Project description DEENESFRITPL New pharmacological treatment for Down’s syndrome Down’s syndrome (DS) is caused by an extra copy of all or part of the long arm of human chromosome 21. DS causes lifelong cognitive impairments (CIs) which have a significant negative impact on the individuals, their families and society. However, there are currently no approved pharmacological treatments for CIs in people with DS. The EU-funded ICOD project aims to fill this gap by providing a feasibility demonstration of a new therapy for CIs in DS. It will perform first-in-humans and efficacy clinical trials with a new molecular entity (AEF0217), which reverses CIs in animal models of DS. AEF0217 belongs to a new pharmacological class developed by the biotech Aelis Farma: the Signaling Specific Inhibitors of the Cannabinoid (CB1) receptor (CB1-SSi). ICOD will enable a safe and efficacious pharmacological treatment of CIs in people with DS. Show the project objective Hide the project objective Objective Cognitive disabilities in individuals with Down syndrome (DS), also known as Trisomy 21, have no approved pharmacological treatment and represent a significant burden on DS individuals, their families and the healthcare system. The ICOD project will deliver a clinical proof of concept for a new therapy of cognitive deficits in DS by performing first in humans (phase I) and clinical efficacy (Phase II) studies with AEF0217. AEF0217 is a new chemical entity which offers a unique opportunity to treat cognitive deficits in DS. AEF0217 targets the CB1 receptor, a hyperactivity of which has been recently linked to cognitive deficit in DS. AEF0217 belongs to a new pharmacological class, named signalling specific inhibitors of the CB1 receptor (CB1-SSi), which allows to reverse cognitive deficit in animal models of DS. Because of its innovative MOA, AEF0217 is able to reduce selectively a hyperactivity of the CB1 without modifying the basal activity of this receptor and consequently without inducing behavioural side effects. The overarching goal of the ICOD project is to make this innovative first-in-class drug available for DS individuals 7 years after clinical development initiation. Fields of science natural sciencesbiological sciencesneurobiologycognitive neurosciencemedical and health sciencesbasic medicinemedical geneticsmedical and health sciencesbasic medicinepharmacology and pharmacy Keywords Down syndrome cognitive deficits signaling specific CB1 receptor inhibitors clinical drug development safety efficacy first-in-human-study Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) SC1-BHC-08-2020 - New interventions for Non-Communicable Diseases Call for proposal H2020-SC1-BHC-2018-2020 See other projects for this call Sub call H2020-SC1-2020-Two-Stage-RTD Funding Scheme RIA - Research and Innovation action Coordinator CONSORCIO MAR PARC DE SALUT DE BARCELONA Net EU contribution € 1 435 167,00 Address Paseo maritim 25-29 08003 Barcelona Spain See on map Region Este Cataluña Barcelona Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 115 250,00 Participants (6) Sort alphabetically Sort by Net EU contribution Expand all Collapse all AELIS FARMA France Net EU contribution € 3 347 172,50 Address Institut francois magendie, 146 rue 33077 Bordeaux See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Nouvelle-Aquitaine Aquitaine Gironde Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Associazione Oasi Maria SS. Onlus Italy Net EU contribution € 392 500,00 Address Via conte ruggero 73 94018 Troina See on map Region Isole Sicilia Enna Activity type Other Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 INSTITUT JEROME LEJEUNE France Net EU contribution € 292 500,00 Address 31 rue galande 75725 Paris See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Ile-de-France Ile-de-France Paris Activity type Research Organisations Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 CENTRE HOSPITALIER UNIVERSITAIRE SAINT ETIENNE - CHU France Net EU contribution € 261 250,00 Address 42055 Saint etienne See on map Region Auvergne-Rhône-Alpes Rhône-Alpes Loire Activity type Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 SERVICIO MADRILENO DE SALUD Spain Net EU contribution € 31 250,00 Address Plaza carlos trias bertran 7 28020 Madrid See on map Region Comunidad de Madrid Comunidad de Madrid Madrid Activity type Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments) Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO LA PRINCESA Spain Net EU contribution € 230 000,00 Address Diego de leon 62 1 planta 28006 Madrid See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Comunidad de Madrid Comunidad de Madrid Madrid Activity type Research Organisations Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00
