Technology Profiles
Research Program in Gene Therapy
Effective Gene Transfer is the fundamental technology required for effective gene therapy. Genetix has pioneered the development of retroviral-based vectors for the permanent transduction of target cells. It has been requested by more than 600 laboratories worldwide, including five multi-national pharmaceutical companies. It has been successfully used in several Phase I/II clinical trials while achieving a uniquely flawless safety record.
Genetix' next generation gene transfer vector is based on an extensively engineered Lentivirus. Lentiviruses are unique in their ability to permanently integrate therapeutic genes into the chromosomes of dividing and non-dividing target cells in both in-vivo and ex-vivo settings. This technology can thus be used to target therapeutic genes to pluripotent human stem cells that give rise to blood and other tissues of the body, as well as non-dividing or slowly-dividing mature cell types such as retinal and neural cells, connective tissues including joints, and solid organs such as the liver.
GENETIX’ approach to gene transfer contrasts with all other gene therapy vectors (e.g. adenoviral vectors, non-viral vectors) which result in transient gene transfer and low levels of gene expression, lasting only for a few days or weeks.
The cure of genetic or chronic diseases by gene therapy requires long term, permanent chromosomal integration of the transferred therapeutic gene into the appropriate target cells. Without chromosomal integration, the transferred DNA becomes rapidly diluted and degraded, as evidenced by numerous failed clinical trials of the past decade.
Lentiviral vectors currently represent the single most active area of vectorology for both the American and European Societies of Gene Therapy.
GENETIX has an exclusive license for the dominating patent on lentiviral vectors as well as a series of patent applications covering extensive safety modifications as well as specific therapeutic uses.
Functional Genomics
GENETIX HAS ESTABLISHED A PRECEDENT-SETTING R&D PARTNERSHIP BETWEEN GENETIX AND THE NATIONAL GENOMIC CANCER TYPING PROGRAM OF FRANCE
The government of France, working with leading research institutions across the country, recently inaugurated and financed a national, multi-disciplinary, multi-million dollar program to identify as exhaustively as possible those genes whose alteration directly causes all the known types of human cancers. Because of its unique expertise in gene transfer technology, GENETIX was chosen as its first business partner.
This program is widely expected to generate a large number of new, therapeutically useful genes and drug targets within the next few years. GENETIX has a broad option for multiple exclusive licenses on all discoveries in the areas of leukemias and lymphomas. This will provide Genetix with a flow of proprietary genes which it will use to develop future gene therapies for these important cancers.
Products in Late Pre-Clinical Testing
GENETIX is building a strong pipeline of pharmaceutical products. GENETIX has made in-house discoveries and has also acquired exclusive licenses to several promising candidate targets, which are already in advanced pre-clinical testing as described below.
A new angiogenic protein for the treatment of ischemic cardiac disease.
GENETIX has identified a protein active in promoting angiogenesis as a treatment for coronary artery disease. This compound is expected to enter human clinical trials in the near future. A therapeutic antibody that induces normal differentiation of leukemia cells
GENETIX is currently negotiating an exclusive license to a patent application that covers the use of an antibody against the CD44 receptor for the treatment of acute myeloid leukemias. This antibody is able to induce normal differentiation of leukemic cells. The antibody is currently in advanced pre-clinical testing.