT AND PEDIATRIC RESPONSES TO THE VSV-ZEBOV EBOLA VACCINE (website)
ZIKAction: Preparedness, research and action network on maternal-paediatric axis of ZIKV infection in Latin America and the Caribbean (website)
ZIKAlliance – A global alliance for Zika virus control and prevention (website)
ZikaPLAN – Zika Preparedness Latin American Network (website)
ZIKAVAX- Fast track development of a Zika vaccine based on measles vector (website)
2015
Antibodies against Nogo-A to enhance plasticity, regeneration and functional recovery after acute spinal cord injury, a multicenter European clinical proof of concept trial (NISCI)
Application of combined gene and cell therapy within an implantable therapeutic device for the treatment of severe hemophilia A (HemAcure)
Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy (VISION DMD)
2014
ADvanced Validation of A Novel TB Active disease diagnostic to address Global unmet needs: a European consortium approach (ADVANTAGE)
Bioinformatics for spatial metabolomics (METASPACE)
Biomarker assay development to expedite ALS experimental therapeutic advancement (DEALS)
Clinical Proof of concept for a RNA-targeting Oligonucleotide for a Cystic fibrosis-F508del MEDication (PRO-CF-MED)
Clinical validation of the CD32b biomarker on the European market (BI-MARK)
Control of the Ebola Oubreak by both innovative Ultrasensitive Detection of EBOV and therapy (IF-EBOla)
Development of a Chimpanzee Adenovirus Type 3 Ebolavirus Zaire Vaccine (EbolaVac)
Development of a digital diagnostics rapid Lung Maturity Test (LMT) for premature infants, to assist neonatologists in the diagnosis of Respiratory Distress Syndrome at birth (SIME-LMT)
Development of a very rapid diagnostic test for malaria, based on break-through detection technology and new biomarkers of infection (MalaDiag)
Ebola Virus Disease – correlates of protection, determinants of outcome, and clinical management (EVIDENT)
Efficacy and safety of low-dose IL-2 (ld-IL-2) as a Treg enhancer for anti-neuroinflammatory therapy in newly diagnosed Amyotrophic Lateral Sclerosis (ALS) patients (MIROCALS)
Eliciting Mucosal Immunity to Tuberculosis (EMI-TB)
Emergency evaluation of convalescent blood and plasma for Ebola Viral Disease (EVD) in affected West African countries (Ebola_Tx)
ERA-NET rare disease research implementing IRDiRC objectives (E-Rare-3)
Evaluation of the efficacy and of the antiviral activity of T-705 (favipiravir) duringEbola virus infection in non-human primates humans (REACTION)
Feasibility study for the validation and industrialization of two diagnostic kits in both ELISA and CLIA formats for the early detection of pancreatic cancer based on the novel biomarkers EZR and ENOA (EDePDAC)
Innovative approach for self-management and social welfare of Cystic Fibrosis patients in Europe: development, validation and implementation of a telematics tool (MyCyFAPP)
Innovative methodology for small populations research (INSPIRE)
Modeling Disease through Cell Reprogramming: a Translational Approach to the Pathogenesis of Syndromes Caused by Symmetrical Gene Dosage Imbalances (DISEASEAVATARS)
Personalized diagnosis and treatment of hyperinsulinemic hypoglycaemia caused by beta-cell pathology (BETACURE)
PRECEDE: PancREatic Cancer Early Detection (PRECEDE)
Preclinical studies in mouse hematopoietic stem cells for gene therapy of Wiskott-Aldrich Syndrome (WASHSCGENETHERAPY)
PreEclampsia diagnosis by Early Kidney Injury Detection (PREKIND)
Providing the right care to the right patient with MyeloDysplastic Syndrome at the right time (MDS-RIGHT)
Regenerative treatment of complete Traumatic Spinal Cord injury with a surgical implantation of a biodegradable device with FGF1 and nerve grafts (SC0806)
Repair of tissue and organ damage in refractory chronic graft versus host disease after hematopoietic stem cell transplantation by the infusion of purified allogeneic donor regulatory T lymphocytes (TREGeneration)
Restoring tissue regeneration in patients with visceral Graft versus Host Disease (RETHRIM)
TBVAC2020; Advancing novel and promising TB vaccine candidates from discovery to preclinical and early clinical development (TBVAC2020)
Validation of blood-brain-barrier permeability as a glioma biomarker by means of the radiotracer 99mTc-tetrofosmin and single-photon emission computer tomography (GLIOMARK)
2013
