Data-Gouv-ML/pcri-informations-sur-les-projets-laureats huggingface dataset:
Consortium for Products across Europe in Duchenne Muscular Dystrophy (SCOPE-DMD)

Framework: FP7 — Cooperation
Programme: Health
Destination: Horizontal topics for collaborative projects relevant for the whole of theme health
Action type: Collaborative project
Countries involved: United Kingdom,France,Netherlands,Germany
Call:  (FP7-HEALTH-2013-INNOVATION-2, 2013)
Start date: 2013-06-01
End date: 2016-05-31
Duration (months): 36.0
Total project cost (EUR): 21358069.0
EU funding (EUR): 5999210.0
Participants: 5
Project status: CLOSED

This project builds on results from the TREAT-NMD and BIO-NMD European projects to develop a personalised-medicinal product through to market for the treatment of Duchenne Muscular Dystrophy (DMD). DMD is an inheritable, childhood rare disease that affects approximately 1 in 3,500 newborn boys. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, typically rendering them wheelchair-bound before the age of 12 and most patients die in early adulthood due to respiratory and cardiac failure. At present, there is no effective treatment for DMD. The goal of the SCOPE-DMD project is to develop a therapy that can restore the expression of a functional dystrophin protein in a targeted DMD patient sub-population. This will be achieved using RNA-based technology, specifically antisense oligonucleotides (AON) inducing exon skipping. The clinical heterogeneity and disease complexity in rare diseases present a major challenge to properly observe statistically and clinically meaningful treatment effects. Innovative clinical study designs and novel outcome measures are required to reduce development timelines. Based on extensive experience from classical clinical trials of two other AONs in DMD patients, the consortium has designed a highly innovative development plan, including an innovative clinical trial design that if successful, could be applied to future clinical trials in DMD and other rare diseases. Also novel biomarkers and functional outcome measures will be incorporated in additional to more established ones. Multiple layers of expertise and scientific knowledge come together in this SCOPE-DMD project consortium to launch an innovative drug product but also to provide a regulatory and pathway-to-market precedence in order to benefit future patients with rare disorders getting earlier access to treatment.

Keywords: innovative paediatric clinical trials,new therapies,rare diseases,biomarker,personalised medicine,neuromuscular diseases
Green roofs that clean SME industrial gas containing low and variable concentrations of Volatile Organic Compounds (GRABGAS)

Framework: FP7 — Capacities
Programme: Research for the benefit of SMEs
Destination: Research for SMEs
Action type: Research for the benefit of specific groups
Countries involved: France,Germany,Spain,France,Spain,Italy,Slovenia
Call:  (FP7-SME-2013, 2013)
Start date: 2014-05-01
End date: 2016-04-30
Duration (months): 24.0
Total project cost (EUR): 1487792.02
EU funding (EUR): 1132972.5
Participants: 8
Project status: CLOSED

Cost-effectiveness for controlling emissions of Volatile Organic Compounds (VOC) in industrial waste gas depend on the characteristics of the gas emitted. SMEs affected by VOC emission Directive 99/13/EC (wood, printing, surface cleaning, dry cleaning, leather, vehicle refinishing) generate low and variable concentrations of several different VOCs in small volumes of waste air. This profile of VOC emissions will be the focus of the new Industrial Emission Directive 2010/75/EU, but it shows the most techno-economic problems for VOC control. GRABGAS will use rhizodegradation to treat small volumes of industrial waste air (1000 m3/h) with low and variable concentrations (30-300 mgC/m3) of several different VOCs with a removal efficiency of >93% to comply with directive emission limit values (20mgC/m3). Rhizodegradation uses plant roots, microorganisms and soil to biologically degrade VOC into CO2 and H2O. It can simultaneously degrade several VOC in very low concentrations or even when they are temporarily absent. GRABGAS will adapt to changing concentration of different pollutants, as well as to weekend and short holiday interruptions without losing removal efficiency. To make VOC mixtures more biodegradable, a solar photocatalytic pre-treatment using doped-TiO2 will be applied before rhizodegradation. To support plant growth and solar photocatalysis with sunlight, GRABGAS will be installed on industries’ roofs, following European green roof standards. Optimal air flow through the rhizofilter will be achieved by a substrate allowing root growth, high active surface for bacterial growth, permeability for air and VOC storage capacity. It will keep the internal temperature and moisture within constant ranges by appropriate sealing, insulation and drainage. Total economic benefits of GRABGAS for the SMEs owning IPR and constructing GRABGAS (LEPRIEURE, AGRIFUTUR and UGN) will be 46 million € in 5 years. Total savings for VOC emitting end users (COLORIS) will be 92,000 € in 5 year

