Fibrocell Science’s newest gene therapy treatment is now spreading its wings after helping treat the first patient suffering from fragile, easily blistered skin associated with “butterfly children.”

The Exton research company’s FCX-007 treatment for recessive dystrophic epidermolysis bullosa (RDEB) has taken flight in mere months.

It was fast-tracked by the Food and Drug Administration in January, kicked off its clinical trial in February, and cleared its first patient without any safety hurdles, according to a Philadelphia Business Journal report by John George.

The company itself also recently underwent a metamorphosis after a setback with its azficel-T vocal chord scarring treatment.

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“During 2016, Fibrocell completely transitioned to a clinical-stage gene therapy company, and we are confident that under (CEO) John Maslowski’s leadership, its significant momentum will continue,” said Chairman Douglas Swirsky.

“We look forward to fulfilling the promise of our proprietary fibroblast platform for patients suffering from rare, devastating genetic diseases of the skin and connective tissue.”

If the ensuing clinical trial is successful, Maslowski noted that Fibrocell has “a manufacturing facility and the infrastructure to make the therapy here, and sales and marketing will not require a large sales force because there are really just 16 medical centers around the country treating RDEB patients. In Europe and the rest of the world, perhaps we would look for a partner.”

Read more about Fibrocell’s progress in the Philadelphia Business Journal here, and check out previous VISTA Today company coverage here.

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