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idw - Informationsdienst
Wissenschaft
The European Rare Diseases Research Alliance (ERDERA) is holding today, October 28, its official launch event, where it will set out its vision and ambition for tackling rare diseases as part of the EU’s research strategy in this field. Representatives of the European Commission, patients’ organizations and ERDERA project groups will present their plans at the event. Michael Schmück-Henneresse, a researcher at the Berlin Institute of Health at Charité (BIH), leads a project group aimed at promoting the success of advanced therapy medicinal products (ATMPs), in particular in vivo gene therapies, through a better understanding of immune responses to these therapies.
ERDERA launched in September 2024, with an estimated budget of €380 million and the goal of improving the lives of 30 million rare disease patients in Europe and beyond. To optimize the diagnosis, prevention and treatment of rare diseases, the alliance aims to strengthen scientific collaboration across Europe and align research and health data.
There are nearly 8,000 known rare diseases, and new ones are discovered every year. So, despite the rarity of the individual diseases, the total number of people affected is high: an estimated 5 percent of the world’s population. Yet the majority of rare and ultra rare diseases still lack effective therapies. In addition, patients often experience a long diagnostic journey – visiting many different doctors over many years – before receiving an accurate diagnosis. About 40 percent of patients are initially misdiagnosed, and many more never learn what is actually wrong with them.
To address these issues, the European Rare Diseases Research Alliance (ERDERA) has been set up to build on the advancements made by former EU- funded projects such as SOLVE-RD, ERICA, RESTORE and the European Joint Programme for Rare Diseases (EJP RD).
Combating rare diseases with novel therapies
Michael Schmück-Henneresse, head of the BIH Research Group on Experimental Immunotherapy at the BIH Center for Regenerative Therapies, is co-leading a project group of the ERDERA initiative, along with Mitchell Thorn from Pfizer. Their aim is to develop innovative diagnostic platforms that can evaluate therapeutic success and immunogenicity when treating rare diseases, especially in the field of gene therapy. They also seek to improve technologies, procedures, standards and tools in order to promote the safety and efficacy of advanced therapy medicinal products (ATMPs).
The project group specifically aims to develop advanced molecular and cellular platforms for monitoring immune responses to ATMPs, especially in the field of in vivo gene therapy, employing in vitro and in vivo models for this purpose. The goal is to ensure that these platforms are robust enough to capture rare immune events and responses to gene therapy vectors, RNA-based treatments and therapeutic proteins. Strategies will also be implemented to prevent or mitigate unwanted immune responses.
“Through standardized tests for evaluating immune responses to novel therapies, we are laying the foundation for reliable comparisons between laboratories,” says Schmück-Henneresse. “Our feasibility studies will identify specific challenges in the characterization of rare immune events, providing a basis for formulating strategies to prevent unwanted immune responses.”
The advanced platforms will help harmonize standards across Europe while making therapies for rare diseases safer and more effective. This will lead to reduced treatment failures and facilitate the approval of innovative treatments. The collaboration among research institutes, industry partners and patients’ organizations will expand the possibilities of novel therapies and benefit not only patients but the entire healthcare system.
Additional links
Further information about ERDERA can be found at https://erdera.org/.
Information about Michael Schmück-Henneresse’s research group can be found at https://www.bihealth.org/en/research/research-group/schmueck-henneresse-lab-expe....
Contact
Press Office
Berlin Institute of Health at Charité (BIH)
Konstanze Pflüger: +49 (0)30 450 543 343
Katharina Kalhoff: +49 151 5757 9574
Ole Kamm: +49 152 2561 0126
pressestelle-bih@bih-charite.de
http://www.bihealth.org
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About the Berlin Institute of Health at Charité (BIH)
The mission of the Berlin Institute of Health at Charité (BIH) is medical translation: transferring biomedical research findings into novel approaches to personalized prediction, prevention, diagnostics and therapies and, conversely, using clinical observations to develop new research ideas. The aim is to deliver relevant medical benefits to patients and the population at large. As the translational research unit within Charité, the BIH is also committed to establishing a comprehensive translational ecosystem – one that places emphasis on a system-wide understanding of health and disease and that promotes change in the biomedical translational research culture. The BIH was founded in 2013 and is funded 90 percent by the Federal Ministry of Education and Research (BMBF) and 10 percent by the State of Berlin. The founding institutions, Charité – Universitätsmedizin Berlin and Max Delbrück Center, were independent member entities within the BIH until 2020. Since 2021 the BIH has been integrated into Charité as its so-called third pillar. The Max Delbrück Center is now the Privileged Partner of the BIH.
https://www.bihealth.org/en/notices/the-fight-against-rare-diseases-erdera-unvei... To the press release
https://erdera.org/ Further information about ERDERA
https://www.bihealth.org/en/research/research-group/schmueck-henneresse-lab-expe... Further information about Michael Schmück-Henneresse’s research group
Symbolic image: rare diseases
BIH/Ole Kamm
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