Joint Transnational Call 2017 – Funded Projects

Joint Transnational Call (JTC2017)

These projects were funded under E-Rare.

General information
Topic: The research projects have to focus on the pre-clinical development of therapeutic approaches in suitable existing animal or cell models.
Budget: 11 milion €
Funded projects: 11
The following parties have decided to open the ninth E-Rare joint transnational call (JTC 2017) for funding multilateral research projects on rare diseases. The call is being opened simultaneously by the parties in their respective countries. In addition, Patient Organisations (PO) – represented in this call mostly by EURORDIS – may also co-fund selected projects based on their mandate and research topic interest:
•    Austrian Science Fund (FWF), Austria
•    Research Foundation Flanders (FWO), Belgium, Flanders
•    Fund for Scientific Research – FNRS (F.R.S.-FNRS), Belgium, French-speaking community
•    Canadian Institutes of Health Research – Institute of Genetics (CIHR-IG), Canada
•    Fonds de recherche du Québec-Santé (FRQS), Québec (Canada)
•    Academy of Finland (AKA), Finland
•    French National Research Agency (ANR), France
•    Federal Ministry of Education and Research (BMBF), Germany
•    German Research Foundation (DFG), Germany
•    General Secretariat for Research and Technology (GSRT), Greece
•    National Research, Development and Innovation Office (NKFIH) of Hungary
•    Chief Scientist Office of the Ministry of Health (CSO-MOH), Israel
•    National Technological Innovation Authority (the “Innovation Authority), Israel
•    Italian Ministry of Health (MoH-IT), Italy
•    Foundation for Biomedical Research and Innovation, Japan
•    State Education Development Agency (VIAA), Latvia
•    National Centre for Research and Development (NCBR), Poland
•    Executive Agency for Higher Education, Research, Development and Innovation Funding (UEFISCDI), Romania
•    National Institute of Health Carlos III (ISCIII), Spain
• Swiss National Science Foundation (SNSF), Switzerland
•    The Scientific and Technological Research Council of Turkey (TÜBITAK), Turkey

  • CureDravet: Curing Dravet Syndrome by Gene Therapy
  • ERAAT: Enhancing Endoplasmic Reticulum Proteostasis to Rescue Alpha1 Antitrypsin Deficiency
  • Cure-AID: IL-18 and MRP neutralization for the treatment of anti-IL-1-refractory autoinflammatory diseases
  • SCA-CYP: Gene Therapy for Cerebellar Ataxias: restoring cholesterol metabolism by targeting brain cholesterol 24 hydroxylase (CYP46A1)
  • EDSCIDPROG: Gene edited lymphoid progenitors for adoptive transfer as a treatment of primary immunodeficiency
  • TREAT-HGPS: Exploring new therapeutic strategies in Hutchinson-Gilford progeria syndrome preclinical models
  • TreatOPON: Preclinical Development of Treatments for OPA1-linked Optic Neuropathies
  • TreatPolyQ: Allele-specific lowering of mutant polyQ proteins as treatment for Huntington disease, spinocerebellar ataxia type 3 and spinocerebellar ataxia type 7
  • MuTaEB : Mutation-targeted gene and pharmacological therapies for dystrophic and junctional Epidermolysis Bullosa
  • TREAT-MTMs: Novel therapies for neuromuscular diseases with altered phosphoinositide metabolism
  • CALSER: The effect of CDNF in ALS and ER stress