GeneH

Building Bridges in Gene Therapy - From Research to Patients

A cross-border collaboration between emerging centres of excellence in gene therapy in Portugal and Slovenia to accelerate the journey from research to treatment. By uniting clinical researchers, industry, hospitals, policymakers and patient advocacy groups, GeneH is building a strong European network to bring innovation and accessibility of gene therapy across Europe.

Crossing Barriers 

Although revolutionary and groundbreaking in its nature, gene therapy accessibility remains limited by its demanding journey from research to patients. Fragmented research ecosystems, high production costs, regulatory hurdles and limited cross-sector collaboration slow responsible and ethical growth in innovation and clinical applications.

Committed to Accelerate Progress and Access

Joint Research
Fostering strong collaboration between academia and industry to drive innovation and bridge gaps in translational research.
Clinical Readiness
Transitioning to Good Manufacturing Practice (GMP) production and conducting preclinical toxicity studies to prepare for clinical trials.
Education Programs
Formar a próxima geração de especialistas em terapia génica através de workshops especializados, programas de mobilidade entre Portugal e a Eslovénia e programas de doutoramento específicos.
Policy Collaboration
Working with policymakers to build supportive ethical frameworks and streamline regulatory pathways for gene therapy.
Business Integration
Partnering with industry to ensure market-driven strategies, commercial viability, and scalability of therapies.
Patients Engagement
Embedding patient engagement and ethical considerations at every stage of research and development to ensure responsible innovation throughout the process.

Establishing a strong 4-helix network

Discover value in our cross-border shared commitment to scientific and innovation excellence in gene therapy.

Get to Know Our Ecosystem

1. Researchers & Clinicians

Engaging in cutting-edge research & development with access to advanced training and mobility opportunities between Portugal and Slovenia. Collaborating within a multidisciplinary, cross-border research environment supported by a broad network of partners

2. Biotech & Pharma

Co-developing next-generation gene therapies through innovation partnerships. Receiving early regulatory guidance and support. Exploring opportunities for manufacturing scale-up and global market entry and/or expansion.

3. Policy Makers

Leveraging real-world data to inform evidence-based policy. Co-creating ethical and regulatory frameworks to guide gene therapy solutions. Benefiting from a replicable cross-border “Excellence Hub” model to support and inform wider EU health innovation strategies and policies.

4. Patients & Public

Shaping research priorities through the direct involvement of patient advocacy groups. Contributing to greater public understanding of gene therapy through clear and transparent communication open events and educational materials. Improving access to treatments for rare diseases and other conditions.

Pilot Projects Ongoing

GeneH is focused on developing gene therapies for 3 rare genetic disorders with no effective treatment available yet.

Spinocerebellar Ataxia Type 3 (SCA3) / Machado-Joseph Disease (MJD)

A neurodegenerative disorder that causes progressive loss of coordination, muscle control, and balance. Coordinated by the University of Coimbra (UC) in Portugal.

CTNNB1 Syndrome Deficiency

A neurodevelopmental disorder characterized by developmental delays, intellectual disability, muscle tone abnormalities, speech impairment, and distinctive behavioural and physical features. Led by the National Institute of Chemistry (KI) in Slovenia in collaboration with the CTNNB1 Foundation.

Kleefstra syndrome

A developmental disorder marked by intellectual disability, severe speech delay, low muscle tone, distinctive facial features, often accompanied by autism spectrum behaviours. Driven by KI in collaboration with IDefine Foundation and other EU collaborators.

Partners

A team of scientists, business professionals, policymakers, and patients, working side by side

GeneT
CTGCT
CNC-UC
CIBB
University of Coimbra
National Institute of Chemistry
IPN
Biocant
Bluepharma
SIG-EEIG
JAFRAL
BioSistemika
ULS Coimbra
APAHE
ZOPS
CCDRC
RRA LUR

Education & Resources

Training Programs
Workshops, courses, PhD/Master’s integration, Webinars, E-learning.
Mobility Opportunities
Opportunities for exchange between Portugal & Slovenia.
Publications
GeneH Toolkit & Materials
Reports & Deliverables

GeneH Events

GeneT
26.03.2026
Symposium Brain in Action – From Endogenous to Induced Brain Plasticity
GeneT
14.04.2026
ESGCT Spring School 2026
GeneT
30.01.2026
CiBB Seminar | Gene Therapy Ecosystem at the University of Sheffield: A Translational Case Study
GeneT
26.01.2026
PDBEB Courses 2025/2026 - Principles and Practices in Drug Development

Latest News & Media

02 February, 2026
GeneH: Paving the Way for New Gene Therapies in Europe
10 December, 2025
Communication with society is as important as research itself | Interview
22 September, 2025
Gene therapy in focus at CTGCT Days 2025: “GENE H Conference – Gene Therapy”
07 August, 2025
GeneH ask Academia and Industry about Gene Therapy