MedCity News May 20, 2022
Frank Vinluan

A Rocket Pharmaceuticals gene therapy for the rare immune disorder leukocyte adhesion deficiency-I has met the survival goals of a pivotal study, paving the way for regulatory submissions that the company expects to file in early 2023.

The serious and life-threatening infections experienced by those born with the rare immune disorder leukocyte adhesion deficiency-I mean that some babies don’t survive beyond infancy. Of those who do, most don’t make it past their fifth birthday. A Rocket Pharmaceuticals gene therapy for the disorder has clinical data showing that not only can it address the underlying genetic problem, it also helps patients live longer.

Rocket presented the preliminary data Thursday during the annual meeting of the American Society of Gene and Cell...

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