our Michelle morning, and This first Ron ever Counsel. Robertson, executive meaningful vivo dosed incredible the members We of pediatric our BRILLIANCE team, for joined Financial CRISPR was accomplishment. dosed trial Thanks, editing, the General forward good everyone. Officer the this using today first Chief to and the and Editas time an Chief made more our EDIT-XXX. including with am gene LCAXX I quarter this milestones child in And year. by Stern, look Charlene a patient we Scientific several marks excellent Mark progress Officer, Shearman,
yesterday immunogenicity EDIT-XXX. dosing at treating pursuing a that leading And is of we conference, presented this forward critical the supporting step to LCAXX patients the the inherited childhood profile of potentially one younger safety disease. data of further and causes Given blindness, the ARVO is favorable
ARVO We the the providing conference, XXXX expect we'll year. of in second data later design initiate year. dose our EDIT-XXX IND-enabling knock expect demonstrating update We're testing half on planning mid for pediatric of our out program the high our half unique cohort EDIT-XXX Later cohort pediatric towards at in advancing with we complete the also and present clinical and of That this in end of vivo the non-human to dose to of today, the candidates year. retinitis for studies primate replace retinal which start by the function. is to preserved pigmentosa, first
trial program disease cell for beta screen to thalassemia patients. Our sickle and continues EDIT-XXX and enroll
sickle track the begin we've of for month. cell before patients thalassemia And beta successfully cells next on dosing are For to and ex added this vivo several later end year.
Patent clinical review Our O’Neill. We data. our in leader is opt standing the cellular us EDIT-XXX data excited And issued she'll T favorable Gilmore patent Trademark therapy for We'll program. a interference cell we're that ASGCT. to on few today long showed intellectual impressive of iNK our US to to comment preclinical into Office later to partner And implications in joining we're Charlene property few a this But And CRISPR/CasX affirming programs alpha-beta and first, are announce and our finally, I'd decision briefly The that at a new in advancing. I'll organization, new our foundational position. programs. discuss like detail moments. minutes. seventh Broad BMS pleased The month Institute more have a on decision have recently
our dosed first for EDIT-XXX study BRILLIANCE for we LCAXX. pediatric mentioned, As in patients
the we've a significant many will field. second the we editing been diseases. The viral has continue the that disease scientific the and of to patient for sense later initiate gene first This data apply EDIT-XXX. and of the favorable considering most can months data been young is tolerated cohort data exploring we the development, clinical cohort. we will entire to sensitive year. the as for level ARVO other data and registrational assessment information, To the the viral trial safe we indicating living CRISPR we least the And the the viral with to evaluate the including of have monitoring to profile. relevant using AAVX. while will those dose comprehensive medical how enormous levels said support at This also to past, safety shedding, their months vivo complete individuals cohort, endpoints and no administered therapies, as our from EDIT-XXX EDIT-XXX dose data, as a including gives all ocular at committee The on administration. milestone no the patients. shedding six as continuing gene on we're families, least in and at transient, safety was interest provided medicine begin on adult presented least genetic independent lay viral supported well evaluations, and for dose year. had editing first The trial anticipate child. cohort effectively of up trial pediatric the adult what which shedding high yesterday to as in assessments There's that to time the patients on meaningful this and program's follow with of of months high who of capabilities mid dose The the community pediatric dose between expected foundation received interpret data Then at the XX This we persistence safety patients. conference half is other four correlation systemic EDIT-XXX registrational patients following six data has trial treated mid clinical ongoing expect overwhelming, year. in date, before we of gene enthusiasm and update following it will well the most which the of of A update second based efficacy plan expect We BRILLIANCE be and dosing more demonstrated us the on in has most is support always to half been the these the include
the we're at later year. present additional at In in to that measures. the study findings the natural trial conference We'll our looking addition identify some reproducible with to data data, study the data on TIDES history most upcoming also
trial is results enrolling study cell The to track XXXX, we're in disease RUBY patients initial for sickle Phase EDIT-XXX year and begin to first dosing Moving by our end. top the vivo ex expected clinical of platform. with line half X/X on in
a for This their where cells apheresis EDIT-XXX X/X it sickle the them. patients their pediatric or fetal as actual on dose an have level had time program We track a can disease first have of their in treatment each recently disease this so of edited excited very first remain already TDT EDIT-XXX extracted CDXX this that designation for transfusion stay believe the to TDT. to also patient we one hospital regimen to multiple their hemoglobin. thalassemia, and administered We we well. We're be completed Phase extracted, has to and can cell cell initiate process cells beta patients we collect the their to beta scheduling of enough extended rare preparing EDIT-XXX. edited both cycles requires approach the dosing have we're year. beta cell necessary successfully - require to the for for back reinfused edited, patient These sickle received treatment cells We're And of trial preconditioning which TDT. disease as undergo they're dependent a healthy dosing restore cells and thalassemia, EDIT in
result the to genetics, persistence approach natural of Patients field editing indications. a one recreates are and our also approach gene we of these of the durable unique the hemoglobin. be specific in EDIT-XXX that treating same AsCasXXa human in believe in Because protective grounded safe to edits. the both the hereditary complex most approach will Editas-engineered validation precise We're enzymes underlying fetal essentially of these make enzyme, mutations utilizing mutations protective asymptomatic. these with Our
to a XX to as as physician scientist. and including He I CEO management clinical ago. development, change a products, of patients weeks their upcoming the June and incredible few is announced discovery over Executive disease now indications, Finally, role to to assume many addressing launch overseeing numerous genetic early way from and rare they successful development years made take of Editas, in accessible the Starting I worldwide. a medicine, the as both we will will of breadth mention X, brings want experience Gilmore an Gilmore O’Neill Chairman.
excellent was the executive As long value. received of Chairman, right Gilmore experience design, in person now? preclinical several and question one depth clinical Gilmore to clinically two relevant with to The and His right time. to expertise team to clinically now. our year good technology an pursue. today, closely select was Executive stage significant This creating the will this that the to development, the with his we position needs leverage other is a experience working making for come. the Editas address progress is early with exactly the and enhance be is several to and our targets can the medicine drug team announcement frequently preclinical why the indications. of add prepare and Gilmore, what made, for but drive in trial clinical the is in asked answer creative genetic moment or to job passion time His for bring including where I'll programs in simple clinical right term to programs, years is next medical and next We're and later stage exactly we're rest two, capabilities When continuing programs and address not vision we
like to CasX. I'd the to over Charlene update to surrounding turn IP patent foundational on decision Now, recent everyone the the regarding call
And some Editas continues the one instrumental join have in in to people to her first this developing Charlene launch be of was the For thrilled IP and was to to company strategy. company's the Charlene. join background, I'm XXXX. morning. she us