14 early career researchers (ECRs) from 10 countries joined drug repurposing experts near Montserrat, Barcelona, for the 2025 REMEDi4ALL Hackathon tackling real-world challenges in drug repurposing for rare neurodevelopmental disorders (NDDs): CDKL5 deficiency disorder, PACS2 syndrome, and Dravet syndrome.
The REMEDi4ALL Hackathon is designed to empower ECRs with a deep understanding of the entire drug repurposing pathway from early discovery to market access and delivering treatments to patients. Over the course of the week, participants worked in multidisciplinary teams to identify potential repurposing candidates for a rare NDD, develop an enhanced Target Product Profile (TPP), and explore elements of a Repurposing Development Plan (RDP).
“We’re hoping to create a new perspective for researchers and scientists to connect lab or computer work with actual people in the real world. We want attendees to see the whole journey and think about how they develop drugs, not just from the early scientific concept, but right through to find a route to patients.”
— Rick Thompson, CEO, Beacon for Rare Diseases
The Scientific Programme
The week began with Repurpolis a bespoke educational game – developed by Rosan Vegter (EATRIS Scientific Lead Rare Diseases and Regulatory Advisory) with the support of the REMEDi4ALL and EOSC-Life projects. Through trial and error, participants experienced the complexities of navigating the drug repurposing pipeline from discovery to market authorisation.
Following this, teams explored key drug discovery tools including the REMEDi4ALL in-silico drug repurposing catalogue and Chemotarget’s SafetyVista to identify potential repurposing candidates for their assigned diseases, assessing factors such as drug safety and efficacy.
The Development Programme: Patient-Centred Drug Repurposing
Participants then shifted focus towards patient-centred drug repurposing. Plenaries from Beacon for Rare Diseases and Syreon Research Institute explored the importance of patient engagement and early health technology assessment (eHTA) while members of EATRIS led discussions on the importance of the TPP/RDP for drug repurposing candidates. Attendees also had insightful sessions with patient organisations CDKL5 UK, ApoyoDravet, and the PACS2 Research Foundation.
“One perspective that I would love for them to walk away with is how important and how impactful it can be to be engaging with patients from very early on.”
— Dalma Hosszú, Syreon Research Institute“We want participants to make sure that the therapy they find and develop will actually reach the market and, ultimately, the patient.”
— Rosan Kreeftmeijer-Vegter, EATRIS
These conversations encouraged participants to refine their TPPs and RDPs with patient perspectives in mind, leading to meaningful shifts in understanding.
“I learned that some of the symptoms that we were really concerned about on the scientific side might not be the most important ones on the patient side.”
— Thales do Valle Moreira, Hackathon participant
Final Progress and 2025 Hackathon Winners!
As the week progressed, the intensity increased. On Day 4, teams integrated market access and regulatory considerations into their plans, supported by expert consultations with REMEDi4ALL Scientific Lead, Don Lo.
On the final day, participants presented their work to peers and a panel of experts. After deliberation from the panel, the team exploring PACS2 syndrome was crowned the winner.
“I wasn’t expecting to be so invested! Our team was doing everything they can to get the best result possible.”
— Aron Schulman, Hackathon participant
“The friends I have made here and the work we have done in multidisciplinary teams is something that is fulfilling and encouraging for the future.”
— Sarah Berger, Hackathon participant
“Being more engaged with patients and with this community to get more knowledge and experience and bring that to our work.”
— Bruna Clemens, Hackathon participant
Looking Ahead
The REMEDi4ALL Hackathon continues to empower a new generation of researchers to approach drug repurposing with both scientific rigour and patient-centred vision.
Interested in learning more about REMEDi4ALL’s training opportunities? Explore the REMEDi4ALL Repurposing Academy.
