The Dallas-based company’s clinical trial – Gene Transfer Clinical Trial for SMA Atrophy Type 1 – will use chariSMA, the gene therapy product developed by Nationwide Children’s Hospital’s Dr. Brian Kaspar, and licensed to AveXis for development and commercialization.
BioNews Texas has been following AveXis’ research on gene replacement therapy for SMA since July of last year. Most recently, the company closed a financing round with PBM Capital Group, which will help develop the company’s gene therapy platform.
A genetic pathology that attacks nerve cells in the spinal cord, SMA is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons, and is currently responsible for the death of more infants than any other genetic disease.
The clinical trial is being led by Dr. Jerry Mendell, at The Research Institute at Nationwide Children’s Hospital, having received IND approval and Fast Track designation in September 2013.
For further information on the trial, see the complete posting here.