Fabry recap | SGMO Message Board Posts


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Msg  167988 of 168914  at  9/29/2022 7:26:22 PM  by

ZFP


 In response to msg 167530 by  ZFP
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Re: Fabry recap

Some incremental updates today during fireside chat at WF
- The expansion cohort design was done after meeting with the FDA in an effort to explore accel approval possibilities though still not a probable outcome.
- FDA already linked ERT to substrate reduction to eGFR reduction for Fabrazyme approval. SGMO hopes data will continue to show supra a GAL expression and kidney biopsy shows substrate reduction along with eGFR slope improvement. Then they can have that conversation.
- Comments about 45% Fabry deaths from cardiac events telling as were reference to patients showing benefit already that had been on ERT prior. LVT metrics are another possible avenue for small N P3 accelerated approval but no way to assess likelihood until data is shared.
- ESGCT data will include some early look at 5e cohort but not expansion cohort which was designed to maximize chance of AA
- First 2 ERT Nave patients in expansion cohort had baseline biopsy and will have 6 month biopsy to compare. Not providing guidance on any other baseline biopsies done.
- JB comments about World seem about right. Patients were dosed in March and April so 6 months to biopsy and 2 months to scrub data cutting close but possible



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Msg # Subject Author Recs Date Posted
167991 Re: Fabry recap rynotheknife 1 9/29/2022 8:15:32 PM




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