Keywords: rhizodegradation,plant-assisted degradation,biofilter,organic compound biodegradation,voc remediation,heterogeneous catalysis,photocatalysis,photo-oxidation,tio2,doped-tio2,green roof.
New AntiBacterials with Inhibitory activity on Aminoacyl-tRNA Synthetases (NABARSI)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Netherlands,Latvia,United Kingdom,United Kingdom,Spain
Call:  (FP7-HEALTH-2013-INNOVATION-2, 2013)
Start date: 2013-06-01
End date: 2016-05-31
Duration (months): 36.0
Total project cost (EUR): 5372277.01
EU funding (EUR): 4102157.5
Participants: 5
Project status: CLOSED

The NABARSI consortium will develop a cutting-edge drug discovery project to increase the antibacterial pipeline. The main goal of NABARSI is to find new chemical entities (NCEs) with antibacterial efficacy in animal models of multi-drug resistant (MDR) bacterial infection and to exploit the results through obtaining a co-development with industry. The NABARSI consortium consists of 5 partners: Omnia Molecular (Omnia, SME; Spain). InhibOx (SME, UK). Latvian Institute of Organic Synthesis (LIOS, Latvia), Leeds University (Leeds, UK) and Erasmus Medical Centre (ErasmusMC, The Netherlands - Coordinator). Antibacterial activity will be achieved through inhibition of essential aminoacyl-tRNA synthetases (aaRS). Individual aaRS are highly conserved across bacteria, enabling the discovery of broad-spectrum antibacterials. To reduce the likelihood of resistance, NABARSI will look for NCEs with inhibitory activity against multiple aaRS enzymes. InhibOx and LIOS will design NCEs by rational and fragment based drug discovery methods followed by synthetic structure optimization. To increase chemical diversity, virtual screening of large (>100 M) compound libraries available at InhibOx will be performed. Limitations of previous aaRS inhibitors will be overcome by novel approaches such as the In Omnia assay: activity of the compounds on pathogenic aaRS enzyme is measured inside a human cell, allowing rejection of compounds acting through human aaRS and identifying compounds that cross biological membranes. The expertise of Leeds in mode of action studies will be used at an early stage. Activity of the NCEs on clinical isolates of MDR strains available at ErasmusMC will be assessed. Resistance appearance frequency and mechanisms will also be assessed early by selection and characterization of resistant mutants by ErasmusMC and Leeds. A co-development agreement with pharmaceutical companies will be intensively sought with the aim of exploiting the NCEs upon finalisation of NABARSI.

Keywords: antibacterial,aminoacyl-trna synthetase,drug discovery,resistance,therapeutic,co-development,new chemical entity
A “Universal” Influenza Vaccine through Synthetic, Dendritic Cell-Targeted, Self-Replicating RNA Vaccines (UNIVAX)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Switzerland,France,France,Czechia,France,United Kingdom,Germany,Norway,Germany,Russian Federation,Germany
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2019-03-31
Duration (months): 66.0
Total project cost (EUR): 7834394.62
EU funding (EUR): 5999457.0
Participants: 14
Project status: CLOSED

