Sarepta Therapeutics Announces that Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial Placed on Clinical Hold Due to an Out-of-Specification Production Lot; No Observed Safety Events
07/25/18 4:34 PM EDT
-- Out-of-specification lot resulted from the presence of trace levels of DNA fragment in research-grade raw material plasmid sourced from third-party manufacturer --
-- Fragment fully characterized; preliminary testing and analysis indicates no safety signals --
-- Subject to
-- Clinical timeline to commence dosing of patients in pivotal trial by year-end 2018 remains on track --
-- Sarepta to host conference call on
“Patient safety is our top priority at Sarepta as we know it is for Nationwide Children’s
Sarepta will host a conference call today,
This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Research Institute’s action plan, including the plan to use GMP-s plasmid for all future production lots; the preliminary in-vivo tests performed by the
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Source: Sarepta Therapeutics, Inc.
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