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Disease mechanisms and new therapies background information

Therapy development for rare diseases

Increase your impact on healthcare and accelerate the development of rare disease therapies by filling the gap between preclinical and clinical science. You can be part of an enthusiastic team with the ambition to develop Radboudumc as a center for rare diseases and innovative drug therapies.

Below you can read more on background information, our ambitions and our positions. You can also go directly to the
overview of available vacancies for this theme
.
Background
Radboudumc has a strong track record in curiosity-driven research with translational impact. Academic patient care is integrated with innovative research and education with a person-centered focus. A prototypic example of personalized medicine within the care domain involves the strong development and positioning of

Radboudumc Centers of Expertise for rare diseases
. The highly specialized and complex care of rare disease patients is an essential and intrinsic part of our university medical center given our central position in society and the care landscape. Given the fact that ~6% of the population is suffering from a rare disease, the vast majority of which lacks causative treatment options, the development and implementation of novel therapies is an important academic responsibility as well. Innovations in genomic technologies have drastically increased the number of diagnosed patients with rare genetic diseases, allowing the rational design of novel treatments based on the underlying molecular mechanisms. Moreover, improved patient stratification offers novel possibilities for targeted and individualized treatments, for example by use of cell-based therapies in rare hemato-oncological diseases.

Ambitions
Radboudumc will commit to developing targeted and personalized therapies for rare diseases with a focus on disease groups in our expertise centers. The expertise in these centers ranges from rare neurological, neurometabolic and kidney disorders to rare infectious diseases and cancer. Based on the widespread knowledge in our center on disease mechanisms, we focus on gene- and cell-based therapies and drug repurposing.

To this end, Radboudumc will:

• Invest in and further develop the necessary infrastructure, including
• GMP facilities such as clean rooms
• Disease models (towards human research models, organoids)
• Clinical trial facilities including outcome measures and support
• Develop methods to investigate the implementation of person-centered therapies. These include statistics in small groups or n=1 trials as well as the selection of appropriate therapeutic approaches.
• Promote later stage development (TRLs 5-7) of drug candidates by strengthening valorization and establishing Public Private Partnership ecosystems, combined with an in-depth knowledge network on academic drug development. This also involves value-based decision making via therapy development paths.
• Build capacity to train professionals who can connect the worlds of research, drug development and valorization. A new specialization in Therapy Development within the Master of Biomedical Sciences will be developed. Furthermore, entrepreneurship and drug development will be prioritized in educational programs for researchers and professionals.

Allocation of positions
The Sectorplan provides funding for 13 structural positions to support these ambitions. Details about the current vacancies for these positions can be found here. Positions that are not yet on the vacancy list will become available within one month. The positions are centered around four main activities:

1. Improving preclinical infrastructure to accelerate therapy development for rare diseases

• Development and standardization of therapy testing in human organ-on-chip models
• Development of RNA and genetic therapies beyond the preclinical stage
• Development of Advanced therapy medicinal products (ATMPs) including cell-based therapies
• Team:
• 1 associate professor
• 1 assistant professor education
• 2 staff scientists (assistant professor level)
• 2 research technicians

2. Improving clinical infrastructure to accelerate therapy development for rare diseases

• Design of “Therapy development pathways” for drug-repurposing, gene/RNA-based and cell-based therapies and facilitating their access
• Development of methods to investigate the effects and implementation of person-centered therapies, so-called n=1 trials
• Support to start-up and implementation of investigator-initiated treatment strategies and trials for rare disorders
• Team:
• 1 associate professor
• 1 assistant professor education
• 1 project manager
• 2 part time hospital pharmacists/researchers
• 1 trial coordinator/research nurse

3. Developing a knowledge network on academic drug development for rare diseases

• Development of a knowledge network both internally in the Radboudumc and by realizing a Public Private Partnership ecosystem with external partners. This will strengthen the development of late stage TRLs (5-7)
• Team:
• 1 project developer public private partnerships
• 1 community manager

4. Building capacity to train professionals who can connect research, drug development and valorization

• Establishment of a new track on Therapy Development within the Master of Biomedical Sciences
• Integration of entrepreneurship and knowledge on drug development in the educational programs for researchers and other professionals
• Team:
• 2 assistant professors education (also linked to teams 1 and 2)
• 1 coordinator of the track on therapy development in the Master program

The researchers indicated above will also be involved in teaching in these programs
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Project

Sector plan: Medische en Gezondheidswetenschappen
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Sector plan: Medische en Gezondheidswetenschappen

About

The ministry of OCW invests 40 million euros annually to enhance research and education in the sector of medical sciences. Radboudumc has seized this opportunity to strengthen and deepen three of its strategic themes: prevention, data-driven & AI, and disease mechanisms & new therapies.

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About

With the sector plans, the Ministry of OCW (Education, Culture and Science) wants to make a structural contribution to improving the cohesion and cooperation between education and research, and making working as a researcher more attractive by using more permanent contracts. In addition, the sector plans contribute to making joint, sharp choices about division of tasks and profiling between and among universities.

Radboudumc fully supports these objectives and has seized this opportunity to strengthen and deepen three important existing strategic themes within Radboudumc using our share of about 5 million euros a year. These are the themes of 1) prevention, 2) data-driven & AI, and 3) disease mechanisms & new therapies, which fit very nicely with the thematic focus of the sector plan, namely 1) prevention, 2) data-driven innovation and 3) the path from foundation to application.

We hope this will not only give a boost to the research within Radboudumc, but also strengthen the unique position of university medical centers: in no other place is the interaction between fundamental research and the treatment of (complex) patients so clearly present, and the potential for impact on both patients and healthy people so great.

Our goal

We aim to accelerate in three of our strategic themes. We achieve this by hiring about 40 people who have the drive to create impact.
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Our goal

What is our goal?

We aim to accelerate in three of our strategic themes:

• Prevention (lifestyle data and interventions)
• Data-driven (artificial intelligence, e-health and medical technology)
• Disease mechanisms & new therapies (therapy development for rare diseases)

Achieving our goal

We are looking for about 40 people with the drive to create impact and pave the way for accelerating healthcare. This is your chance to be part of this movement and help shape the future of our research and education, creating impact on the lives of many.

Application and selection process

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Application and selection process

We’re searching about 40 people to attain our ambitious goals in the fields of prevent
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