Children's Hospital, Genethon get FDA OK for gene therapy trial

Fri, 07/22/2011 - 8:41am
Mass High Tech: The Journal of New England Technology

Children’s Hospital Boston has won approval from the U.S. Food and Drug Administration for a gene therapy trial in conjunction with French biotech Genethon to treat the rare immunodeficiency known as Wiskott-Aldrich syndrome (WAS).

Nonprofit laboratory Genethon and Children’s Hospital Boston announced their partnership on a gene therapy to treat WAS earlier this year. The condition is described in a release as “a severe immunodeficiency disease leading to death before adulthood” from “significant bleeding due to low platelet count and increased incidence of serious infections.” Funding for the Boston trial came from the National Heart, Lung and Blood Institute through its Gene Therapy Resource Program.



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