Although vaccination is the cornerstone of prophylaxis, current vaccines provide only moderate protection. Most employ inactivated or protein-based, including multimeric antigen, vaccines requiring annual updating. Their limited antigen loads provide limited capacity for inducing robust immune defences, without assurance that both humoral and cell mediated (CMI) responses, as well as durable immunity, will be induced. Replicating vaccines may provide several rounds of antigen production, increasing potential for humoral and CMI defence induction. Neither live, attenuated nor vector vaccines can be produced synthetically, being reliant on cell culture or egg production. They cannot be targeted to immune cells; interference from pre-existing immunity is also a risk. Efficacious, synthetic vaccines would be the answer, as seen with self-replicating RNA replicon (RepRNA) technology – these replicate and translate without producing infectious progeny. RepRNA produced in vitro is combined with synthetic delivery vehicles targeting dendritic cell (DC) receptors –essential for efficient immune defences– with glycoconjugate ligands. The UniVax project promotes the first synergising of approaches with synthetic targeted delivery systems for RepRNA. The innovation integrates technologies of (i) RepRNA vaccines, (ii) lipoplexes (biodegradable lipid/adjuvant/RNA for cytosolic delivery), (iii) polyplexes (biodegradable, polysaccharide vehicles), (iv) glycoconjugates targeting DC receptors, (v) adjuvants with well-defined molecular targets and effector functions. This promotes efficacious mucosal and systemic responses, ensuring for the first time both humoral and CMI responses. Components developed in PANFLUVAC and Replixcel projects allow UniVax to create the first Universal” Flu vaccine prototypes. This innovative approach creates the first synthetic vaccine of its kind, promoting consortium SMEs to a unique position of world leaders.

Keywords: universal vaccine,replicating rna,synthetic particle delivery,glycan-targeting dendritic cells,mucosal adjuvants antibody,cell-mediated immunity,long-lasting immunity,heterosubtypic immunity
Validating predictive models of radiotherapy toxicity to improve quality-of-life and reduce side-effects in cancer survivors (REQUITE)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,Germany,Belgium,United Kingdom,Belgium,United Kingdom,France,Italy,Spain,United Kingdom,United States,Netherlands
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2018-09-30
Duration (months): 60.0
Total project cost (EUR): 7820545.67
EU funding (EUR): 5997408.0
Participants: 16
Project status: CLOSED

Long-term side-effects of radiotherapy impact on the quality-of-life (QoL) of cancer survivors. These side-effects could be reduced if predicted in advance. Previous work identified clinical and biological predictors but a major, coordinated approach is needed to validate them so they can be used clinically. The EU has ~17.8 million people living with a prior diagnosis of cancer of whom ~7 million received radiotherapy. In the long-term, potentially 20% of those suffering with mild to severe side-effects (~1.4 million) might benefit from alleviation of symptoms, with resulting reductions in the cost of care in the EU. REQUITE aims to develop validated clinical models and incorporate biomarkers to identify before treatment cancer patients at risk of side-effects and use the models to design interventional trials aimed at reducing side-effects and improving QoL in cancer survivors who underwent radiotherapy. REQUITE will: 1. carry out a multi-centre, longitudinal, observational study to collect standardised data and samples in breast, prostate and lung cancer patients; 2. validate biomarkers with published evidence of predictive value; 3. replicate published clinical models and incorporate replicated biomarkers to create validated predictive algorithms; 4. use the prospectively validated models and biomarkers to design interventional trial protocols aiming to reduce side-effects and improve QoL in high-risk patients. REQUITE builds on collaborations with a proven history of data sharing, enlarged to a consortium with expertise in patient recruitment, knowledge management, biomarker testing and predictive model development. SME involvement for biomarker assays will facilitate future clinical implementation and commercial exploitation. The outcome of this project will be validated predictive models for three common cancers and trial protocols using the models to investigate interventions aimed at reducing long-term side-effects and improving the QoL of cancer survivors.

