The Global Innovation Competition 2030 aims to "bring out the talents and future champions of the French economy by identifying and supporting the growth of French and foreign entrepreneurs whose innovative project has a particularly strong potential for the French economy" said the Elysee at launch.
Gene therapy is an area with high medical and economic potential. Indeed, this innovative therapy, which involves replacing a diseased gene with a functional gene, has increased the therapeutic success for rare diseases (affecting 3 million people in France, 30 million in Europe) than for common diseases. Future needs can therefore involve tens of thousands of patients a year. The challenge is to industrialize the manufacturing processes of gene therapy vectors, these "carriers" of gene-drug complex to produce. The objective of Généthon program called IGT, aims to develop a generic method for the production of lentiviral vectors to target industrial. Clinical studies have indeed demonstrated the reliability of these vectors, their efficiency and enhanced security compared to their precursors retroviral (see press release of April 21, 2015 "New gene therapy success in a rare disease of the immune system: Wiskott-Aldrich syndrome"). Therefore, this process will produce a large scale, in pharmaceutical terms that is to say, according to Good Manufacturing Practices (GMP), developing a specific business model. It is with this objective that the World Innovation has allocated $ 1.298 million euros at Genethon IGT program that responds to public health issues and economic development on high value-added industry competition and that with a view to mastering the production of innovative biotherapies in France.
"This award is a real pride and recognition of the expertise of our laboratory in the field of gene therapy and its industrial applications. Généthon represents the excellence of French research and plays a major role in the development of a essential sector of innovative biotherapies for the benefit of patients, and high economic value" said Frédéric Revah, CEO of Généthon.
A pride widely shared by Laurence Tiennot-Herment, President of AFM-Telethon and Généthon: "Since the first hour, the AFM-Telethon has chosen innovation. Because everything had to be invented, because for our patients there was urgency, rare diseases have been the catalyst for therapeutic innovation for the benefit of the greatest number. We are very proud to receive this award today and, through it, the trust and State involvement (ou the State's commitment) in this difficult and costly fight (ou action) against rare diseases."
About AFM-Téléthon - http://www.afm-telethon.fr
The French Muscular Dystrophy Association (AFM) federates patients with neuromuscular diseases and their parents. Thanks in great part to donations from France's annual Telethon (€89,3 million in 2013), the AFM-Telethon has become a major player in biomedical research for rare diseases in France and worldwide. It currently funds about 30 clinical trials in different genetic diseases affecting the eye, blood, brain, immune system, and muscles... Thanks to its Généthon research lab, the AFM-Telethon stands out through its unique ability to produce and test its own gene-based medicines.
About Généthon - http://www.genethon.fr
Created by the AFM-Telethon, Généthon's mission is to make available to patients innovative gene therapy treatments. Having played a pioneering role in deciphering the human genome, Généthon is today, with more than 200 scientists, physicians, engineers and regulatory affairs specialists, an international research and development center for preclinical and clinical gene therapy treatments for rare diseases. Généthon has the largest site in the world for GMP production of gene therapy products, Généthon Bioprod. In 2012, Généthon was the first associative laboratory to receive the 2012 Prix Galien for Pharmaceutical Research (France). As part of a therapeutic program on genetic diseases of the blood and immune system, Généthon working for over 10 years on gene therapy of Wiskott-Aldrich syndrome. The laboratory is currently conducting clinical trials for this disease in Europe, Paris and London, and the United States with the Children's Hospital Boston.
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Development of an industrial process for producing large scale lentiviral gene therapy vectors
Gene therapy is an area with high medical and economic potential. Its development is now limited by the industrialization needs of vectors manufacturing processes, the "means of transport" gene-drug complex to produce. The objective of the program conducted by IGT Genethon is to develop an industrial production process of large-scale gene therapy vectors kind of lentiviral (LV).
Lentiviral vectors: reliability and efficiency
Lentiviral vectors have emerged as powerful vectors, versatile and enhanced security compared to their retroviral precursors. Studies show that they are the most effective for gene transfer in stem cells and they transmit the functional gene to stably target cells. No complications were observed in clinical studies. Their effectiveness has been demonstrated in 3 families of rare and common diseases:
- Genetic diseases: ex vivo transduction of the patient's hematopoietic stem cells: beta-thalassemia, X-linked adrenoleukodystrophy, Wiskott-Aldrich syndrome (see press release of April 21 "title" link)
- Viral diseases: Adoptive transfer of T cells transduced in HIV
- Cancers: Adoptive transfer of T cells expressing a chimeric antigen receptor (CAR) in different types of cancer: leukemia, melanoma, lymphoma, colorectal cancer, neuroblastoma.
More than 50 clinical trials now underway in the world, using lentiviral vectors.
World market for gene therapy all strategies:
$ 9.5 billion in 2018
$ 20 billion in 2023
The IGT process, adds value to the development of gene therapy:
The current vector production process lentiviral available on the market is "artisanal" and applicable only for indications requiring limited amounts of medicine for rare diseases. However, future needs may involve tens of thousands of patients per year (eg sickle cell disease, cancer...).
The process developed by Genethon fulfils several criteria for industrialization: robustness and predictability; reproducibility (yields and quantities); portability (a center to another); economies of scale; efficiency.
Genethon Bioprod expertise, production center for innovative drug therapies
To meet the growing needs of gene therapy clinical grade drugs for human trials (the standards of Good Manufacturing Practice, GMP), Généthon created Généthon Bioprod. Unique in the world, the center is dedicated to the production of gene therapy vectors in sufficient quantity and quality for clinical trials in rare diseases.
- 5000 m² dedicated to biomanufacturing and control of gene therapy products for clinical trials including 2500 m² of classified laboratories and confined
- 4 suites GMP production, 2 suites of production for the transduction of hematopoietic stem cells and aseptic filling two rooms (insulator)
- 200 m2 for the industrialization of production methods
- 600 m2 of Quality Control Laboratory
- Design of the next building HQE targets (High Environmental Quality)
- 15 air handling units to ensure the required level of purity of the process
- Air decontamination
- 3 km circular ducts
- Flows personal and controlled materials
- Computer control of the environment and equipment
- Treatment of liquid waste decontamination station
- 70 experts biomanufacturing, pharmacists, engineers and technicians
20-30 clinical batches / year lentiviral vectors or AAV according processes
About 20 non-clinical batches large scale / year