Dallas-based AveXis, a biotech company developing a synthetic biology platform, has enrolled and dosed the first patient in the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1, performed in collaboration with Nationwide Children’s Hospital. The company is testing a gene therapy product called chariSMA for the treatment of spinal muscular atrophy (SMA).
AveXis was granted the license for the development and commercialization of the treatment, which uses a gene therapy product developed by Dr. Brian Kaspar at the Nationwide Children’s Hospital. ChariSMA delivers functional SMN genes to patients using the self-complementary AAV9, which has been proven effective in crossing the blood-brain-barrier to deliver SMN to motor neurons in preclinical studies.
The company expects to improve the survival rate and motor function of SMA patients, as effectively demonstrated in a previous animal model. ”Over the past few months so many individuals, organizations and foundations have worked tirelessly to bring chariSMA from the bench to the bedside. We are thrilled to be at this inflection point and hopeful that the nonclinical results we have seen will be manifested in the clinic,” said John Carbona, CEO of AveXis.
The study, a non-randomized trial that aims to evaluate the safety and efficacy of the intravenous delivery, is being led by Dr. Jerry Mendell at the Research Institute at Nationwide Children’s Hospital. The investigation received Investigational New Drug approval and was nominated Fast Track in September 2013.
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