Keywords: radiotherapy cancer survivors quality-of-life long-term symptoms of treatment toxicity predictive model biomarkers personalised medicine breast cancer prostate cancer lung cancer observational study
Novel antifungals to treat resistant organisms (NOFUN)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,Spain,Germany,Sweden
Call:  (FP7-HEALTH-2013-INNOVATION-2, 2013)
Start date: 2013-07-01
End date: 2016-06-30
Duration (months): 36.0
Total project cost (EUR): 6106868.4
EU funding (EUR): 4550286.0
Participants: 5
Project status: CLOSED

Invasive fungal diseases are estimated to kill 1.5 million people each year. The incidence of mortality has risen significantly across the EU over the last 20 years due to an expansion of at risk patient populations. Given the obvious importance of these diseases it is perhaps surprising that only four classes of drug are available to treat systemic fungal infection. The azole class of antifungals provide the front line role for most disease treatment but recently resistance has emerged and it is of growing concern that levels are rising dramatically. European researchers have led the world in identifying the extent of the problem with some centres reporting itraconazole resistance in the Aspergillus species as high as 20% and in Candida, resistance to posaconazole upto 30%. Additionally the epidemiology of serious fungal infections is changing with more intrinsically resistant organisms now being seen more frequently. This represents a major problem for clinicians who are increasingly treating infections for which there is no current effective therapy. This proposal brings together leading European SME’s and academics to address this problem through the development of novel classes of antifungals and the identification of novel drug targets. The NOFUN consortium has identified potent novel broad spectrum antifungal molecules that are active against multi-resistant fungal pathogens and intends to qualify these as drug candidates. One of these assets is already at the lead identification stage. Cutting edge fungal genomics will be used to identify novel druggable targets and advance these to develop qualified tractable chemical inhibitors. With its wide ranging expertise across medicinal chemistry, ADMET, fungal biology, chemical genomics and drug development the partners will build and progress a broad pipeline of agents that have the potential to reach the clinic within 5 years.

Keywords: resistance,novel antifungal,new target,candida,aspergillus,genomics
Universal Influenza Vaccines Secured (UNISEC)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Netherlands,United Kingdom,Denmark,Hungary,Norway,Germany,Sweden,Sweden,United Kingdom,Israel
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2018-03-31
Duration (months): 54.0
Total project cost (EUR): 7824984.91
EU funding (EUR): 5972180.5
Participants: 14
Project status: CLOSED

UNISEC brings together a European consortium of 3 University partners, 5 National Health Institutes and 3 SMEs, all with leading expertise in influenza vaccine research and development, to work on promising recently developed concepts for a universal influenza vaccine. The consortium is exceptionally strong, interdisciplinary and intersectional in nature, and encompasses a unique combination of scientific and technological expertise from lead identification to clinical trial execution. UNISEC will bring a number of vaccine concepts together into a single study in order to identify, develop and clinically test the 3 most promising leads for a universal influenza vaccine, which must (1) demonstrate adequate (universal) protection, and (2) be technically suitable for development to a marketable product. Preclinical and clinical methodologies will be developed allowing a comparative evaluation of the vaccine concepts. Head-to-head comparisons in animal models will also be performed, with the most promising concepts to then be ready for clinical assessment. Independent comparative clinical studies will be executed and carried out by 5 established Clinical Trial Site partners. The expected impact of the UNISEC work programme will be the resolution of current problems of non-uniformity in the development and evaluation of universal influenza vaccines in Europe by a unified approach using an expert and sustainable vaccine expertise network. The UNISEC results will directly contribute to a timely, longer-term, broader, more effective and cost-effective universal influenza vaccine for the prevention of annual and pandemic influenza. The success of UNISEC will be based on the leading experience of its consortium partners; the proven track record of its clinical trial site partners; and a coherent and well-defined management structure and dissemination program, underpinned by the fact that several UNISEC partners were previously part of the successful EU FLUSECURE project.

Keywords: vaccine concepts,preclinical development,formulation,correlates of protection,comparative clinical study
Treatment of late onset bacterial sepsis caused by vancomycin susceptible bacteria in neonates and infants aged under three months (NEOVANC)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Collaborative project
Countries involved: Italy,United Kingdom,France,Estonia,Italy,Spain,Greece,United Kingdom,Spain,United Kingdom
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2014-02-01
End date: 2020-07-31
Duration (months): 78.0
Total project cost (EUR): 7721444.45
EU funding (EUR): 5993000.0
Participants: 13
Project status: CLOSED

