Our 240 professionals are committed every day to developing gene therapy products to treat rare diseases.
80 Unique Roles Driving Gene Therapy Forward
Research, clinical development, bioproduction, partnerships, cross-functional roles… our professions are at the crossroads of science, technology, and purpose.
Joining us means committing to a mission of public interest within a laboratory renowned for its unique model. It means becoming part of a demanding, open, and supportive team where every talent matters.
Together, we transform research into concrete solutions for rare diseases.
Academic and translational research teams
Genethon’s academic and translational research is structures around four laboratories:
- neuromuscular diseases and gene therapy,
- progressive muscular dystrophies,
- genome editing,
- immunology and liver diseases.
Their shared mission is to develop proof of concept for innovative gene therapies.
These teams combine complementary expertise—genetics, pathophysiology, vectorology—to design innovative therapeutic strategies.
They also study immune responses, develop new vectors, and leverage cutting-edge approaches such as gene editing, omics and single-cell analysis, artificial intelligence, and advanced imaging— all to advance gene therapy for the benefit of patients.
Technological development
Our technology development team designs and improves gene therapy manufacturing processes.
Their mission is to make production more efficient, reliable, and accessible by optimizing vectors, ensuring precise quality control, and reducing treatment costs.
From laboratory research to large-scale manufacturing, they support every step of the process, working closely with our industrial partners such as Yposkesi to ensure clinical batches meet the highest standards.
Clinical and Regulatory Affairs
Our clinical and regulatory teams oversee the development of gene therapies, from first-in-human administration to advanced trial phases.
They coordinate clinical operations, ensure patient safety (pharmacovigilance), and ensure compliance with regulatory requirements in France and internationally.
Program Management
The Program Management Department coordinates all therapeutic projects, ensuring their progress at each stage of development.
It facilitates cross-functional collaboration between scientific, regulatory, clinical, and industrial teams, and manages partnerships with external partners (CROs, GMP bioproduction, etc.).
Through rigorous and agile project management, it ensures that the development of each treatment follows a clear path, from the laboratory to clinical trials.
Business Development & Partnerships Department
To accelerate the development of its programs, Genethon relies on strategic partnerships with public and private actors in France and abroad.
The management team coordinates three complementary areas of expertise:
- Business development, which develops targeted collaborations to fund specific gene therapy programs;
- Grant office, which identifies and secures public funding necessary for innovation;
- Intellectual property, which protects and promotes research outcomes.
Working closely with scientific, legal, financial, and project teams, this department plays a central role in turning scientific breakthroughs into concrete solutions for patients.
Cutting-edge technology platforms
Genethon relies on cutting-edge technology platforms to evaluate gene therapies before they enter clinical trials, in line with ethical and regulatory standards.
The preclinical and translational evaluation platform brings together complementary expertise to analyze, validate, and document the efficacy and tolerance of treatments on appropriate models.
The bioinformatics platform supports data analysis and interpretation across departments.
Genethon also hosts a human cell and product bank, certified according to ISO 20387. As Europe’s leading bank for genetic diseases, it provides high-quality human cell and product banking services to the scientific community.
Support functions
Beyond scientific research, support functions play an essential role in the smooth running of our laboratory. Human resources, communication, finance, information systems, legal, quality, general resources, health, safety, and environment: all these areas of expertise support and structure our projects on a daily basis.
In constant contact with scientific teams, they ensure a smooth, secure working environment that complies with national and international standards. Their commitment allows everyone to focus on their mission : serving innovation and patients.
