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Gamida Cell Announces Feasibility Study With Weill Cornell Medical College
Date:12/2/2009

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 gl
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