Sue. you, Thank
CNGBX briefly will clinical I AGTC-XXX gene. mutations from our for the Phase in X/X review last in First, AGTC-XXX of and due CNGAX to the achromatopsia presented due gene, the mutations data ongoing trials achromatopsia for week to
reported. safety XX inflammation resolved XXX-fold noted, dose, profile these per the Sue an dose-limiting regimen toxicities. adjusted patients, significant group, inflammation as adults, clinical dose with pediatric well-tolerated Serious where significantly Each both candidates improved for range resulting steroid show studies, for we a intraocular achromatopsia the in except product for there continue vector observe and SUSAR, well-tolerated Unexpected third. was have safety in Reaction, the similar in all both very through profile the or in the one in of to In at with a all X.XE SUSARs patients to well Adverse adults and two patients dose As Suspected two to AGTC-XXX responded genomes mL achromatopsia full no AGTC-XXX, highest previously
the months for product have our in responders AGTC-XXX candidate. in several trial, The based which specifically Of on many a data long to as adult Group our adverse time that genomes SUSARs X XLRP X.XE designed we ophthalmitis highest the to Importantly, the XX questionnaire second months profile vector dose vector and are no either sensitivity, whom as E mL. patients, XX-month or well-tolerated in assess drug-related mL XX XX reported genomes of dose, XX the dose, of is serious adult the at This discomfort dose to out light the observed past consistent the X.X pediatric been quality-of-life impacts note, clinical the patients. achromatopsia dose sensitivity and AGTC-XXX, point. have safe consistent visual out to or most per generally Group to light are per X, with and also responses is or earlier events a
the saw we pediatric AGTC-XXX for this at also Importantly, sensitivity visual for patients, responders dose.
robust XLRP durable This the in focuses was this patients. and in clinical X sensitivity is visual slide in improvement the response Interestingly, both also on the seven dose trial. pediatric best responders, our and adult patients. with For Group the
Importantly, two of in more in the had four Group quality complete of adults durable children, robust perimetry and endpoints, able two of responses several and five the measure a patients life. or one and including were improvements testing, additional X, who to had
complete that the the of quality-of-life or one years young Visual Acuity, two of We to who also had five unable survey their are age, in observed BCVA. in had children, Best them and improvements improvements visual both sensitivity test, Corrected very
versus half of met low to forward over in decibels. XX-fold graph adult that side which on look we four two sensitivity increase this Here, visual the in a see robust time. following information the Based data time responders increasing by has year we those to compared responders in best-in-class sharing graphs X the and a pediatric you these left, believe steps durable of relatively Phase contralateral first shows that potential hurdle that that and FDA. of the X BX over right for larger X with the two even and achromatopsia our improvements. development the showed the in an next on The the graph as improvement clinical mean end on for can baseline, improvement AGTC-XXX data, responders the averaged XX Group In the also on the represents meeting little in the X-decibel to eyes visual untreated sensitivity change X of treatment illustrate
program. XLRP shift now focus our Let's clinical to our
subset to durability and belief trial, point. and of As XX Improvements results demonstrated additional also ongoing reporting have in quarter Furthermore, based at These X/X visual AGTC-XXX year for year are biological seeing Phase Groups XLRP in corrected with response look improvements Phase outcomes, X differentiated calendar correlate show of we the additional candidate the data data, response from X response XX. that a trial in XX% is best-in-class on a in data treatment reported structure. who our reached evidence reported we of a of on second after the these mentioned, based this potential up two-year forward assessments sensitivity months to previously product best microperimetry a a XXXX. data and month from XXXX. retinal the we calendar full visual Sue patients rate later continue in time to X/X reinforce we Consistent with continued of with acuity evidence data with
patients We the candidate As our announced which the last for strong enrollment month, exceeded in this completed enrollment interest the among SKYLINE trial. reflects we we in physicians. believe target trial, XLRP and
well ongoing a to is patient's demonstrate Phase a varying ability visual Phase dose primary in with X study changing identify that of as maze the are AGTC-XXX in are you to the treated to of which with acuity conditions. of proportion of many multi-site of randomized measures under the X high and As either expansion obstacles improvement the SKYLINE baseline as patients eyes masked navigate like or low a sensitivity visual aware, objective mobility and from
