Gene Therapy for ADA-SCID
1 هزار بار بازدید -
5 سال پیش
-
Bobby Gasper, MD Chief Scientific
Bobby Gasper, MD Chief Scientific Officer of Orchard Therapeutics talks about his company’s gene therapy in development to treat children with adenosine deaminase severe combined immunodeficiency (ADA-SCID).
“These children are born without functional lymphocytes and they are very vulnerable to infection,” noted Dr. Gasper, adding, “so the kind of coughs and colds that we get. Those are fatal to these children and without any treatment the majority of these children will die.”
“The current standard of care is a bone-marrow transplant but that comes with complication using somebody else's cells and so we developed gene therapy for ADA-SCID,” stated Dr. Gasper.
“We've completed a 20 patient registrational study and that has shown really very effective results. We've been able to show a 100% survival in that cohort and also 100% event-free survival.”
Dr. Gasper is hopeful that the company can file with the US Food and Drug Administration (FDA) in 2020 to gain approval for the gene therapy.
ADA-SCID (aka bubble boy disease) is a rare, life-threatening, inherited disease of the immune system due to mutations in the ADA gene. Symptoms usually begin during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive. Without treatment, the rare condition can be fatal within the first two years of life.
“These children are born without functional lymphocytes and they are very vulnerable to infection,” noted Dr. Gasper, adding, “so the kind of coughs and colds that we get. Those are fatal to these children and without any treatment the majority of these children will die.”
“The current standard of care is a bone-marrow transplant but that comes with complication using somebody else's cells and so we developed gene therapy for ADA-SCID,” stated Dr. Gasper.
“We've completed a 20 patient registrational study and that has shown really very effective results. We've been able to show a 100% survival in that cohort and also 100% event-free survival.”
Dr. Gasper is hopeful that the company can file with the US Food and Drug Administration (FDA) in 2020 to gain approval for the gene therapy.
ADA-SCID (aka bubble boy disease) is a rare, life-threatening, inherited disease of the immune system due to mutations in the ADA gene. Symptoms usually begin during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive. Without treatment, the rare condition can be fatal within the first two years of life.
5 سال پیش
در تاریخ 1398/08/07 منتشر شده
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