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Sangamo Therapeutics, Inc.

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Msg  173427 of 175487  at  6/8/2023 8:14:21 PM  by

MartyCh


 In response to msg 173235 by  MartyCh
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Re: 2023 Events

Jun 8 Jefferies HC
• Delivery
o Recap of single gene repressor specificity
o Overt statement that Biogen gave programs back because they didn’t have an active capsid for the targets. Programs will sit until they eventually find capsid thru SIFTER evolution group.
 
• NAV 1.7/Prion
o Intention is to keep both Prion and NAV 1.7 in house
o Standard tox study is all that remains to file IND for NAV 1.7
o 40k TAM intrathecal delivery for first target neuropathy
o Prion- believe they now have a capsid. 500/yr diagnosed with acquired
 
Prion
• Fabry patients don’t use ERT as recommended because of inconvenience
o 20% take ERT more than 20x per year. (recommendation is every 2 weeks)
o 50% 5-20x per year
o 10% less than 5x per year
o Met with board yesterday to discuss plan for meeting with agency on P3 design
o Don’t feel showing more data is necessary until next year when Phase 3 is on its way
 
• CAR Tregs
o Only company in clinic with editing capability or GMP mfg
o Tx200 Low dose biomarker data by end of year
o Biopsy with transplant and 2nd some time later to see health of kidney will help inform
o 1st cohort through SMC
 
• Investor meetings: To de-risk company needs 2-3 things
o Raise money - SGMO has plan that doesn't need to involve equity
o Show clear path forward for Fabry P3- Summer meeting with agency. 30 days after meeting will have FDA summary b4 disclosing to market
o Find a way to invest more in Tregs portion of company


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Replies
Msg # Subject Author Recs Date Posted
173429 Re: 2023 Events rynotheknife 0 6/9/2023 6:43:49 AM
174607 Re: 2023 Events nocashonhand 0 8/23/2023 3:38:01 PM
174867 Re: 2023 Events MartyCh 31 9/6/2023 9:17:27 PM




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