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7.3¢ percheron therapeutics limited

Sarepta gene therapy study missed key target in Duchennes, page-7

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Uboy

4,680 Posts.

1083

08/01/21

14:00

Post #: 50036111
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Originally posted by Uboy ↑

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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Yes that was from mid year, but recent trial results have been a bit meh, raising dystrophin results from 5% t0 20% of baseline. Still a lot of work to do for FDA to approve imo.

ANP Price at posting: 14.5¢ Sentiment: Buy Disclosure: Held
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