The combo trial will show how ATL1102 performs by itself, how the exon skipping drug performs by itself, and how they perform together.
It would be interesting if the ATL1102 arm outperforms the other two.
Gene therapy shows lots of promise hypothetically, but has not performed well when it hit arms. its initial dosing caused some adverse events. It is interesting that the FDA has revamped its accelerated approval pathway, to ensure that drugs that initially show promise buf do not work when used, are not kept in market. This places more pressure on those DMD gene therapy companies.
interesting times ahead
Ann: Quarterly Activities/Appendix 4C Cash Flow Report, page-17
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