HOME  THE CLUSTER Focus areas Biotechnologies |
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Molecular & cellular therapies |
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Living material is a key industrial resource and contributes significantly to current pharmaceutical and therapeutic developments (more than 60 % of the drugs in clinical development are biotech-derived). It has been estimated that the world market for industrial applications of genome engineering will exceed €5 billion by 2010 and will then double in the following 10 years.
Molecular & cellular medicine draws together the main medical applications of biological and genetic engineering techniques. It covers gene therapy, genome surgery and cell therapy, along with new diagnostic technologies and methods for the molecular characterization of biological samples. Its technological foundations extend from pharmacogenomics and recombinant protein production to drug targeting and cell culture techniques.
Many of these approaches are recent and still in the development or clinical evaluation phases but will become widespread over the next few years.
The Paris Ile-de-France region is particularly well placed in this field, since it benefits from:
- an abundant, high-level technology offering, materialized by a range of facilities and services and underpinned by an immediately-accessible academic and industrial fabric.
- integrated expertise for clinical assessment and investigation, adapted to the specific nature of the various therapeutic fields concerned, easily accessible for businesses via a single point of contact.
In view of its size, patient resources, hi-tech capability and renowned research groups, the Paris Ile-de-France region's network of university medical centers has attained the critical mass needed to seize a world-leading position in molecular & cellular medicine.
Global leadership in genome engineering
The Paris Ile-de-France region is home to the leading genome engineering company (Cellectis) and numerous innovative and emblematic players involved in biological engineering such as Myosix, Bioprotein Technologies or Eucodis, developing powerful know-how in animal engineering for biomanufacturing and molecular & cellular engineering.
Several Ile-de-France-based players in the highly strategic vector/targeting field
A number of outstanding issues in molecular & cellular medicine concern vectors and targeting techniques. Whether it is a matter of targeting a particular tissue, adapting molecular or viral vectors or more generally administering macromolecules to cellular targets, this is often a rate-limiting step.
The Paris Ile-de-France region features internationally-known specialist players in this field, with notably Centelion (ex-Gencell); this Sanofi-Aventis subsidiary occupies a key position and has significant production capacity for pharmaceutical-grade inert vectors. The Genethon institute at Evry produces viral vectors. In addition, several players (such as Diatos, Immuno-Designed Molecules and Monoclonal Antibodies Therapeutics) are developing major vector- or antibody-based targeting technologies.
Strong positioning in cell therapy
Cell therapy (whether in the R&D phase, in pre-clinical development or already clinically applied) is strongly present in the Paris Ile-de-France region. A range of players master the production, culture, controlled differentiation and reimplantation techniques. Indeed, cell therapy is already an industrial and clinical reality. Genzyme (associated with Myosix for its cardiac cell therapy trials in the Paris Ile-de-France region) intends to evaluate other therapeutic applications for myoblasts. Equally, Genethon and companies like ABCys and Cell Tissue Progress master culture technologies for stem cells (including embryonic cells). The Human Stem Cell Research Institute (INSERM U421) also constitutes a research base which is unique in France.
The presence of the headquarters of the French Blood Agency (EFS) with its nationwide cell therapy and cell engineering infrastructure completes the Paris Ile-de-France region's capability. Lastly, pre-clinical evaluation is facilitated by the presence of the Mondor and Montsouris experimental surgery centers, the Alfort Ecole Veterinary School and a number of INRA (National Agronomic Research Institute) research units.
Powerful resources in genomics
Paris Ile-de-France abounds with major private- and public-sector technical resources for structural and functional genomics. The region concentrates unique know-how in genotyping, genomic analysis and pharmacogenomics and in the industrialization of these techniques (arrays, high throughput/high information content multiplex screening, support IT and statistically intelligent bioinformatics, etc.), notably at the Genopole campus in Evry but also in many R&D-based small companies.
The region can also rely on major players in this field, such as Sanofi-Aventis, Nautilus, Genethon and the National Sequencing Center, together with specialist start-ups like Biomethodes, ImStar, Genomic Vision, Genewave, Genescore and Serial Genetics.
First-rate clinical research facilities and access to large patient cohorts, notably in rare diseases
A number of recent successes and spectacular world clinical firsts have been performed in the Paris Ile-de-France region and promoted by the AP-HP, notably via Professor Alain Fisher's group on gene therapy for severe combined immunodeficiency syndrome, Professor Klatzmann's group on gene therapy for cancer and Professor Menasche's group on cardiac cell therapy.
In fact, almost 60% of France's clinical investigation centers (run by the INSERM/AP-HP or university medical centers) for biotherapies (i.e. molecular & cellular medicine) are located in the Paris Ile-de-France region: the Necker, St Louis and Pitié-Salpêtrière and Mondor Hospitals, and the Gustave Roussy Institute. Furthermore, these CICs are reinforced by 8 clinical research units. For rare diseases, 24 of France's 34 Rare Disease Reference Centers are located in the Paris Ile-de-France region, which naturally facilitates access to patient cohorts.
Lastly, the French Muscular Dystrophy Association (AFM, Association Française contre les myopathies), the Genethon and the Institute of Myology constitute a unique critical mass focused on muscle disorders and featuring a range of technical assets and rare biological resources. This accumulation of resources, know-how and expertise is an attractive proposition for projects dealing with any of the muscle disorders covered.
Training programs which are unique in France (and probably in Europe)
The Universities of Paris V, VI & VII offer scientific courses covering all the classic disciplines. Paris XI, XII & XIII (as well as Evry University and Versailles University) have long favored targeted, Masters-level training; methodologies in clinical research (Paris XI), the fundamental basics of biotherapies (Paris XII-Evry), biotherapy exploitation (Paris XII), surgical sciences (Paris XI-XII) and business aspects of biotherapy (Paris XIII).
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