BioLife, a synthetic biology platform company soon to be renamed AveXis, has been granted exclusive rights to the spinal muscular atrophy gene therapy program developed at The Research Institute at Nationwide Children’s Hospital and The Ohio State University in Columbus, Ohio.
The license enables the company’s focus on the treatment of children with spinal muscular atrophy (SMA), a motor neuron disease which is the leading genetic killer of children under the age of two. SMA is the second most common pediatric autosomal recessive disorder with an incidence of one in 6,000 live births. SMA is known to be caused by lack of expression of survival motor neuron (SMN) protein due to a genetic loss of the SMN1 gene.
The licensed technology encompasses the unique ability to deliver the SMN gene across the blood brain barrier or through the cerebrospinal fluid to effectively target motor neurons throughout the brain and spinal cord. In pre-clinical efficacy studies in the severe rodent model of SMA, a one-time treatment of the gene therapeutic improved survival from the average lifespan of 14 days to over 400 days. Additionally, the one-time treatment was safe and well tolerated.
AveXis-BioLife has secured the key intellectual property developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. The findings have been led by Brian K. Kaspar, Ph.D. a Principal Investigator at The Research Institute at Nationwide Children’s Hospital and Associate Professor at The Ohio State University College of Medicine along with collaborator Arthur Burghes, Ph.D. Professor at The Ohio State University. Dr Kaspar stated, “We are thrilled to partner our SMA program with AveXis-BioLife to rapidly move our findings to SMA patients and look forward to initiating human clinical trials in early 2014.”
A clinical trial has been planned with Jerry R. Mendell, M.D., director of the Center for Gene Therapy and Principal Investigator in The Research Institute at Nationwide Children’s Hospital and the team recently received approval for an investigational new drug application in September.
“Being in partnership with this brilliant team of people, who are not only compassionate but driven to eradicate this disease, is an honor for our company,” said John A. Carbona, Chief Executive Officer.
The license enables the company’s focus on the treatment of children with spinal muscular atrophy (SMA), a motor neuron disease which is the leading genetic killer of children under the age of two. SMA is the second most common pediatric autosomal recessive disorder with an incidence of one in 6,000 live births. SMA is known to be caused by lack of expression of survival motor neuron (SMN) protein due to a genetic loss of the SMN1 gene.
The licensed technology encompasses the unique ability to deliver the SMN gene across the blood brain barrier or through the cerebrospinal fluid to effectively target motor neurons throughout the brain and spinal cord. In pre-clinical efficacy studies in the severe rodent model of SMA, a one-time treatment of the gene therapeutic improved survival from the average lifespan of 14 days to over 400 days. Additionally, the one-time treatment was safe and well tolerated.
AveXis-BioLife has secured the key intellectual property developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. The findings have been led by Brian K. Kaspar, Ph.D. a Principal Investigator at The Research Institute at Nationwide Children’s Hospital and Associate Professor at The Ohio State University College of Medicine along with collaborator Arthur Burghes, Ph.D. Professor at The Ohio State University. Dr Kaspar stated, “We are thrilled to partner our SMA program with AveXis-BioLife to rapidly move our findings to SMA patients and look forward to initiating human clinical trials in early 2014.”
A clinical trial has been planned with Jerry R. Mendell, M.D., director of the Center for Gene Therapy and Principal Investigator in The Research Institute at Nationwide Children’s Hospital and the team recently received approval for an investigational new drug application in September.
“Being in partnership with this brilliant team of people, who are not only compassionate but driven to eradicate this disease, is an honor for our company,” said John A. Carbona, Chief Executive Officer.
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