Next generation advanced therapies to treat highly prevalent and high burden diseases with unmet medical needs

Call Updates

Feb 1, 2022 11:54:52 AM

We recently informed the applicants about the evaluation results for their proposals submitted under this call. The results of the evaluation are as follows:

• Number of proposals submitted (including proposals transferred from or to other calls): 84
• Number of inadmissible proposals: 1
• Number of ineligible proposals: 2
• Number of above-threshold proposals: 41
• Total budget requested for above-threshold proposals: EUR 298.969.154

Oct 4, 2021 12:09:08 PM

Call HORIZON-HLTH-2021-TOOL-06 closed on 21 September 2021. 84 proposals were submitted. The breakdown per topic is: HORIZON-HLTH-2021-TOOL-06-02: 31 proposals

Evaluation results are expected to be communicated on Friday 28 January 2022 at the earliest.

Aug 20, 2021 4:09:36 PM

Although the submission of the document titled ‘Template for essential information to be provided for proposals including clinical trials/studies/investigations/cohorts’ is not mandatory for the call topic you have accessed, if your proposal includes a clinical study (as it is defined in the template), you are encouraged to complete and submit this template as part of your grant application. Please disregard the second footnote in the template.

Jun 23, 2021 10:17:54 AM

The submission session is now available for: HORIZON-HLTH-2021-TOOL-06-02(HORIZON-RIA

Next generation advanced therapies to treat highly prevalent and high burden diseases with unmet medical needs

TOPIC ID: HORIZON-HLTH-2021-TOOL-06-02

Programme: Horizon Europe Framework Programme (HORIZON)
Call: Tools and technologies for a healthy society (2021) (HORIZON-HLTH-2021-TOOL-06)
Type of action: HORIZON-RIA HORIZON Research and Innovation Actions
Type of MGA: HORIZON Action Grant Budget-Based [HORIZON-AG]
Deadline model: single-stage
Planned opening date: 22 June 2021
Deadline date: 21 September 2021 17:00:00 Brussels time

Topic description

ExpectedOutcome:

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination 5 “Unlocking the full potential of new tools, technologies and digital solutions for a healthy society”. To that end, proposals under this topic should aim for delivering results that are directed, tailored and contributing to all of the following expected outcomes:

• Competent authorities, researchers and developers use assays for the valorisation and/or assessment of efficiency, delivery, safety, potency or mode of action of novel advanced therapeutic interventions based on either pluripotent stem cells, genome editing or RNA, that are aligned with regulatory standards.
• Clinicians, researchers and developers test several new advanced therapies based on pluripotent stem cells, gene editing or RNA ready through clinical trials meeting the regulatory requirements.

Scope:

The recent development of advanced therapies has been hampered by the lack of robust research on certain key parameters e.g. safety, upscaling, immunity, potency assays, cost-effectiveness, and early on in development. This topic aims to ensure that the next wave of advanced therapies, based on either pluripotent stem cells, gene editing or RNA, are established in a timely fashion and in accordance with the appropriate regulatory standards for further clinical testing. It will support preclinical research platforms for disorders with high prevalence and burden[1] that tackle the bottlenecks currently encountered in the field, ensuring that promising advanced therapies can reach the market within the next decade. Applicants should justify the disease or disorder to be targeted with its prevalence level, the related burden and unmet needs. Applicants could propose activities in one or several of the following areas, and should take into consideration the Oviedo Convention, eligible actions and ethical principles as defined by the Horizon Europe Framework Programme [2]:

• Method development for the production and differentiation of pluripotent stem cells[3] (defined as cells that can give rise to cells from all three embryonic germ layers), to include defining appropriate potency assays. Complementary activities to assess mode of action, safety, in vivo validation or upscaling procedures could be considered.
• Development and validation of biological assays and methods that can demonstrate efficacy, delivery, specificity, and safety (including off-target effects) of genome editing products in targeted cells and tissues (e.g. base editing, prime editing, transcription activator-like effector nucleases, zinc-finger nucleases, clustered regularly interspaced short palindromic repeats). Complementary activities to assess in vivo validation or upscaling procedures could be considered.
• Development and validation of novel RNA-based therapeutics targeting non-communicable diseases. Complementary activities to assess mode of action, delivery, safety, in vivo validation and/or upscaling procedures could be considered.
• Study, analysis and tackling of different immune responses, taking into account factors like sex and age, generated by any of the above-mentioned advanced therapies in vivo, facilitating regulatory approval for next phase of research and development.

[1]As defined by www.who.int/medicines/areas/priority_medicines/en/

[2]https://data.consilium.europa.eu/doc/document/ST-6692-2021-ADD-3/en/pdf

[3]Definition: Embryonic stem cells and induced pluripotent stem cells are pluripotent stem cells. www.nature.com/subjects/pluripotent-stem-cells.