At UTSA, Brian Kaspar will discuss a revolutionary, single dose gene therapy that has been developed to treat the root cause of spinal muscular atrophy.
(April 4, 2019) -- The UTSA Neurosciences Institute will partner with the UTSA Brain Health Consortium (BHC) to present its 2019 UTSA Distinguished Public Lecture, Translation of Gene Therapeutics in Neurological and Neuromuscular Diseases. At the event, AveXis Scientific Founder and Chief Scientific Officer Brian Kaspar will discuss a revolutionary, single dose gene therapy that has been developed to treat the root cause of spinal muscular atrophy (SMA).
SMA Type 1 is the leading genetic cause of infant mortality around the world. It results from a defect in the SMN1 gene, and about one in 50 Americans are genetic carriers. In the absence of a functional SMN1 gene, infants rapidly lose the motor neurons they need to breathe and swallow. By age two, children who are missing a functional SMN1 gene typically require permanent ventilation.
AveXis, Inc., a Novartis Company, is a clinical-stage gene therapy biotechnology company, focused on the treatment of neurological diseases. It is currently advancing the first SMA gene therapy in human trials. Kaspar in this lecture will discuss his translational research journey.
“This is a transformative approach,” said Jenny Hsieh, the Semmes Foundation Endowed Chair in Cell Biology at UTSA and director of the UTSA Brain Health Consortium. “The success of SMA gene therapy not only saves patient lives, it has opened up the doors to other widespread monogenic disorders.”
Since August 2004, Kaspar has served as a principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and was formerly an associate professor in the Department of Pediatrics and Department of Neuroscience at The Ohio State University College of Medicine. He is the author of more than 100 published articles in peer-reviewed journals and was named a fellow of the American Association for the Advancement of Science in 2013.
This Distinguished Lecture is the 11th in a series of annual lectures hosted by the UTSA Neurosciences Institute since 2009. It is also the first time the Neurosciences Institute will partner with the Brain Health Consortium.
“The lectures bring neuroscientists of exceptional accomplishment to UTSA to share the excitement of discovery in brain science with the entire UTSA community and San Antonio at large,” said Charles Wilson, the Ewing Halsell Distinguished Chair in Biology, and the director of the Neurosciences Institute.
The UTSA Brain Health Consortium is a robust network of 39 researchers in science, engineering and psychology who are using multidisciplinary approaches to better understand the nature of brain disorders and translate new discoveries to improve patient lives. Its four focus areas include stem cells and precision medicine, neuroscience, neuroengineering, and psychology and behavior.
Researchers in the UTSA Neurosciences Institute are investigating the neural bases of experience, behavior and disease. The Institute also promotes neurosciences education and research for both students and faculty at UTSA. In addition to the annual public lecture, the institute sponsors an annual scientific symposium on brain research, weekly career development meetings for UTSA faculty and graduate students, a weekly neurobiology seminar series and a podcast featuring conversations with prominent neuroscientists.
Translation of Gene Therapeutics in Neurological and Neuromuscular Diseases is scheduled at 5:30 p.m. on Wednesday, April 10, 2019 in the Main Building Auditorium (MB 0.104) on the UTSA Main Campus. The 5 p.m. reception will precede the lecture. Both are free and open to the public.
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