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news.Celladon, a clinical-stage biotechnology company applying its leadership position in the field of gene therapy and calcium dysregulation has announced an exclusive, global license from Enterprise Partners Venture Capital for gene therapy applications of the membrane-bound form of the Stem Cell Factor gene (mSCF) for treatment of cardiac ischemia.
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Stem Cell Factor is a critical cytokine which contributes to cell migration, proliferation, and survival of cardiac stem cells.
Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully tested mSCF gene therapy to reverse heart damage following myocardial infarction in animal models.
These results were recently published in Circulation Research and are available on the Company’s website www.celladon.com. "mSCF gene therapy promoted a regenerative response in damaged hearts similar to that observed with stem cell therapy and may be one of the first non-cell therapies to increase cardiac muscle precursors in the heart," said the study’s senior investigator Roger J. Hajjar, M.D., Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai and the Arthur & Janet C. Ross Professor of Medicine at Mount Sinai. "We are excited by the future possibility of testing this gene therapy in patients."
Celladon plans to commence further preclinical work, building on available data from Mount Sinai in myocardial infarction.
"We believe mSCF gene therapy has the potential to be a powerful therapeutic approach, harnessing the potency of stem cell therapy without the associated complications of developing cells as products," said Krisztina Zsebo, Ph.D., Chief Executive Officer of Celladon.
"We’re delighted that this promising program will be taken forward by Celladon, leveraging our extensive gene therapy research and product development experience. Our initial focus will be to generate clinically acceptable gene therapy vectors in support of potentially conducting a future clinical trial in patients who have suffered cardiac damage, as well as exploration for potential other applications."