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EB HE nib. "Gibbons, Gary -- E]" nih.gov>, "Panepinto, Julie (NIH/NHLBJ [E]" ih.gov>, "Dieffenbach, Carl (NIH/NIAID) [E]" \niaid.nih.gov>, "Fauci, Anthony (NIH/NIAID) [E]" HEB 2 viaid. nin. gov> Ce: "Tabak, Lawrence (NIH/OD) [E]" HE oni. gov>, "Schwetz, Tara (NIH/OD) [E]" ME i cov Subject: Re: Seeing Bill Gates tomorrow Date: Tue, 27 Sep 2022 07:57:33 -0400 Importance: Normal Hi Francis, Yes, the Cure SCD team is tracking this landscape. We are intrigued by progress being made toward the vision of "single shot" in vivo gene editing with homing to HSCs. Indeed Lieber was invited to present to our Cure SCD Executive Meeting last week to give us an update of his work. In addition to what is in the JCI paper you found; they have also used their approach to editing with a SCD mouse model. They are in the process of spinning up a company. Given the Jim Wilson experience with Ad5S many years ago; they've made lots of modifications of the vector to manage the immune response and cytokine storm to mitigate acute toxicity -- a likely issue they will undoubtedly need to still navigate with the FDA. We are also still interested in non-viral strategies ; but Lieber's work is clearly at the CSC vanguard. Overall, the SCD component is very much on track toward the vision we outlined 4 years ago. Best Gary