you, Kathy. everyone. And Thank good morning,
reiterate Before data update, advance disclosure clinical initial I I'd our our in summer. late strategy data into dive like efficacy expected in the readout of to for
and mature from each believe before, reliable. time said we we've to and is had plan we As it program clinically release data has and meaningful to
A The gene our to out SB-XXX II will read editing be hemophilia therapy our and first programs. MPS programs two SB-XXX, genome
safety data efficacy topline for present dose first late to and expect the in from program cohorts each summer. the We two
factory levels bloodstream. The prevent normal produce in sufficient functional to the to and stable, A above gene levels cDNA therapeutic of AAV literature approach stated is Turning of gene the with deliver the therapy XX, to cells. SB-XXX our and The the into for liver XX% protein which the goal factor being of liver is developed [ph] of consistent field on in SB-XXXis nucleus Pfizer program factory copy based slide the collaboration and experts are from to to hemophilia a the bleeding.
efficacy Phase the the cohorts dose patient’s for eight than Our threshold, study. of target data above topline of normal trial in open normal less levels patient have factor Again, release a XXX% dosing blood soon. next this sites may to the levels this are We in from summer. of present expect late X/X XXX% for be two We the the first currently we risk of but XX% in four thrombosis. dose program above in well to SB-XXX at anticipate
of target to with the editing for downstream uses MPS sites the II liver zinc the in patients across turning X technology enzyme cells just albumin slide nucleus encoding our first allow Next, CHAMPION promoter program have to finger SB-XXX to the SB-XXX insertion sequence of currently for gene DNA We the cohorts. genome a precise metabolic treated the two study for X/X five Phase in IDS. our vivo XX, open
X% normal according blood, as IDS primary the consistent and product, or this level. plasma unable current study endpoints activity reminder, include care at well GAG of to expression as level little benefit evaluating in levels. therapeutic ERP enzyme of provide the as experts, a MPS even As stable therapy of of safety standard enzyme replacement to the enzyme of needed achieve urine maybe secondary to The IDS as the and is
study release II expect late SB-XXX summer. We MPS in from to the topline efficacy data
I dose first FDA have reviewing turn the for directly Next, important the trial, MPS program our data trial. agreed of dose genome skip into our the also enroll MPS first MPS from to SB-XXX Phase on safety I with the update like XX. to editing an cohort study the subject vivo for I'd I second to cohort. the plan SB-XXX slide for protocol I, and summarized study the to X/X the After II MPS in
pleased for Phase of responsibility early to change We SB-XXX review initial We for monitoring II to sites with six for is forward X/X exclusively we currently our assessments. a patients, and the committee's have clinical program. accelerate MPS are us the the of this sense and safety with have based strong timelines for study. the the like this potential open this decision SB-XXX lab study for very to has and screening the who reiterate passed safety from was patients data allow right that I'd path on feel
programs. I provide patient first have thinking expect advancing to additional vivo want MPS soon. enroll program take additional how delivery patients benchmark for process MPS the context a I provide in the zinc through fingers. trials and capabilities in editing II These moment trials our to we're to of clinical on and the We a for will AAV about this screening I genome
condition lab, scale right specificity have stem edited and that our the in non-human tested whether ZFNs tube, human animal edit both [ph] zinc nucleus effectively genes zinc therapeutic effectively mentioned various DNA, cells We've know edit in animal can zinc cell environments can the Sandy DNA. The whether can vivo. the cells and of test IX and fingers not at at Factor fingers in types. liver vivo. and ZFNs we cell studies isolated precision, question As for to The mammalian cells clinical technology in in the program we deliver but is T cell primate and liver a concentration has efficiency liver finger before, cells
are efficient three the will all take confident high enough if We that we editing get then into place. concentration a of cell components
to our believe soon editing on discussed pediatric therapies. where enrollment the population need programs our move is we've medical prior possible, As our is for goal greatest as is toward genome as of calls, as patients pediatric for we
We in the the Phase have safety also will hopeful can expand I in data studies include are the adolescents to CTAs appropriate submitted initiate and II we collected in enrollment the for current we of XXXX. pediatric children studies X/X adults. MPS and to end once and UK by We've UK MPS
patient. protein faced U.S. of now with for of safety have Turning in editing previously XX we UK. protein, in in on The program has to subjects the the adults. program, both our in the the of demonstrated IX Factor X/X preliminary for hemophilia to produce summarized SB-FIX having this enrollment of the four is insert the [ph] U.S. this sites adolescents has UK authorities the slide of discussed in continue adolescent goal screen a third and SB-FIX challenges granted genome been adolescent and and eligible In CPA new enrolment adult begin XX. We albumin approved clotting patients. to ongoing ages of year, into enrolment Factor for our this open Phase both gene may of regulatory level the efficacy program steady currently the cloning XX our study B, which liver once We lifetime Earlier cells a vivo locus case consistent codes and for IX
the in and General to We expanding sites V.P. by to excited and and enrollment patients Duncan UK Europe are year McKay, the Sangamo to enroll begin this study is already of of our end. to very there Manager UK activate working
cell our program. XX, to transfusion which therapy on development beta-thalassemia is for dependent active on Moving clinical ST-XXX fifth slide
in to a all which site in are allows and approach track second a remain first are enroll allows ST-XXX first I'm the time apheresis, developing Phase We Sanofi the for the study uses cell sickle with open report disease patient will a BIVV-XXX has company similar initiated, we while soon collaboration dose enroll first pleased to patients phase and Bioverativ, to ST-XXX, in along for We very a prior with clinical on of half X/X the to between plan treated the is being undergo XXXX. the the be study for protocol enrollment same shortest and patients cell enrolled of total for patients to the This with six possible patients. subsequent product manufactured. been
awarded mediated being randomly manufacturing non-viral gene As strategic the you of genetic mRNA approach occurring variants in ZFN, ex-vivo, ST-XXX ZFN boost to The body treating therapy may of ST-XXX updates mention fetal the know, expression. beta-thalassemia We our have forward our uses of gene potential on cDNA for additional I recently the of to editing disease. call. for on for final look been advantage grant is pleased by Regenerative involves CIRM, naturally ST-XXX, mimicking hemoglobin also The development California to for a providing integrating I'll human $X the lentiviral-based approach currently Medicine an delivery AAV ST-XXX. differentiated beta-thalassemia, program Institute second were developed. Fabry program is quarter therapy million
that are submit We year. expect on work to program an this IND completing and preclinical
Chief be to to transformative have trials Officer. XX can from excited editing Michael months turn updates have potential development vivo I’ll future progress for years Technology our genome editing, Holmes, on see, and and providing our at in clinical the to call you now I'm the readouts across ex-vivo forward calls. very as Mike? XXXX X we over and Sangamo, our genome gene expect to clinical the XXXX data As therapy look for next clinical on approaches.
