Celladon Licenses mSCF For Gene Therapy
Clinical stage biotech firm Celladon Corporation announced that it has signed an exclusive, global license from Enterprise Partners Venture Capital for the membrane-bound form of the Stem Cell Factor gene (mSCF) as treatment for cardiac ischemia. The license agreement will allow Celladon to use mSCF in gene therapy applications.
Stem Cell Factor is an important cytokine which plays a role in cell migration and survival as well as survival of cardiac stem cells. Scientists from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully used mSCF gene therapy to undo heart damage in animal models after myocardial infarction. According to the study’s senior investigator Roger J. Hajjar, Director of the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai, “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.” The findings were published in Circulation Research.
The biotech firm revealed that it intends to pursue further preclinical development in myocardial infarction. Dr. Krisztina Zsebo, CEO of Celladon, said that mSCF gene therapy holds the potential to be as potent as stem cell therapy but without complications in cell development. “We're delighted that this promising program will be taken forward by Celladon… 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,” she said.
The company recently announced a new clinical development initiative for its lead product candidate Mydicar in patients with end-stage renal disease (ESRD) undergoing surgery for arteriovenous fistula (AVF) creation in preparation for hemodialysis. The lead drug will also be investigated in patients with advanced heart failure with systolic dysfunction. The company said it is planning a series of trials for Mydicar.