A european pilot network of reference centres in refractory epilepsy and epilepsy surgery (E-PILEPSY)
Advances in Small Trials dEsign for Regulatory Innovation and eXcellence (ASTERIX)
Advancing and strengthening the methodological tools and practices relating to the application and implementation of Health Technology Assessment (HTA) (ADVANCE_HTA)
Biopharmaceutical therapy for treatment of Primary Hyperoxaluria (ELIMOX)
Clinical safety, immunogenicity and efficacy of a therapeutic vaccine that combines peptides mimicking antigen receptors on autoimmune B and T cells associated with myasthenia gravis (MYASTERIX)
Closed-loop Molecular Environment for Minimally Invasive Treatment of Patients with metastatic Gastrointestinal Stromal Tumours (MITIGATE)
Collaborative European NeuroTrauma Effectiveness Research in TBI (website)
Combined Molecular Microscopy for Therapy and Personalized Medication in Rare Anaemias Treatments (COMMITMENT)
EURO EWING Consortium – International Clinical Trials to Improve Survival from Ewing Sarcoma (EEC)
European Expert Paediatric Oncology Reference Network for Diagnostics and Treatment (ExPO-r-NeT)
European registry and network for Intoxication type Metabolic Diseases (E-IMD)
Human pluripotent stem cell differentiation, safety and preparation for therapeutic transplantation in Huntington’s disease (REPAIR-HD)
Identifying correlates of protection to accelerate vaccine trials: systems evaluation of two models of experimentally-induced immunity to malaria (SYSMALVAC)
Integrated DEsign and AnaLysis of small population group trials (IDEAL)
Integrative Analysis of Gene Functions in Cellular and Animal Models of Pancreatic Cancer (CAM-PAC)
Microbubble driven multimodal imaging and theranostics for gliomas (THERAGLIO)
Multimodal Imaging of rare Synucleinopathies (MULTISYN)
Multivessel versus culprit lesion only percutaneous revascularization in patients with acute myocardial infarction complicated by cardiogenic shock (CULPRIT-SHOCK)
New Molecular-Functional Imaging Technologies and Therapeutic Strategies for Theranostic of Invasive Aspergillosis (MATHIAS)
Phase I/II Gene Therapy Trial of Fanconi anemia patients with a new Orphan Drug consisting of a lentiviral vector carrying the FANCA gene: A Coordinated International Action (Eurofancolen)
Reach alfa-synuclein-dependent neurodegeneration: clinical development of therapeutic AFFITOPE vaccines for Parkinson’s disease and multisystem atrophy (SYMPATH)
Surveillance of cerebral Palsy in Europe (SCPE network)
Systems biology for the functional validation of genetic determinants of skeletal diseases (SYBIL)
Understanding The Causes Of The Rna Gain Of Function Diseases (RNA DISEASES)
2012
A phase I/IIa clinical trial in Duchenne muscular dystrophy using systemically delivered morpholino antisense oligomer to skip exon 53 (SKIP-NMD)
Advancing diagnosis, care and treatment for those living with neuromuscular diseases around the world (Treat-NMD)
An EU Rare Diseases Registry for Wolfram syndrome, Alström syndrome, Bardet-Biedl syndrome and other rare diabetes syndromes (EURO-WABB)
Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia (BESTCILIA)
Clinical Development of Nitisinone for Alkaptonuria (DEVELOPAKURE)
Clinical European study on the outcome of surgical and hormonal therapy and psychological intervention in disorders of sex development (DSD) (DSD-Life)
Clinical trial of gene therapy for MPS VI – a severe lysosomal storage disorder (MEUSIX)
Development of a bioartificial liver therapy in acute liver failure (BALANCE)
Development of a prophylactic treatment for the prevention of fetal/neonatal alloimmune thrombocytopenia (FNAIT) (PROFNAIT)
European Consortium for High-Throughput Research in Rare Kidney Diseases (EURenOmics)
European Consortium for the Study of a Topical Treatment of Neovascular Glaucoma (STRONG)
European network and registry for homocysinurias and methylation defects (E-HOD)
European registry of patients with McArdle disease and very rare muscle glycogenolytic disorders (MGD) with exercise intolerance as the major symptom (PR-MDMGD) (EUROMAC)
European Surgical Registry for Rare Endocrine Tumours (EUROCRINE)
First Targeted Therapy to FIGHT Hemophagocytic Lymphohistiocytosis (HLH): A novel approach to HLH (FIGHT-HLH)
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) (NET4CGD)
IMproved Pregnancy Outcomes by Early Detection; personalised medicine for pregnant women: novel metabolomic and proteomic biomarkers to detect pre-eclampsia and improve outcome (Improved)
