Project description
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.
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.
Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being
MAIN PROGRAMME
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H2020-EU.3.1.3. - Treating and managing disease
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
RIA - Research and Innovation action
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) H2020-SC1-BHC-2018-2020
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
08003 Barcelona
Spain
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.