PER 3.57% 8.7¢ percheron therapeutics limited

Sarepta gene therapy study missed key target in Duchennes, page-6

  1. 4,686 Posts.
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    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.
 
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