Orphans Unite: chILD better together European Management Platform for Childhood Interstitial Lung Diseases (CHILD-EU)
Platform for sharing best practices for management of rare diseases (RARE-Bestpractices)
Preclinical development of drugs and drug delivery technology for the treatment of inherited photoreceptor degeneration (DRUGSFORD)
RD-CONNECT: An integrated platform connecting registries, biobanks and clinical bioinformatics for rare disease research (RD-Connect)
Single Hub and Access point for paediatric Rheumatology in Europe (share)
Support for international rare disease research to serve the IRDiRC objectives (Support-IRDiRC)
Targeting common mechanisms of pathogenesis in diseases of sterol homeostasis associated with lysosome dysfunction; development of novel and rapidly translatable clinical therapies (STEROLOSOME)
THALAssaemia MOdular Stratification System for personalized therapy of beta-thalassemia (THALAMOSS)
To decipher the optimal management of systemic sclerosis (DESSCIPHER)
A Treatment-Oriented Research Project of NCL Disorders as a Major Cause of Dementia in Childhood (DEM-CHILD)
An EU rare diseases registry for Niemann-Pick Disease type A, B and C. (NPDR)
DARPin Targeted Magnetic Hyperthermic Therapy for Glioblastoma (DARTRIX)
European clinical study for the application of regenerative heart valves (ESPOIR)
European Clinical trials in Rare Sarcomas within an integrated translational trial network (EUROSARC)
Genetic studies of pre-eclampsia in Central Asian and European populations (InterPregGen)
IMmune MOdulating strategies for treatment of MErkel cell Carcinoma (IMMOMEC)
Innovative strategies to generate human hepatocytes for treatment of metabolic Liver diseases: Tools for personalized cell therapy (InnovaLiv)
International study for treatment of childhood relapsed ALL 2010 with standard therapy, systematic integration of new agents, and establishment of standardized diagnostic and research (IntReALL)
NOX enzymes as mediators of inflammation-triggered neurodegeneration: modulating NOX enzymes as novel therapies (NEURINOX)
OPtimizing TArgets and Therapeutics In high risk and refractOry Multiple Myeloma (OPTATIO)
Ovarian Cancer Therapy – Innovative Models Prolong Survival (OCTIPS)
Overcoming clinical relapse in multiple myeloma by understanding and targeting the molecular causes of drug resistance (OVER-MyR)
The European Wilson’s Disease Network (Eurowilson)
The production of a 3d human tissue disease platform to enable regenerative medicine therapy development (TissueGEN)
Treatment of Adrenal Insufficiency in neonates- Development of a Hydrocortisone Preparation for the treatment of Adrenal Insufficiency in neonatesand infants. (TAIN)
2010
A systems biology approach to dissect cilia function and its disruption in human genetic disease (syscilia)
Activation of vasculature associated stem cells and muscle stem cells for the repair and maintenance of muscle tissue (Endostem)
Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency (CELL-PID)
Augmenting PBGD expression in the liver as a Novel Gene therapy for Acute Intermittent Porphyria (AIPgene)
Building Consensus and Synergies for the EU Registration of Rare Disease Patients (EPIRARE)
Clinical development of Enzyme Replacement Therapy in alpha-Mannosidosis patients using recombinant human enzyme.(ALPHA-MAN )
Development of OPN-305 as an orphan drug for the treatment of Delayed Graft Function post solid organ transplantation (MABSOT)
Development of the European portal of rare diseases and orphan drugs (JA-Orphanet Europe)
Immunoglobulin IgY pseudomonas A clinical trial for cystic fibrosis treatment (IMPACTT)
INtegrated HEart Research In TrANslational genetics of dilated Cardiomyopathies in Europe (Inheritance)
Nuclease Immune Mediated Brain and Lupus-like conditions (NIMBL): natural history, pathophysiology, diagnostic and therapeutic modalities with application to other disorders of autoimmunity (Nimbl)
Pharmacodynamic Approaches to Demonstration of Disease-Modification in Huntington’s Disease by SEN0014196 (PADDINGTON)
Phase I/II ex vivo gene therapy clinical trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector (Orphan drug designation (EU/3/09/630)) (GENEGRAFT)
Preclinical study of Recombinant human Anti-C5 for the Treatment of atypical HUS (Prath)
