Gamida Cell announces feasibility study with Weill Cornell Medical College

Published December 7th, 2009 - 04:02 GMT

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


    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


    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

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