Did anyone notice that one of the leading firms in the "Bubble Boy" cure was a subsidiary of AVXT? The firm, Génopoïétic, was purchased by AVXT in August, 2000.
Here's a backgrounder that I found at europa.eu.int
<<Approximately one in ten of us will develop an inherited genetic disease at some time in our lives. There are approximately 2,800 known conditions that are caused by defects (mutations) in one or more of our genes. Many of these mutations cause a particular biomolecule to be absent or ineffective in the body. One approach for treating such genetic diseases is gene therapy: that is replacing the defective gene with a normal working gene so that the missing biomolecule is produced and the disease is corrected. This requires using a carrier known as a "vector" to transport the therapeutic gene into the cells of the body.
A success story
Five infant boys suffering from a rare genetic disease were recently successfully treated with a gene therapy by the team of Alain Fischer at the NECKER Hospital in Paris. This project was supported by INSERM, APHP and AFM (Association Française contre les Myopathies) in collaboration with Génopoïétic. These immunodeficient patients were extremely prone to infection and had to live inside a sterile bubble. Indeed this disease is so severe that patients usually die if they are not treated within the first year after birth. Conventional treatment consists of bone marrow transplantation, an invasive and expensive procedure, which requires a compatible donor and has a survival rate of only 60 to 90% with half of all patients still requiring life-long treatment. Compatible donors must be found and bone marrow transplants must be carried out very quickly. These five infants would presumably not have received transplants in time and without gene therapy they would most likely not have survived.
Cells were extracted from the patients' bone marrow and exposed to a virus vector carrying the missing, healthy gene. The virus entered the cells and the healthy gene thus became part of their genetic material (DNA). The cells were then re-injected into the patients' blood stream. After just one month, the missing cells, that fight infections, were detected in the patients' blood and after three months they were able to leave their sterile bubbles. After five to six months the children were able to return home. Most encouragingly, one of the boys even managed to fight off a bout of winter flu.
The question is what made these trials so successful where others have failed?
The company
The company Génopoïétic has produced the viral vector developed by the team of Alain Fischer that has enabled these five infants to be treated. Indeed, in 1993, Génopoïétic was founded with the aim of developing and producing more efficient and safer vectors for use in gene therapy. Since its creation Génopoïétic has grown substantially and is now located in Lyon (France). The main research is done in collabortion with the Pierre & Marie Curie University at the Pitié-Salpêtrière Hospital in Paris. The workforce has also increased, from just one employee at the beginning to 25 experts with the know-how covering all areas of gene therapy. Génopoïétic is now one of the few companies in the world that is able to master all aspects of gene and cell therapy technologies that are required for the development of these novel class of treatments. It has recently engineered new original gene therapy vectors after expanding GMP (Good Manufacturing Practice) production facility.
The founders
Génopoïétic was founded by two academic scientists with the help of the Pierre & Marie Curie University, Paris to increase the value of the university's patents: Professor David Klatzmann, one of the first scientists to work on gene therapy in 1988, and Professor Jean-Loup Salzmann, who moved into the field in 1993. Through his participation in the start-up of a biotechnology company by his university, David Klatzmann gained extensive expertise in commercial exploitation, learnt to use basic research for clinical application and acquired kowledge about industrial vector development. He is also a university
professor and a hospital practitioner in immunology at the Pierre & Marie Curie University, the coordinator of the Gene Therapy Unit at the Pitié-Salpêtrière Hospital, Paris and the director of the CNRS (French National Centre for Scientific Research) research unit.
Génopoïétic and the EC
Following several successful participations in EC-funded projects, Génopoïétic participated in a further research contract with the European Commission in 1999 to allow it to continue with its invaluable research and development. There is a very large number of genetic diseases that could be treated by gene therapy and Génopoïétic's aim is to produce versatile therapeutic systems that can be adapted to any disease and to any gene. However, for this to be possible, effective and, most importantly, safe vectors must be developed. For clinical application of this technology, the genetic make up of the virus must be modified and redesigned so that it serves its clinical purpose, with no or minimal side effects. Thus, Génopoïétic is using EC funding to study and to construct optimal viral vectors in the laboratory before carrying out clinical trials.
The take-over
In August 2000, the American company, AVAX Technologies Inc., signed a Stock Contribution Agreement with Génopoïétic S.A. and its corporate affiliates, who became wholly owned subsidiaries of AVAX. The combined multinational company will continue to specialise in cell and gene therapy. As part of the take-over bid, AVAX acquired all the licenses for the patents held by Génopoïétic and retained the company's exclusive research agreement with the Pierre & Marie Curie University. It is unusual for American biotechnology companies to invest in research and development in France. This take-over will serve to strengthen the development of AVAX through its collaboration with the Pierre & Marie Curie University.
Future prospects
The current aim is to develop novel targeted vectors for use in the treatment of autoimmune diseases and even cancers. The possibility of treating cancers is particularly exciting - the idea is to construct viral vectors that specifically recognise tumour cells, but not normal healthy cells. Side effects should thus be minimal, a compelling argument in favour of this approach when compared to chemotherapy and radiotherapy. Trials are already underway for gene therapy and autologous vaccine approach to cure liver, ovarian, and skin cancer. With their unique patented technologies and team of experts, Génopoïétic has the potential technology and know-how to cure many more genetic diseases.>>
Marc |
