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HEDoctoral network2022–2026

GetRadi · Gene Therapy of Rare Diseases

Horizon Europe — Marie Skłodowska-Curie Actions

Duration
2022-09-01 → 2026-08-31
EU contribution
€2,785,810
Participants
15
Scheme
HORIZON-TMA-MSCA-DN

Lines connect the coordinator with its partners.

Results in brief

Gene Therapy of Rare Diseases

A rare disease is a disease that affects less than 1 in 2 000 people. However, since more than 6000 rare diseases are known, approx. 300 Mio people are suffering from rare diseases worldwide, including 30 Mio in the EU alone. Not surprisingly, the EU recognised rare diseases as an important unmet health problem. Most of these rare diseases are caused by a genetic mutation and thus, a genetic therapy based on gene augmentation or, ideally, direct gene correction, is the only possibility to offer a permanent cure. Therefore, the overall goal of our network “Gene Therapy of Rare Diseases” (GetRadi) is to train 10 Early Stage Researchers (ESR) to become exceptional experts in gene therapy of rare diseases. In their research projects, the ESR will aim to overcome major technical challenges that still prevent the widespread use of somatic gene therapy in the clinic. All projects are embedded in a tight and synergistic academic-industrial collaboration to develop marketable products for the application of gene therapy. This GetRadi training will allow young scientists to spearhead future research efforts in gene therapy of rare diseases and thus increase the speed that desperately needed novel gene therapies are developed. GetRadi has 3 scientific ojectives: 1. Develop and test new methods overcoming current limitations for transfer of genome editing tools into target cell 2. Develop and test innovative modifications or alternatives to classical CRISPR genome editing. 3. Establish models to quantify specific safety risks and develop methods and equipment to reduce gene therapy related risks

Data: CORDIS, © European Union

Project objective

More than 300 million people worldwide are suffering from more than 6000 rare diseases. Nearly all of these rare diseases are caused by a single inherited mutation and cannot be treated effectively. Repair of the defective gene by gene editing is the only possible curative therapy. However, only for very few rare diseases such gene editing therapy has reached the clinic. The “Gene therapy of Rare Diseases” (GetRadi) consortium aims now to contribute strongly to the establishment of more gene therapies for rare diseases. This will be accomplished by training of future leaders in gene therapy of rare diseases preforming ambitious research projects, with the following objectives: (i) Improving transfer of genome editing tools to target cells, (ii) improving gene editing efficiency, and (iii) improving safety of gene therapy. Strong participation of the pharmaceutical industry to research projects and training, development of novel in vitro and in vivo models for rare diseases to test gene therapies in relevant settings, and application of several unique genome editing tools developed by the applicants to the treatment of rare diseases are hallmarks of the network. GetRadi brings together strong industrial beneficiaries (AstraZeneca, Miltenyi Biotec) supervising 3 ESR, highly innovative academic beneficiaries supervising 7 ESR, 3 additional industrial partners, and 4 student enrolling universities. Professional outreach training with access to widely used social media channels will be provided by the partner “European Consortium for Communicating Gene and Cell Therapy Information”. Complementary unique expertise that is spread efficiently by network-wide training and secondments, excellent quality of supervision and intersectoral interactions, innovative transferable skill training, and efficient consortium management will result in an exceptional training of future leaders in gene therapy of rare diseases.

Original text from CORDIS.

Participants

  • KOBENHAVNS UNIVERSITET · KOBENHAVNCoordinatorDenmark
  • ACADEMISCH ZIEKENHUIS LEIDEN · LeidenNetherlands
  • ALBERT-LUDWIGS-UNIVERSITAET FREIBURG · FreiburgGermany
  • ASTRAZENECA AB · SodertaeljeSweden
  • CHARITE - UNIVERSITAETSMEDIZIN BERLIN · BerlinGermany
  • HELSINGIN YLIOPISTO · HelsinkiFinland
  • MAGLE CHEMOSWED HOLDING AB · MALMOSweden
  • MAX DELBRUECK CENTRUM FUER MOLEKULARE MEDIZIN IN DER HELMHOLTZ-GEMEINSCHAFT (MDC) · BerlinGermany
  • MILTENYI BIOTEC BV & CO KG · Bergisch GladbachGermany
  • Synvolux Therapeutics BV · LeidenNetherlands
  • THE UNIVERSITY OF EDINBURGH · EdinburghUnited Kingdom
  • UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II · NapoliItaly
  • UNIVERSITAETSKLINIKUM FREIBURG · FreiburgGermany
  • UNIVERZITA KARLOVA · Praha 1Czechia
  • USTAV MOLEKULARNI GENETIKY AV CR V.V.I. · Praha 4Czechia

Links

Data: CORDIS, © European Union