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Rapid development of an n-of-few gene therapy for SPG50 through duantless collaboration

June 17, 2024 at 12:30 pm US EDT

Steven Gray and Terry Pirovolakis will share the story of the development of a gene therapy treatment for Spastic Paraplegia Type 50, Terry from the patient advocacy perspective and Steven from the scientific perspective.  Leveraging the knowledge from previous gene therapy approaches developed by Dr. Gray, combined with the tenacity of Terry, a gene therapy treatment for SPG50 was developed and moved into a clinical trial in under 3 years.  Terry and Steven have both taken efforts to make as much information about this program public as possible, to aid others in the development of similar gene therapy treatments.    

Terry Pirovolakis, a co-founder of CureSPG50 alongside his wife Georgia, encountered the formidable challenge of addressing his child's SPG50 diagnosis in 2019. Leveraging robust fundraising and community backing, he spearheaded ground-breaking research, successfully treating his own child within three years through a Canadian CTA and two more children via an FDA IND.

Furthermore, Mr. Pirovolakis established Elpida Therapeutics, functioning as a social purpose corporation with a non-profit ethos, committed to addressing ultra-rare, non-commercially viable conditions through gene therapies. Collaborating with industry leaders, Elpida initially focuses on SPG50 and CMT4J, with plans to address three more ultra-rare diseases in 2024, allocating profits to sustain and expand programs. Pirovolakis extends his impact by assisting other patient foundations, conducting Gene Therapy 101 classes, and engaging in collaborations, all driven by the overarching goal of saving as many children as possible.

Dr. Steven Gray received a B.S. degree with honors from Auburn University followed by a Ph.D. in molecular biology from Vanderbilt University and postdoctoral training at the UNC Chapel Hill Gene Therapy Center.  He is currently an Associate Professor in the Department of Pediatrics at the University of Texas Southwestern Medical Center.  Dr. Gray is the director of the UTSW Viral Vector Facility and maintains affiliations with the Department of Molecular Biology, the Department of Neurology and Neurotherapeutics, the Eugene McDermott Center for Human Growth and Development, and the Hamon Center for Regenerative Science and Medicine at UT Southwestern.  Dr. Gray’s core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system.  His research focus has also been heavily focused in developing AAV-based treatments for neurological diseases, some of which have translated into clinical trials. He is inventor on over 20 awarded or pending patents and author on over 90 publications related to gene therapy. 



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