'Bubble Boy' Biotech Soars 250 Percent After Cure Announcement - (Bloomberg via NewsPoints Desk)

  • Shares in Fortress Biotech's Mustang Bio unit surged more than 250 percent in response to news that eight infants were cured of X-linked severe combined immunodeficiency following treatment with the drugmaker's gene therapy, Bloomberg reported Thursday.

  • "I certainly believed this would work and work well, but I am surprised at this level of effectiveness with such a benign toxicity and side-effect profile," remarked Fortress CEO Lindsay Rosenwald.

  • In the study, the results of which were also published in the NEJM, eight infants with the disorder received a one-time gene therapy, in which stem cells are engineered to express a gene that the children did not receive at birth.

  • Mustang chief executive Manny Litchman said the treatment could be submitted for FDA approval by the end of 2021.

  • In total, 10 children have received the gene therapy to date.

  • "The data are extraordinary for every single patient," Litchman commented, continuing "it's that compelling nature of the data, in particular for those who don't have a matched sibling donor, that we believe will convince the FDA to [grant approval]."

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