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Pfizer's Gene Therapy Candidate For Duchenne Muscular Dystrophy Receives Fast Track designation

Pfizer Inc. (PFE) said the company's investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the FDA. The designation was granted based on data from the phase 1b study that indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The company started the phase 1b multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 in 2018.

"We are working to advance our planned Phase 3 program as quickly as possible," said Brenda Cooperstone, Chief Development Officer, Rare Disease, Pfizer Global Product Development.

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