EPTRI integrated services

Provided by EPTRI


EPTRI provides integrated services for research developments through its five Thematic Research Platforms according to their specific research area of expertise:
1. PAEDIATRIC MEDICINES DISCOVERY, which relates to the early-stage discovery and development of druggable molecular targets specific to paediatric disease, as well as discovery of new drugs specific for the paediatric population.
2. PAEDIATRIC BIOMARKERS, performing research to identify, characterise and validate new biomarkers for diagnosis, prognosis and personalised therapy in paediatric diseases; provide access to/deposit of annotated paediatric biological samples.
3. DEVELOPMENTAL PHARMACOLOGY, concerning studies on the pharmacological characteristics of drugs and related body response, based on the knowledge of growth and development/maturation of the organs and functions
4. PAEDIATRIC MEDICINES FORMULATIONS, which develops appropriate age-specific formulations and adequate delivery systems for preterm neonates, infants, toddlers, children and adolescents.
5. PAEDIATRIC MEDICAL DEVICES to cover the gap in medical devices tailored for children addressing the changes in growth and psychosocial maturation, physiology, and pathophysiology, and avoid inappropriate repurposing of adult technologies.

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Brokerage service Connection application

Provided by SOLVE-RD

Rare Diseases Models & Mechanisms – Europe (RDMM-Europe): This brokerage service will catalyse connections between people discovering new genes in rare disease patients and basic scientists who can study those genes and pathways in model organisms. Solve-RD brokerage service are to:
*provide functional validation of human genetic variants that cause disease;
*supplement clinical disease gene discoveries by generating functional data so as to get higher impact publications;
*develop insights into potential rationale for treatment (e.g. identification of candidate drug targets) via knowledge of a disease gene pathway; and
*establish longer term collaborations between basic scientists and clinicians that will lead to subsequent grant funding in support of outstanding basic and/or applied research

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Services for research, clinical trials and technology transfer



CIDSTEM has a GMP-authorized facility for the implementation of advanced therapies and the technical and professional skills to manage the GMP laboratory and clinical trials of these therapies.
It therefore possesses the know-how and instrumentation necessary to offer companies:
-an implementation service for research and clinical trials, approved by regulatory bodies, of advanced therapies involving cell cultures in GMP;
-A consulting service for the technological transfer of clinical and preclinical trials in GMP and for the production of cell cultures for advanced therapies.
-An implementation service of research aimed at the industrialization of cell cultures for the development of ATMPs;
-a testing service of new technologies (nanomaterials, scaffolds for the realization of 3D cellular tissues, laboratory equipment, software for clinical trials and for the management of biological data, GMP compliant logistics, etc.).

Translated with www.DeepL.com/Translator (free version)

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Expert mentoring service for rare diseases researchers



Within the EJP RD, EATRIS offers an expert mentoring service for rare disease researchers. Advancing promising technologies and therapies towards clinical application is a complex undertaking. Academic research often lacks easy access to regulatory, therapy development expertise, manufacturing, and healthcare practice, leading to high failure rates. The menotirng prgrammes prvides menotirng on
Translational feasibility
Regulatory compliance
Product classification
Quality assurance and control
Intellectual property strategy
Suitability of analytical readouts
Therapy development

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Directory of professionals and expert centers

Provided by ORPHANET


Orphanet provides information on professionals whose activity(ies) in the field of rare diseases is (are) registered in Orphanet. This includes, for example, consultant and physicians in charge of an expert centre, biologists in a laboratory, researchers, representatives of patient organisations, coordinators of networks, principal investigators of clinical trials, managers or contact person of registries and biobanks, and experts writing or peer-reviewing data for Orphanet.

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RaDiCo supports the implementation of national programs in any country that would need it, by sharing its expertise in database management and implementation of national cohorts on rare diseases. Beyond its own projects, RaDiCo promotes the commitment of French actors to European and international projects needing the implementation of borderless cohorts. We advise and support you for the setting up of propositions and for the management of your programs in this field..).

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transnational access Calls focussed on pathogenic agents



To facilitate Transnational access (TNA) to ERINHA’s member facilities, ERINHA periodically opens a call for applications. Free of charge TNA includes administrative & logistical support, remote use of ERINHA high containment facilities, and technical & scientific support.
Transnational Access is either:
– In person/on-site, and provided to selected Users that visit the facilities, on the necessary conditions that the users have appropriate background and successfully complete the required training
– Remote, through the provision of remote scientific services to selected Users
Note: Remote access will systematically apply for in vivo experiments

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