Originally published on June 26, 2014

AveXis, Inc., a synthetic biology platform company, announced today that the first patient in the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type I has now been enrolled and dosed by Nationwide Children’s Hospital. This trial utilizes chariSMA, the gene therapy product developed by Dr. Brian Kaspar, of Nationwide Children’s Hospital, and licensed to AveXis for development and commercialization. chariSMA utilizes AAV9 to deliver the functional SMN gene to patients. In preclinical studies, AAV9 has been shown to cross the blood-brain-barrier, delivering SMN to motor neurons and significantly improve survival and motor function in an animal model of SMA.

“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 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 visit www.clinicaltrials.gov.

Cure SMA’s Role in This Project

  • In 2010, we awarded $100,000 to Dr. Brian Kaspar at Nationwide Children’s Hospital for systemic gene therapy development
  • In 2012, we awarded Dr. Kaspar $750,000 to advance a CNS-delivered gene therapy to treat older and bigger SMA patients
  • In 2013, NINDS awarded $3.8 Million to Dr. Kaspar in collaboration with Cure SMA to advance the CNS-delivered gene therapy to IND

Please visit our therapeutic approaches page for more information on gene transfer and on our role in this project.

Cure SMA would like to thank all those who have contributed funding for this particular program, including special gifts from The Michael and Chandra Rudd Foundation, The Miller McNeil Woodruff Foundation, and The Jacob Isaac Rappoport Foundation.