HORIZON EUROPE
Europeo
Specific Challenge:Curative or near curative therapies for rare and orphan diseases have been a long-held desire for many in the biomedical research and development arena, including patients. Rare diseases are often very severe, genetically driven illnesses with high morbidity and mortality that place a large burden on families of patients and healthcare systems. Gene therapy and cell therapy provide an opportunity for a curative, single treatment for many of these devastating diseases, eliminating the need for chronic treatment. This topic aims to accelerate the research and development of advanced therapy medicinal products (ATMPs) by filling gaps in our knowledge base in, and tools for, gene and cell therapy. This will provide medicines developers and regulators with the information they need to more swiftly move these potentially transformative medicines forward so that they can benefit patients in need.
Conventional medicinal product characterisation, clinical safety/efficacy, and regulatory requirements already pose challenges to developing treatments for rare monogenic diseases. These challenges are amplified for gene and cell therapies due to knowledge gaps in...
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Specific Challenge:Curative or near curative therapies for rare and orphan diseases have been a long-held desire for many in the biomedical research and development arena, including patients. Rare diseases are often very severe, genetically driven illnesses with high morbidity and mortality that place a large burden on families of patients and healthcare systems. Gene therapy and cell therapy provide an opportunity for a curative, single treatment for many of these devastating diseases, eliminating the need for chronic treatment. This topic aims to accelerate the research and development of advanced therapy medicinal products (ATMPs) by filling gaps in our knowledge base in, and tools for, gene and cell therapy. This will provide medicines developers and regulators with the information they need to more swiftly move these potentially transformative medicines forward so that they can benefit patients in need.
Conventional medicinal product characterisation, clinical safety/efficacy, and regulatory requirements already pose challenges to developing treatments for rare monogenic diseases. These challenges are amplified for gene and cell therapies due to knowledge gaps in our understanding of these ATMPs for viral or non-viral approaches. By addressing these existing knowledge gaps, we hope to accelerate and improve the feasibility of product development and decrease development time and costs to bring effective new advanced therapies to patients. For many aspects of ATMP biology and safety, regulatory agencies have to consider theoretical concerns in this emerging field, largely due to a lack of supporting data and evidence. This can be a major burden for the efficient development of ATMPs.
Scope:The main focus of this topic is to develop a product characterisation framework and methodologies that are limited to the pre-competitive space. Though much of the work will be to understand aspects of gene or cell therapy in general without a particular disease focus, there may be some work that utilises disease models to accomplish the appropriate characterisation. The disease focus will be on non-oncological, monogenic rare diseases. Therefore, this topic intends to utilise both therapeutically relevant systems, as well as model systems that rely on the use of marker transgenes. In order to develop such a framework, there is a need for a coordinated and substantive effort to acquire and analyse the currently available data and then design preclinical and clinical studies to fill the knowledge gaps. This information will help to address gene/cell therapy risks and also guide product developers and regulators to determine and implement an appropriate and effective characterisation framework to enable efficient and safe development of gene/cell therapies.
The main objectives of the topic, intended to address existing knowledge/data and tools gaps focused on viral-mediated gene therapy and cell therapy, are to:
1) develop better, standardised models for predicting product immunogenicity in humans;
2) build our understanding of gene/cell therapy drug metabolism inside a host and explore any loss of efficacy (persistence), particularly with non-integrating viral vectors or cell therapy;
3) understand the clinical factors around pre-existing immunity limiting patient access to ATMP therapy, and adaptive immune responses affecting product safety, efficacy and persistence, including for integrating vectors-based therapies;
4) engage regulators to ensure that the models and data generated through the funded action will provide the necessary information to support regulatory filings and to address regulatory and safety concerns.
Expected Impact:Primarily, the action funded under this topic will fill gaps in our knowledge base around gene/cell therapy host responses, which will allow for the data-driven development of a product characterisation framework to aid researchers, developers and regulators to more rapidly move effective and safe gene/cell therapies forward.
Understanding the host immune responses and the prevalence of pre-existing immunity in humans in broad geographic areas will be instrumental for finding the best immune-modulating regimens, thus increasing patient access to advanced medicines. Understanding the determinants of immunogenicity may enable re-dosing with gene/cell therapy products, while studying the mechanisms of persistence will help to define the optimal age for gene/cell therapy intervention.
Finally, joint efforts across pharma, biotech, academia, and regulatory functions will inform patient inclusion criteria, limit sub-therapeutic dosing, and define the impact of the pre-existing and adaptive immunity on the efficacy and persistence of gene/cell therapy. This broad understanding will help to focus industry resources on actual (not theoretical) risks and will facilitate the harmonisation of regulatory requirements. These improvements will, in turn, enable accelerated cures for rare diseases via a defined regulatory framework.
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Características del consorcio
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La ayuda es de ámbito europeo, puede aplicar a esta linea cualquier empresa que forme parte de la Comunidad Europea.
