Rare diseases and gene therapy
3 European H2020 organizations list this as part of their work.
Most active in this area
- ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
Europe's largest hospital system providing clinical trial infrastructure, patient cohorts, and biomarker validation across liver disease, vaccines, rare diseases, and AI-driven diagnostics.
“Active in Solve-RD (European Reference Networks for unsolved rare diseases), RECOMB and CureCN (AAV-vector gene therapy), and GENE FOR CURE.”
FR72 projects - FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA
Barcelona hospital research institute specializing in stroke, clinical trial platforms, rare diseases, and translational nanomedicine with direct patient access.
“Participated in RECOMB (gene therapy for SCID), EJP RD (European rare disease programme), and contributed to rare disease data sharing and FAIR principles initiatives.”
ES46 projects - MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER
Israeli hospital research fund specializing in RNA biology, Crohn's disease mechanisms, gene therapy, and rare disease diagnostics across European consortia.
“RECOMB focuses on stem-cell based gene therapy for SCID, while EJP RD contributes to European rare disease research infrastructure.”
IL9 projects