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BioWorld - Friday, May 8, 2026
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Home » Not gene therapy Leary, preclinical efforts in LSDs tripping over each other
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Not gene therapy Leary, preclinical efforts in LSDs tripping over each other

Oct. 16, 2018
By Randy Osborne
Sarepta Therapeutics Inc.'s potential licensing deal with Paris-based Lysogene SA drew more attention to the already hot space of gene therapy for lysosomal storage disorders (LSDs), the news coming just a week after Amicus Therapeutics Inc. – eager to guard its franchises in Pompe and Fabry disease – signed a pact with Perelman School of Medicine at the University of Pennsylvania (Penn) to pursue gene therapy treatments for those two conditions as well as CDKL5 deficiency and an additional rare metabolic disorder that was not disclosed.
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