Características del Proyecto
Los costes de personal subvencionables cubren las horas de trabajo efectivo de las personas directamente dedicadas a la ejecución de la acción. Los propietarios de pequeñas y medianas empresas que no perciban salario y otras personas físicas que no perciban salario podrán imputar los costes de personal sobre la base de una escala de costes unitarios
Los otros costes directos se dividen en los siguientes apartados: Viajes, amortizaciones, equipamiento y otros bienes y servicios. Se financia la amortización de equipos, permitiendo incluir la amortización de equipos adquiridos antes del proyecto si se registra durante su ejecución. En el apartado de otros bienes y servicios se incluyen los diferentes bienes y servicios comprados por los beneficiarios a proveedores externos para poder llevar a cabo sus tareas
La subcontratación en ayudas europeas no debe tratarse del core de actividades de I+D del proyecto. El contratista debe ser seleccionado por el beneficiario de acuerdo con el principio de mejor relación calidad-precio bajo las condiciones de transparencia e igualdad (en ningún caso consistirá en solicitar menos de 3 ofertas). En el caso de entidades públicas, para la subcontratación se deberán de seguir las leyes que rijan en el país al que pertenezca el contratante
Características de la financiación
The IMI2 JU 18th Call for proposals topics text as well as the Call Conditions are available here.
The budget breakdown for this Call is given at the end of the Call topics text, in the Call Conditions section, as well as the following information :
1. Eligible countries: described in article 10(2) of the Rules for participation in Horizon 2020 and in article 1 of the Commission Delegated Regulation related to IMI JU.
2. Eligibility and admissibility conditions: described in the IMI2 Manual for evaluation, submission and grant award. See also the Commission Delegated Regulation related to IMI JU.
Proposal page limits and layout: Please refer to Part B of the proposal template in the submission tool below.
3. Evaluation:
Submission and evaluation process, including evaluation criteria and procedure, scoring and threshold are described in the IMI2 Manual for submission, evaluation and grant award. See also the proposal templates for your specific action in section 5, below.
4. Indicative time for evaluation and grant agreement:
Notification of outcomes of stage 1 evaluations: maximum 5 months from deadline for submitting proposals.
Notification of outcomes of stage 2 evaluations: maximum 5 months from deadline for submitting full proposals.
Signat... Please read carefully all provisions below before the preparation of your application.
The IMI2 JU 18th Call for proposals topics text as well as the Call Conditions are available here.
The budget breakdown for this Call is given at the end of the Call topics text, in the Call Conditions section, as well as the following information :
1. Eligible countries: described in article 10(2) of the Rules for participation in Horizon 2020 and in article 1 of the Commission Delegated Regulation related to IMI JU.
2. Eligibility and admissibility conditions: described in the IMI2 Manual for evaluation, submission and grant award. See also the Commission Delegated Regulation related to IMI JU.
Proposal page limits and layout: Please refer to Part B of the proposal template in the submission tool below.
3. Evaluation:
Submission and evaluation process, including evaluation criteria and procedure, scoring and threshold are described in the IMI2 Manual for submission, evaluation and grant award. See also the proposal templates for your specific action in section 5, below.
4. Indicative time for evaluation and grant agreement:
Notification of outcomes of stage 1 evaluations: maximum 5 months from deadline for submitting proposals.
Notification of outcomes of stage 2 evaluations: maximum 5 months from deadline for submitting full proposals.
Signature of grant agreements: maximum 3 months from the date of informing successful applicants.
5. Proposal templates, evaluation forms and model grant agreements (MGA):
IMI2 Research and Innovation Action (IMI2-RIA) and Innovation Action (IMI2-IA):
Standard evaluation form
Proposal templates are available after entering the submission tool
Proposal template stage 1
Proposal template stage 2
IMI2 Model Grant Agreement
Clinical trial template – the Clinical Trial template is compulsory at stage 2 only !
6. Open access must be granted to all scientific publications resulting from Horizon 2020 actions.
Where relevant, proposals should also provide information on how the participants will manage the research data generated and/or collected during the project, such as details on what types of data the project will generate, whether and how this data will be exploited or made accessible for verification and re-use, and how it will be curated and preserved.
Open access to research data
The Open Research Data Pilot has been extended to cover all Horizon 2020 topics for which the submission is opened on 26 July 2016 or later. Projects funded under this topic will therefore by default provide open access to the research data they generate, except if they decide to opt-out under the conditions described in Annex L of the H2020 main Work Programme. Projects can opt-out at any stage, that is both before and after the grant signature.
Note that the evaluation phase proposals will not be evaluated more favourably because they plan to open or share their data, and will not be penalised for opting out.
Open research data sharing applies to the data needed to validate the results presented in scientific publications. Additionally, projects can choose to make other data available open access and need to describe their approach in a Data Management Plan.
Projects need to create a Data Management Plan (DMP), except if they opt-out of making their research data open access. A first version of the DMP must be provided as an early deliverable within six months of the project and should be updated during the project as appropriate. The Commission already provides guidance documents, including a template for DMPs. See the Online Manual.
Eligibility of costs: costs related to data management and data sharing are eligible for reimbursement during the project duration.
The legal requirements for projects participating in this pilot are in the article 29.3 of the Model Grant Agreement.
Members of consortium are required to conclude a consortium agreement prior to the signature of the grant agreement.
7. Additional documents:
Summary of the most relevant provisions for participating in IMI2 actions
1st Amended IMI2 Amended Annual Work Plan 2019
IMI2 Regulators Guidance tool for researchers
IMI JU derogation to H2020 Rules for Participation
Horizon 2020 Rules for Participation
Horizon 2020 Regulation of Establishment
Horizon 2020 Specific Programme
Información adicional de la convocatoria
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