5th International Conference on Paediatric Liver Disease
From 09 to 10 September 2022
At : Hamburg, Germany
Events
7th International Myology Congress
From 13 to 16 September 2022
At : Nice, France
3rd International Workshop on Klinefelter Syndrome, Trisomy X, and XYY
From 13 to 15 September 2022
At : Leiden, the Netherlands
International Scientific Symposium on the Ehlers-Danlos Syndromes and Hypermobility Spectrum Disorders
From 15 to 19 September 2022
At : Rome, Italy and Online
INTERNATIONAL HUMAN GENE THERAPY CONFERENCE
From 27 to 29 September 2022
At : Online and in Melbourg, Australia
ICPerMed Conference – Prelude to the Future of Medicine
From 06 to 07 October 2022
At : Paris, France
From Paradigms to Paradoxes in Immunity and Immunopathology’ (PPII) international conference
From 07 to 09 October 2022
At : Freiburg, Germany
Innovation Bootcamp in Rare Diseases 2022 (IBRD2022)
On 12 October 2022
At : Brussels, Belgium
8th International Symposium on NBIA
From 14 to 16 October 2022
At : Lausanne, Switzerland
The International Congress on Spinal Muscular Atrophy
From 22 to 24 October 2022
At : Barcelona, Spain
ASHG Annual Meeting 2022
From 26 to 30 October 2022
At : Los Angeles, USA
CLINICAL TRIALS IN RARE DISEASES : A VIRTUAL EVENT
On 08 December 2022
At : Online
Third International Summit on Human Genome Editing
From 07 to 09 March 2023
At : London, United Kingdom
2023 RE(ACT) Congress & IRDiRC Conference
From 16 to 19 March 2023
At : Berlin, Germany
The Translational Science of Rare Diseases - From Rare to Care IV
From 04 to 06 May 2023
At : Tutzing, Germany
Grants
Innovative Health Initiative launches two calls for proposals
The Innovative Health Initiative (IHI) has launched its first calls for proposals, with topics on diseases such as cancer, cardiovascular disease and neurodegenerative diseases, as well as cross-cutting issues like health data and early stage studies of medical devices.
- IHI will contribute up to EUR 135 million to projects funded under IHI call 1. When preparing their budgets, applicants will have to ensure that at least 45 % of the total project costs are met via contributions from IHI private partners and/or IHI contributing partners. IHI call 1 is a single-stage call, meaning applicant consortia will need to include industry / contributing partners who will bring in kind or financial contributions to the project. They will have to submit a full proposal by the deadline: 20 September 2022
- IHI call 2 is a two-stage call, meaning applicant consortia should not include industry / contributing partners. They will have to submit a short proposal by the deadline. The winning consortium will then work with the industry partners that designed the topic and submit a full proposal at a later date. For IHI call 2, the IHI contribution is around EUR 22 million.
Friederichs’s Ataxia research grants: 2022 programme
In order to stimulate research and studies on Friedreich's Ataxia, the French Friedreich's Ataxia Association (A.F.A.F.) will be offering in 2022 grants up to 30.000 €/year/project, for a maximum of two years. The grants should be awarded in November 2022. The fields considered will include the basic aspects of the disease (genetics, biochemistry, neurobiology), clinical studies or more fundamental research in animal models. All the applications must clearly indicate the expected consequences on the treatment of the disease. Projects including therapeutic studies or research aiming at identifying new therapeutic strategies for Friedreich’s ataxia will be particularly encouraged. Projects with a clear impact on the patients’ quality of life will equally be appreciated (e.g., physiotherapy, speech therapy or focusing on vision and hearing problems...) as well as grant applications that foster international collaboration.
Grant application forms are available from AFAF:
Madeleine SCHMEDER
00336 37 47 67 78
Application deadline: August 26th 2022
EIC funding call for RNA-based therapies and diagnostics for rare genetic diseases
The European Commission (EC) has adopted the 2022 work programme of the European Innovation Council (EIC), opening funding opportunities worth over €1.7 billion in 2022 for breakthrough innovators to scale up and create new markets. This includes €60.5 million to tackle three Transition Challenges, notably one on the development of RNA-based therapies and diagnostics for complex or rare genetic diseases.
The EIC Transition 2022 (HORIZON-EIC-2022-TRANSITION-01) call open on 1st March is intended to meet the following specific objectives:
- Advance, beyond the state-of-the-art, RNA delivery methods, including robust mRNA formulations, that would enable effective and safe delivery of mRNA into the cells;
- Design, develop and preclinical validate of novel miRNAs (miRNA lncRNA, tRNA or siRNA-based) therapies for complex or rare genetic diseases;
- Develop and validate novel RNA-based diagnostics and RNA-based predictive biomarkers that would allow for early and more accurate diagnosis and for favourable or non- post-treatment prognosis, respectively.