Vancomycin is the critically important antibiotic to treat neonatal Late Onset Sepsis (LOS) due to Gram positive bacteria in neonates, including Coagulase Negative Staphylococci (CoNS) and Staphylococcus aureus. These organisms also create biofilms which are extremely resistant to antibiotics. The increased incidence of LOS due to bacteria such as CoNS and MRSA in NICUs has led to a marked increased use of vancomycin, which is now the third commonest antibiotic used in European NICUs. However, a standardised dosing regimen for premature infants has not yet been defined and there is no data about the serum concentrations needed to ensure bacterial kill for CoNS in humans. In view of the lack of any firm dosage for neonates and infants, vancomycin has been included in the EMA list of off-patent drugs addressing unmet therapeutic needs in children. Accordingly NeoVanc consortium has already submitted a Paediatric Investigation Plan (PIP) which has provisionally received a favourable 120 day opinion and this application is built on what is included in the approved PIP. This project aims to:-develop a new age-appropriate formulation of vancomycin; define the circulating concentration of vancomycin that is needed to kill CoNS in in vitro biofilm and animal model, and use that data to derive the concentration and best PD target that will be maximally effective in neonates; define the neonatal dosage that is needed to attain the concentration that can kill CoNS and enterococci by conducting a systematic meta-analysis of all available PK data and develop an optimal dosing and therapeutic drug monitoring regimen. NeoVanc will then conduct a Phase 2 b randomised clinical trial to compare the proportion of neonates reaching the PD target derived from the pre-clinical studies when treated with the current standard vs new “optimised” treatment regimens and to obtain data on dosing, efficacy and short and long-term safety to be included in the SPCs leading to a PUMA.

Keywords: vancomycin,antibiotic,neonate,sepsis,pharmacokinetics,pharmacodynamics,toxicity,randomised controlled trial,clinical efficacy,central venous catheter,modelling
Combined Molecular Microscopy for Therapy and Personalized Medication in Rare Anaemias Treatments (COMMITMENT)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: Germany,Switzerland,Netherlands,Spain,United Kingdom,Germany,Denmark,United Kingdom,Germany
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2018-09-30
Duration (months): 60.0
Total project cost (EUR): 7724726.52
EU funding (EUR): 5999982.25
Participants: 10
Project status: CLOSED

The aim of CoMMiTMenT is the development of combined imaging technologies based on optofluidic microscopy and scanning ion conductance microscopy. An integrated, completely simultaneous device named µCOSMOS will be used as a proof-of-principle for therapeutic interventions in rare anaemias. Both techniques rely on molecular detection: optofluidic microscopy relies on molecular biomarkers, and scanning ion conductance microscopy on functional imaging of molecular structures. µCOSMOS allows for the exploration, diagnosis and development of therapeutic interventions for several rare anaemias, including hereditary xerocytosis, overhydrated hereditary stomatocytosis, familial pseudohyperkaliemia, cryohydrocytosis, certain types of spherocytosis, hereditary spherocytosis, sickle cell anaemia, thalassemia and phosphofructokinase deficiency. The non-invasive nature of the combined imaging technologies, which probe the function of molecular effectors, facilitates the testing of medications and their dosage in a personalised manner. For some of the rare anaemias, CoMMiTMenT will provide an initial proof-of-principle, whereas for others, such as sickle cell anaemia, personalised medical interventions should become available and will be tested in clinical practice within the project. CoMMiTMent will ensure an effective therapy with diminished undesirable adverse effects, largely replacing splenectomy. CoMMiTMenT includes clinical partners and builds a strong relationship with the European Network for Rare and Congenital Anaemias (ENERCA). Furthermore, CoMMiTMenT acts as a bridge between the technology-driven SMEs and ENERCA and, as such, supports the competitiveness of Europe in this area. The applications will advance (personalised) treatment of rare anaemias and, therefore, contribute to the goal of the International Rare Diseases Research Consortium (IRDiRC) to deliver novel diagnostic modalities and 200 new therapies for rare diseases by 2020.

Keywords: rare anaemias,ion conductance scanning microscopy,optofluidic microscopy,red blood cells,channelopathies