GeneH – Center of Excellence to Accelerate Innovation in Gene Therapy
Program: Horizon Europe
The GeneH project is designed to accelerate the development and deployment of gene therapy, particularly for rare genetic diseases. Through a comprehensive approach based on the quadruple helix model, it brings together key stakeholders—research institutions, industry, healthcare professionals, regulators, and civil society—to strengthen the translation of scientific discoveries into clinical practice.
Challenges addressed: Despite significant research breakthroughs, the field still faces challenges such as a limited number of clinical trials, complex regulatory pathways, a lack of specialized infrastructure, and insufficient funding. GeneH aims to overcome these obstacles through targeted interventions.
Project objectives:
- Foster interdisciplinary collaboration among researchers, industry, healthcare providers, regulators, and patient groups.
- Align research with market and societal needs to drive innovation.
- Promote faster and safer clinical translation for patient benefit.
- Secure sustainable funding by attracting public and private investments.
- Develop training and educational programs to enhance the skills of researchers and healthcare practitioners.
- Establish robust ethical and regulatory frameworks to guide the development and use of new therapies .
By integrating these strategies, GeneH will significantly contribute to groundbreaking solutions in gene therapy and reinforce Europe’s position as a leader in the field—ultimately benefiting patients who have yet to gain access to effective treatments (rralur.si).
Project partners and roles:
Slovenia:
- Chemical Institute (KI): Provides excellence in chemistry and biotechnology.
- ZOPS – Alliance of Patient and User Organizations: Represents patient interests and promotes better healthcare services.
- SIH EGIZ (SIH EEIG / SRIP Health – Medicine): Supports innovative development and accelerates research translation.
- Biosistemika R&D: Brings technological expertise in biotechnology.
- RRA LUR (Regional Development Agency): Strengthens regional development through public sector collaboration.
- Jafral R&D: Contributes to technological innovation.
Portugal:
- University of Coimbra (UC): Project coordinator and lead research institution.
- APAHE: Represents patients with hereditary ataxias.
- Biocant: A biotech technology park facilitating innovation transfer.
- Bluepharma: Key pharmaceutical industry partner.
- CCDRC: Fosters regional coordination and stakeholder collaboration.
- IPN: Drives technology transfer and market-oriented strategies.
- Centro Hospitalar e Universitário de Coimbra (CHUC): Vital for clinical translation
Funding:
Total project budget: €4,885,375
RRA LUR’s grant: €140,000
Duration: January 1, 2025 – December 31, 2028