EJPRD Mid monitoring meeting 2023

On November 28th and 29th, 2023 the European Joint Programm for Rare Diseases (EJPRD) Mid monitoring meeting took place in Luxembourg. All 18 EJPRD therapy development 2021 funded projects were presented. The CHARLIE project presented by prof Clara van Karnebeek on behalf of our consortium was well-received by the panel as well as colleague scientists and aptient organisations.

The EJPRD panel provided valuable feedback on our CHARLIE project. Compliments were given for the comprehensive overview of our strategy to target 2 rare diseases with one intervention, the motivation, progress and results so far, as well as risk management strategy and workplan going forward. Also collaboration between partners and patient organisations were regarded as CHARLIE's strengths. Finally questions were asked regarding organ targeting and rescue of enzym restoration. We will address this going forward, united in our efforts to deliver therapies for PDE and GA1.

Background information

In December 2019 the European Joint Programme on Rare Diseases (EJP RD) launched its second Joint Transnational Call (JTC2020) co-funded with the European Commission to fund multinational research projects focusing on “Pre-clinical research to develop effective therapies for rare diseases”. The aim of the call was to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with a clear benefit for patients.

Following a two-step submission and evaluation procedure, 18 consortia with a foreseen budget of about 24.5 Mio€ were selected for funding, including ~2.7 Mio€ of co-funding from the European Commission.

This Midterm Monitoring Meeting for projects funded under JTC2020, was intended for following-up on the progress and outcomes of the funded projects, with the aid and feedback from both scientific and patient experts. Furthermore, EJP RD aimed to bring together researchers from the different consortia funded under this call, together with scientific and patient experts in the rare diseases field, as well as funders, to facilitate exchange and discussion and enhance collaboration amongst the rare diseases research community.

Finally, this meeting provided an opportunity to expose the funded researchers to the various additional tools and services that EJP RD provided to accompany their research endeavours by participating in an interactive workshop on therapy development support organized by EATRIS.

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This project has received funding from the European Union’s Horizon 2020 research and innovation programme under the EJP RD COFUND-EJP N° 825575.


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