HUGE TECHNOLOGICAL AND
HEALTH CHALLENGES
Innovative therapies represent a new paradigm in human health for the treatment of diseases such as cancer (CAR-T), genetic diseases (gene therapy) or organ regeneration (stem cells). They are based on very complex molecular and cellular biology technics requiring new and specific means of production and characterization.
The technologies implemented, which stem from fundamental research, are developed and adapted for use in human health. They incorporate regulations, which are themselves in constant evolution, guaranteeing both patient safety and the benefits of scientific innovations for the treatment of most serious diseases.
The stakes are even higher because the types of treatment and scientific approaches used are multiple and very different. These include:
• Viral vectors such as rLV and AAV
• Nucleic acids such as messenger RNA
• CAR-T cells derived from genetically modified T lymphocytes
• Stem cells or differentiated pluripotent cells
CUTTING-EDGE SCIENCE
THAT GIVES NEW HOPE
Last hope treatments when conventional protocols have failed, potential of definitive cure, extraordinary benefits for patients... the application is huge and constantly evolving for innovative therapy products.
These treatments require a very tight proximity to the patient, which makes them so-called personalized or tailored medicine. The most emblematic example is the case of autologous product, for which cells are taken from the patient and modified ex-vivo before being reinjected to the same patient in the case of CAR-T treatment for cancers. But a new generation of cell therapy products is emerging. It allows the treatment of more patients in permanent waiting, and uses cells that have been derived from healthy donors and made compatible. This is called allogeneic cell therapy.
Gene therapy products, to repair a deficient or altered gene in genetic diseases such as cystic fibrosis, stand out mainly in the complexity of the viral vector and the diversity of approaches (in-vivo or ex-vivo). Nucleic acids, such as messenger RNA for immunotherapy treatments and obviously the enormous hopes of vaccines, imply the use of advanced technics. These innovative therapies require new kinds of industrial capabilities and facilities, both mass and individualized, and highly complex along the entire value chain, from the sourcing of materials to the final patient.
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Cell or gene therapy, nucleic acids... innovative therapy products are constantly evolving to treat patients
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AN ECOSYSTEM THAT CREATES
PERMANENT INNOVATIONS
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Thanks to the constant progress of science, innovative therapies are a credible answer towards a definitive cure
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Advanced therapy products are quite recent, a few decades since the first treatments of genetic diseases, with a first gene therapy trial in 1995.
The last ten years have seen a tremendous acceleration. Breakthroughs in scientific knowledge, the mobilization of enormous human and financial resources, risk-taking, and close collaboration with regulatory authorities (accelerated procedures for orphan diseases and pandemics) have made it possible to take giant steps forward for the benefit of patients. The goal is a definitive cure, whereas some conventional drugs relieve pain or prolong life.
Replacing a diseased organ with a new one, repairing a gene for ever, inventing new therapeutic technics based on biology or other principles such as magnetic stimulation in diseases of the central nervous system, coupling complementary technologies, potentiating the intrinsic means of defense of the human being as in the case of immunotherapy, developing implants to restore sight... the field of innovative therapies is infinite and will combine multiple scientific disciplines, with biology at the heart.
COMMITTED AND COOPERATIVE INTERNATIONAL STAKEHOLDERS
AND ACTORS
The manufacture of advanced therapy products is governed by the regulations applicable to biopharmaceutical products such as BPF (Europe) or GMP (United States). In Europe, Part IV of Eudralex Vol 4 has been implemented in recent years to adapt the rules to the specificities of this category of products. In recent years, the FDA has also published specific guidelines for these products and their use in the clinical phase and the use still in limited numbers approved for commercial supply.
New and complex, advanced therapy products are positioned in an evolving regulatory framework, favoring a risk analysis approach adapted to these products and their manufacturing processes.
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The regulatory landscape is therefore highly evolving, requiring increased vigilance for companies involved in the development and marketing of these products.
Close collaboration is, and will be, increasingly needed between regulatory authorities, researchers, industry and many professional associations (ISPE, ICH, Biophorum). The goal? Accelerate the availability of innovative therapies for the patients who wait them for so long.
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