Gene Therapy: a strategic area
of therapeutic innovation
One of the most dynamic therapeutic field
- 53 gene therapy approved, of which 38 in the last 5 years
Multiple therapeutical applications
- Neuromuscular
- Cardiovascular
- Diabetes and metabolism
- Oncology
- CNS (Central Nervous System)
- Infections
- Opthtalmology
- Blood disorders
- Immune disorders
- Vaccines
- Kidney
France at the origins of many breakthrough innovations
- Marketed Products: Zolgensma® | Skysona® | Zynteglo® | Luxturna®
- Technological Contributions: CAR-T | AAV
27
DNA gene therapies
(16 oncology, 9 rare diseases, 2 vascular diseases)*
26
RNA gene therapies
(15 rare diseases, 10 COVID-19, 1 Chronic Fatigue Syndrome)*
+3 000
Products in Clinical Development
*ASGCT Q3 2023
GenoTher has been awarded “Biocluster” label, in the framework of the “France 2030” investment programs, showing France priorization of the development of gene therapy as a strategic area of therapeutic innovation.
GenoTher’s missions
Based in Evry, GenoTher is a biocluster of excellence serving the needs of gene therapy project leaders, helping them to develop new therapies and meet their development challenges.
- Gene Therapy Preclinical platforms
- Vector lab: screening of capsids and preclinical in vitro and in vivo analysis.
- Gene editing lab: quality control of in vivo and ex vivo genome editing projects.
- Lipid Nano Particle formulation lab: screening and non-GMP production of LNP.
- Non viral delivery platform: screening of nanoparticles and non-GMP production.
- Immune response lab: analysis of the immune response against gene therapy products.
- Bioproduction
- Production of non-GMP viral vector batches for preclinical studies.
- Production of clinical and commercial grade GMP viral vector batches.
- Production and quality control of ex vivo gene therapy drugs.
- Clinical trials and Medtech
- Data warehouses gathering specific data on rare diseases and muscle-related disorders.
- Innovative digital tools to capitalize on patient data and optimize clinical trial design and monitoring.
- Platform for histology and biomarker discovery in muscle diseases.
- Medical device development platform for muscle-related disorders.
- Promote and organize curricula dedicated to:
- Research & Development,
- Biomanufacturing,
- Biotech management and Entrepreneurship.
- Incubation and seed funding to assist high potential start-ups in their early stages of development, with a focus on scientific support, business and team development.
- Preparation and support for fundraising.
- Encourage the initiation of ambitious projects, provide equipment and shared services, as well as patient databases and biobanks.
- The creation of an exceptional gene therapy ecosystem is set to significantly attract international talents and industry players, amplifying our global visibility and outreach around all most promising technologies raising in the field. Through groundbreaking research and strategic collaborations, we aim to make a lasting impact on a global scale.
- Development of next-generation gene and cell therapy approaches clinically validated for pathologies currently without therapeutic solutions.
- Disruptive innovation in bioproduction to enable access for a greater number of patients.
- Innovation and sharing of clinical excellence to optimize design and conduct of clinical trials, accelerate the development of therapeutic solutions and optimize the evaluation of treatments in real-life settings.
R&D leader since gene therapy inception.
Founding research and IP for Zolgensma.
13 products in the clinic internationally.
Pioneer US gene therapy company and member of the Roche Group.
Launched the first FDA approved gene therapy for a genetic disease.
38 hospitals covering all medical expertises.
Largest group of university hospitals in Europe
2 GMP platforms.
Owner of million patient data.
1st French biocampus dedicated to genomics derived industrial applications, and company creation.
Worldwide Ranking for Genomics Research.
Dedicated Applied Mathematics and Computer sciences
Robotics for production.
EU’s manufacturing leader in gene therapies.
10,000 m2 of GMP manufacturing capacity.
Among the largest research academic institutions in the world for medicine and biology.
Leader of 3 flagship actions of the national biotherapies-bioproduction strategy.
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The strength of GenoTher ecosystem
2,000+
Scientist
and Clinicians
10,000+
Publications
2,000+
Patents
in the field
50+
Clinical programs
in gene therapy
40+
Licenses
15+
Spinoff created