JERUSALEM, Israel, December 2 /PRNewswire/ -- Gamida Cell announced today a feasibility study evaluating the preclinical (animal study) safety and efficacy of a gene therapy product which combines technology from Weill Cornell Medical College and Gamida Cell. The product may have potential to treat inherited anemias such as Cooley's Anemia.
Gamida Cell is developing a series of technologies to expand, ex vivo, populations of stem/progenitor cells. The company's lead product, StemEx(R), comprised of expanded populations of adult stem cells derived from umbilical cord blood (UCB), is used today as an experimental treatment for blood cancers like leukemia and lymphoma (see ExCell trial at http://www.stemexstudy.com).
Using lentivirus-based gene transfer, Weill Cornell is researching methods to successfully transfer intactglobin genes, coding for functional hemoglobin proteins, into hematopoietic (blood) stem cells with the goal of developing a cure for beta-thalassemia, generally known as Cooley's anemia and Sickle cell anemia. These are the most commonly inherited diseases.
"The current treatment for Cooley's and sickle cell anemia is chronic blood transfusion, which is used to prevent an otherwise likely fatal outcome. Stem cell-based gene transfer, an ex vivo procedure commonly known as transduction, offers a potential means to cure these diseases through the permanent integration of potentially therapeutic genes into the hematopoietic stem cells of the patient. The limitation of this therapy is the low number of transduced stem cells. Expanding the number of transduced stem cells in vitro before reinfusion offers the possibility of creating a more effective therapy and consequently, a potential cure. Using Gamida Cell's expansion technology, we hope to transfer a substantively increased number of stem cells, transduced with an intact globin gene coding for a functional hemoglobin protein, through an expanded population of hematopoietic stem cells, large enough to make a therapeutic impact," said Dr. Stefano Rivella, assistant professor of genetic medicine in pediatrics at Weill Cornell Medical College.
"This initiative is of great importance as it could lead to some revolutionary breakthrough science. It also demonstrates yet another example of how Gamida Cell's proprietary technology can complement other existing technologies to potentially find cures for deadly and chronic diseases," said Tony Peled, chief scientific officer and vice president of research & development of Gamida Cell.
In the event the effort is successful, Gamida Cell and Weill Cornell will have the option to enter into further co-development and commercialization agreements.
About Gamida Cell
Gamida Cell Ltd. is a world leader in stem cell expansion technologies and therapeutic products. The company is developing a pipeline of products in stem cell transplantation and in tissue regeneration to effectively treat debilitating and often fatal illnesses such as cancer, hematological, autoimmune and ischemic diseases. Gamida Cell's therapeutic candidates contain populations of adult stem cells, selected from non-controversial sources such as umbilical cord blood and bone marrow, which are expanded in culture. Gamida Cell was successful in translating these proprietary expansion technologies into robust and validated manufacturing processes under GMP. Gamida Cell's flagship product, StemEx, is now being studied as a therapy for patients with blood cancers in an international pivotal trial at leading transplant centers in the U.S., Europe and Israel. StemEx has orphan drug designation in the U.S. and in Europe. Gamida Cell's current shareholders include: Elbit Imaging, Biomedical Investment, Israel Healthcare Venture, Teva Pharmaceutical Industries, Amgen, Denali Ventures and Auriga Ventures. For more information, please visit: http://www.gamida-cell.com. For information regarding the ExCell study: http://www.stemexstudy.com.
CONTACT: Press contact: Marjie Hadad, Media Liaison/IR, Gamida Cell,