About GeneH

Cutting-Edge Innovation and Research in Gene Therapy

Targeting rare genetic disorders, a cross-sectoral consortium involving academia, industry, public authorities and civil society is dedicated to delivering safe, effective and accessible gene therapy across Europe.

An Excellence Hub in Gene Therapy fostering Life-Changing Treatments

Our vision is to address existing challenges and accelerate the clinical translation of advanced therapies by creating a supportive and flexible environment for gene therapy initiatives.

Challenges: Moving Beyond Clinical Setting

The journey from conceptualization to commercialization of gene therapy products is complex, involving multiple steps from research to GMP-grade production. Despite recent successes, accessibility remains limited.

COST
COST
High production costs discourage investments and development of gene therapies, thus securing cost-effectiveness is crucial.
REGULATION
REGULATION
Regulatory issues often hinder investments and consequently affect patients’ access to treatments. Flexible and responsive regulatory policies, combined with ethical oversight, are a key to making progress.
FRAGMENTATION
FRAGMENTATION
There is a need of strategic and aligned coordination of resources as funding, expertise and infrastructure, to enable a meaningful joint impact.

4-Helix Approach

A strong, collaborative ecosystem is vital to tackle challenges in gene therapy industry.

Sustainable Growth: Delivering Impact Across Sectors

For researchers

Shared research infrastructure, collaborative research environment supported by a broad network of partners, joint programs, mobility.

For industry

Innovation pipelines, development of market-oriented strategies, sustainable deployment of gene therapies.

For policymakers

Estruturas baseadas em evidências.

For Patients

Participation in research, better access to emerging therapies.