Sarepta are in a world of pain now.
Gene Therapy was always a 7 to 10 year dream...back to the drawing board.
They still need to provide the FDA with efficacy data for Exondys, their only commercial product...otherwise that's gonski too!
Exondys brings in circa US$400m revenue and only applicable for 13% of DMD boys.
Combination trial with ATL1102 is on the cards...PUL2 efficacy data will romp it in for Sarepta.
They have a +US1.5B cash war chest...interesting times.
Sarepta gene therapy study missed key target in Duchennes, page-6
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