Thread by @DrTiffanyL

@AkshaySharmaMD now up! Check out his QR code his slides below. Thanks for sharing. Will try to do a quick summary in this thread 1/n #ASH22

@AkshaySharmaMD This product has guideRNA for cas9 cutting at these two sites -- indels at the HGB1 +/- HGB2. Found 80-85% indel deletion in preclinical validations. Eligibility for the study here. #ASH22 2/n

@AkshaySharmaMD These patients had exchange pRBC transfusion x 2 months at least, then mobilized, busulfan based conditioning, and then at least 2 years (and at least 13 more in long term study). So far 3 pts have been infused, today presenting 2 of those with 6 mo of f/u. #ASH22 3/n

@AkshaySharmaMD Check out these doses -- and need for multiple manufacturing batches. NO VOC, stroke, priapism, ACS in any of these patients!! but still some mild hemolysis noted. Participant 2 had a hip replacement w/o periop SS treatment! #ASH22 4/n

@AkshaySharmaMD Data on %F here in these two participants. See the line -- those show how many RBCs had some %F, almost 90% of the RBCs! Conclusions here. #ASH22 5/5

@AkshaySharmaMD Questions: Clonal analysis? Not yet, we actually don't know what transcription factor binds to that site. Dr. Mark Walters asks do you think these pts wil have enough protection cerebrovascularly? They are assessing this ongoing. #ASH22

@AkshaySharmaMD More ?s: Manufacturing could only accommodate 20x10^6 which is why they had 3 manufacturing cycles in participant 2 (who had 40x10^6 collected in his second pheresis). #ASH22