AveXis Announces Opening of Enrollment for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
AveXis, Inc., a clinic-ready synthetic biology platform company, announced that the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 has now formally begun enrollment at Nationwide Children’s Hospital.
This trial will utilize chariSMA, the gene therapy product developed by
Dr. Brian Kaspar, of Nationwide Children’s Hospital, and licensed to
AveXis for development and commercialization.
“I'm thrilled to see chariSMA translate from the bench to the bedside and am hopeful that we will be able to make a difference for SMA patients,” said Dr. Brian Kaspar.
“We are quite excited about achieving this key milestone and that Nationwide Children’s Hospital can now begin formally recruiting and enrolling candidate subjects for this trial,” said Mindy Du, Director of Scientific and Corporate Affairs at AveXis. The clinical trial is 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 details on the trial, enrollment criteria, eligibility and contact information please see the complete posting, which can be found at http://clinicaltrials.gov/ct2/show/NCT02122952.
“I'm thrilled to see chariSMA translate from the bench to the bedside and am hopeful that we will be able to make a difference for SMA patients,” said Dr. Brian Kaspar.
“We are quite excited about achieving this key milestone and that Nationwide Children’s Hospital can now begin formally recruiting and enrolling candidate subjects for this trial,” said Mindy Du, Director of Scientific and Corporate Affairs at AveXis. The clinical trial is 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 details on the trial, enrollment criteria, eligibility and contact information please see the complete posting, which can be found at http://clinicaltrials.gov/ct2/show/NCT02122952.
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