Welcome to Genetix
Pharmaceuticals
Genetix’ Mission is to develop and commercialize effective gene therapies for the treatment of serious, chronic human diseases and cancers.
Gene Therapy Technology
Genetix’'s key proprietary technology is a next-generation gene therapy vector, a modified lentivirus extensively engineered to be safe for human use. By capitalizing on the unique and powerful attributes of lentiviruses, this technology has achieved unprecedented levels of stable transduction of target cells in multiple in-vitro and in-vivo preclinical model systems, including non-dividing primary human cells.
The planned initial human use for this technology is the transduction of bone marrow stem cells to correct an inherited mutation leading to the development of a fatal childhood malignancy. Affecting thousands of children worldwide, no effective treatments currently exist for this condition.
Functional Genomics
To support its mission in gene therapies and provide a productive source of proprietary therapeutic genes, Genetix has entered into a partnership with the National Genomic Cancer Typing Program of France involving 14 prominent universities. This national, multi-disciplinary, multi-million dollar program is expected to generate a large number of new, therapeutically useful genes in the coming years. Genetix has acquired a broad option for multiple exclusive licenses on all discoveries in the areas of leukemias and lymphomas.
Cardiovascular Therapy
Genetix focuses on the identification of gene vectors and biologics that promote angiogenesis for the treatment of coronary artery disease. An innovative approach has been identified and is currently in advanced preclinical testing.
Research Program in Gene Therapy
LEADER IN GENE TRANSFER AND GENE THERAPY
GENETIX has developed several of the key gene transfer vectors widely utilized by the scientific and business communities for both gene-based discoveries and gene therapy. Our retrovirus-based gene integration system, has been requested by more than 600 laboratories world-wide 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.
DOMINANT TECHNOLOGICAL AND PATENT POSITION ON LENTIVIRAL VECTORS, THE FOREMOST GENE TRANSFER VECTOR OF THE POST GENOMICS ERA
Lentiviral vectors currently represent the single most active area of vectorology for both the American and European Societies of Gene Therapy. Lentiviruses are unique in their ability to permanently integrate therapeutic genes into the chromosomes of both dividing and non-dividing target cells in both in-vivo and ex-vivo settings.
Genetix has developed a heavily modified lentivirus engineered to be safe for human use while retaining its powerful transduction efficiency. In multiple in-vitro and in-vivo assays, this lentivirus has achieved unprecedented levels of target cell transduction as well as long-term gene expression.
Genetix’s initial use for this lentivirus will be the ex-vivo transduction of bone marrow stem cells to correct Fanconi's Anemia, an inherited mutation leading to the development of fatal childhood anemia and malignancies. FA affects thousands of children worldwide, and no effective treatment currently exists.
GENETIX holds an exclusive license for the dominant patent on lentiviral vectors, as well as a series of patent applications covering extensive safety modifications and specific therapeutic uses.
Technology Profiles
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. The initiation and success of these trials will trigger multi-million dollar milestone payments to GENETIX.
A therapeutic antibody that induces normal differentiation of leukemia cells.
GENETIX is currently negotiating an exclusive license for 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.
