|
|
|
|
||
Re: Unfiltered Notes SGMO at DB (some gaps due to technical issues) December 2015 presentation had ZERO data on SB318 causing proteins to get into the brain. The proteins getting into mice brains came out in SGMO's March 2 press release. When you watch Dr. Chester Whitley's RAC presentation for SB318, you are struck by the idea that Hurler's is really bad, and the ERT while helpful, certainly does not cure. With proteins in mice brains, Chester Whitley may be thinking that SB318 should be tried in infants as a cure. The SB318 trial is over 18 only. Dr. Wexler (SGMO works on animal studies) made some odd statements at the December 2015 RAC meeting that the MPS 1 mice used for SB318 pre-clinical can be "cured". Then the March 2 SGMO press release said something to the effect that "both MPS1 and MPS2 mice treated with SB318 performed maze tests as well as wild type" as if the MPS 1 and MPS 2 mice had been cured. Curing an MPS mouse may have profound implications as enzyme replacement therapy for MPS 1 (Biomarin's Aldurazyme) is nothing close to a cure. At 23:53 of the SB318 RAC meeting, Chester Whitley states unequivocally that "...we know the (ERT) enzyme does not cross the blood brain barrier." Chester Whitley doesn't think that about SB318 enzyme. More likely Dr. Whitley is thinking, "If the SB318 enzyme got out of the blood and into the brain, what else might get in?". Dr. Whitley has been treating svere neuro disorders for decades. I think SB318 trial runs out of his clinic. (Who knows why SB318 goes into mice brains and Aldurazyme does not. Possibly Aldurazyme is recombinant, possibly no one ever checked before, possibly continuous expression of SB318 is preferred for crossing blood brain barrier) (kabonk posted pearlier today on EXT1 as a possible target for gene expression siliencing for treatment of alzheimers) |
return to message board, top of board |
Msg # | Subject | Author | Recs | Date Posted |
81278 | Re: Unfiltered Notes SGMO at DB (some gaps due to technical issues) | tnkersdamn | 13 | 5/5/2016 7:29:32 PM |