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Nýustu fréttir frá FSMA í USA | FSMA á Íslandi
AveXis Announces Dosing of First Patient for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1.
AveXis, Inc., a synthetic biology platform company, today announced that the first patient in the Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 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.
Click here for the entire press release.
Click here for a poster AveXis presented at the Annual SMA Conference with the latest details on the project. .
“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 see the complete posting, which can be found at http://clinicaltrials.gov/ct2/show/NCT02122952.
What has FSMA’s role been in the project?
-In 2010, FSMA awarded $100,000 to Dr. Brian Kaspar at Nationwide Children’s Hospital for SMA gene therapy development.
Please click here to read the announcement.
-In 2012, FSMA awarded Dr. Kaspar $750,000 in drug discovery funding to advance a CNS delivered gene therapy for SMA.
Please click here to read the announcement.
–In 2013, NINDS awarded $3.8 Million to Dr. Kaspar in collaboration with FSMA to advance a CNS-directed gene therapy to IND.
Please click here to read the announcement.
Families of SMA would like to thank all those who have contributed funding to FSMA 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.
Click here to read about the clinical trial process.
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