The starting point in the project should be a preliminary technology or protocol of an RNA-based therapy for complex or rare genetic diseases with unmet medical needs that demonstrates, in a lab or preclinical context, the essential features that underpin the disruptive nature of the innovation. The endpoint in the project should be a completely functional version of the technology suitable for clinical validation, supported by a sound and implementable commercialisation/exploitation strategy.
The funding call has the following deadlines: May 4th and September 28th.
FDA funding opportunity for rare disease research
The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) has announced a grant to support efficient and innovative natural history studies that advance medical product development in rare diseases with unmet needs. The funding opportunity announcement is entitled “Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01) Clinical Trials Not Required”. The purpose of this grant is to address critical knowledge gaps and facilitate rare disease product development.
The receipt dates for this funding opportunity are 15 February 2022 and 13 February 2024.
Courses and educational initiatives
Rare Disease Cures Accelerator- Data and Analytics Platform Workshop 2022
The Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP) will hold a workshop on 13-14 September 2022 in order to present the impact of the platform on drug development and data analysis in rare diseases. The workshop will also address case studies and will include industry expert presentations as well as panel discussions on the importance of building a rare disease community that works together to share optimal data and generate actionable solutions for the benefit of all rare disease patients.
The workshop will be held as a hybrid event, both virtually and in Arlington, Virginia, US.
The deadline for registration is 11 September 2022.
EJP RD Workshop: Quality assurance, variant interpretation and data management in the NGS diagnostics era
The "Quality assurance, variant interpretation and data management in the NGS diagnostics era" course by EJP RD is made up of 3 days of training, organised by the University Hospital in Tübingen (EKUT) and aims at providing researchers and clinicians with specific trainings on the interpretation of genetic variants and quality standards. It will be held online on 19-21 October 2022. The training course is open to the international research community, clinicians, medical specialist, laboratory scientists (EBMG registered), junior laboratory scientists, clinical geneticists, policy makers and assessors for laboratory accreditation, and patient representatives, with a basic knowledge in biology or medicine.
Registration deadline is 15 August 2022 23:59 CEST.
EJP RD MOOC “Diagnosing Rare Diseases: from the Clinic to Research and back” open continuously for enrollment
The MOOC “Diagnosing Rare Diseases: from the Clinic to Research and back” co-developed by EJP RD, the Foundation for Rare Diseases, ERN ITHACA and ERN Genturis in the context of European Joint Programme on Rare Diseases, is now open continuously for enrolment. The MOOC is conducted online and is free for 5 weeks. This course offers an exploration of key questions around rare disease diagnostic research and issues today, as well as insights into patient experiences.
This course offers an exploration of key questions around rare disease diagnostic research and issues today, as well as insights into patient experiences. The topics covered include: the diagnostic process and the types of genetic tests available for rare diseases; the differences in rare genetic diseases patient pathways; the technological advances for diagnostic research; the role of collaborative studies and data sharing in rare diseases diagnosis; the impact of having a diagnosis or lacking a diagnosis on patients' lives; the role and place of physiopathology approaches as well as the social sciences research in the context of rare diseases diagnosis.
EURORDIS Open Academy
The EURORDIS Open Academy provides courses to empower patient advocates with knowledge and skills to take part in patient engagement roles with all stakeholders and to advocate for rare diseases on a European and national level.
Trainings are delivered in English and comprise-learning modules, pre-training webinars and face-to-face/online intensive days.
RareLaunch programme
NORD’s RareLaunch programme provides an accessible educational platform that allows an individual to explore the steps to start a nonprofit and/or to engage in starting or expanding research efforts as a nonprofit leader. Twi courses are available, Forming a Foundation and Research Ready. Forming a Foundation provides education and training for interested patients and caregivers looking to form rare-disease-focused nonprofits. Research Ready supports the efforts of patient organizations to effectively prepare for scientific research and the creation of rare disease registries. Both courses emphasize capacity building and expanding confidence at a pivotal stage in a rare disease community’s growth and development.
EJP RD Funded ERN Training Workshops
The EJP-RD is holding workshops aiming at training ERN researchers and clinicians in relevant innovative themes with a cross-ERN added value. These 2-days ERN workshops are open to interested persons (clinicians/scientists) affiliated to ERNs or Affiliated Partner Institutions.
Training themes may include innovative research methodologies, diagnostic research methodologies, interdisciplinary treatment approaches, such as gene therapy and transplantation, etc. Topics have to be proposed by the ERNs or by investigators belonging to EJP RD beneficiary institutions. Registrations are now open for several workshops.
ERN EpiCare e-learning modules
The ERN EpiCARE has developped new e-learning modules in collaboration with the ILAE Academy. The modules are dedicated towards professionals who wish to practice diagnosis of rare and complex epilepsies. The first online six modules are patient-centered cases.