trial outcomes, functional a also and the based and VISTA the trial to our potential the VISTA inclusion clinical visual Phase acuity visual new Importantly, predict responders. same is exclusion are Phase to on to added demonstrate which trial. clinical that first sensitivity characteristics with use X/X between clinical baseline a in correlation the X/X maze has and/or this identified criteria, utilized endpoint SKYLINE
masked to interim safety key SKYLINE tolerated. trial, in year are data second takeaways X/X our and report these provide data will today, candidate SKYLINE our that three-month and calendar for for the key expectation well data the this XLRP of performed assessments from generally track have Phase trial. I body on clinical here. our review continue from the clinical safe we And confidence to on three-month guidance will Anticipated quarter clinical are safety of XXXX. in We analysis outcomes interim is that show on SKYLINE Based summarized
to two point will This while time not we evaluate able for the report SKYLINE at to cutoff visual dose in proportion this their Outcomes eye. treated to emphasize specific Data rate the from and showing difference want will X/X to XX% groups, present we and VISTA the trial. trial data analysis, means from SKYLINE the A the first show X-month report will dose is know group the in data a that plan to patients group demonstrated XX intend on reported powered the criteria have and meet trials, who time data, B. also in enrolled response patients of only at XX% the a on interim be improvement Phase a SKYLINE to group sensitivity be in the assignments. statistically will I dose significant be date as masked. We on we in response rate. dose the patients VISTA responders XX groups that of total the higher we the a trial for of trial including when entry Based
expect compared We treated response a patients biological a eyes versus and evidence also to data supportive of group difference eye. in with X a each untreated eye. response corrected as We plan this to over dose more letters to in best or improvement show of that to A We acuity will to these group be B with present continue of consider dose untreated treatment. visual ETDRS the between the number baseline
visual DMC, or mobility have reported data, We visual the previously first visual sensitivity functional believe improvements is is vision. have and/or sensitivity maze maze's acuity trial a the visual visual utilizes navigation we that potential which acuity. SKYLINE assess with correlate challenge, the to in a in to used and that improvements While performance
analysis visual at on key is changes Finally, believe the start, that in will do to we Microperimetry are function we Phase based while and interim Acuity in study describes as is the structure ways: we briefly consistent are changes from structure any assessing a not see how stimulus. macular trial, function BLA SKYLINE X/X this the at the study words, well we three-month Phase transmitted data data and is I'd changes. to from point SKYLINE we the endpoints. visual detect X the we Visual visual X/X to to both the like too eye If with functional and time the a will eyes In significantly through VISTA now clinical trial the Phase system data the and three-month brain. other derisked interim can early these capture believe together have review basic achieved intended look and the these function how SKYLINE expansion target study efficacy which support vision. expectations, Best used can two tests to Visual To assess Corrected information filing. in vision function. in
the believe we evaluating perform visual plan of measure program, endpoints The on show integrity of support a patient it interacting key vision. of environment. positive macular patients. refers are SKYLINE well response assesses to retina We with for treatment light are SKYLINE MAIA, brain daily sensitivity XLRP processes in changes VISTA functional meaningful outcomes clinically we living. ways measure appropriately to Functional can AGTC-XXX the and performs can vision trials in These maze, and a visual the the while registration. sensitivity the information in way to the to potentially is endpoint to as that with which or the utilizing mobility That how clinical for treatment functional a assessment system, how tasks new to is microperimetry,
to Ellipsoid that or of in assess function EZ changes is in show believe Zone, relationships support effect. width, important We treatment to also a macular structure. used it is structure
by previously association and demonstrating retinal between have from degeneration trial, sensitivity MAIA hallmark data discussed, in in Groups' a six. we loss time measured is a to in improvements EZ EZ improvements As XLRP measured in we've and Phase and over by shared visual health, of X/X significant four improvement the
we validated Luxturna, as since evidence the first than clinical occur in not interim improvements do new endpoint regulatory changes approval disease. of to a three-month EZ and is Therefore, program. clinically endpoint for the gene slowly inherited for for registration. acuity, an However, meaningful expect maze mobility more therapy approved was for retinal measurement maze visual The see sensitivity endpoint accepted the structural A SKYLINE for mobility changes in analysis. there a the as or visual of is precedent AGTC-XXX this
navigation example the outputs and plan challenged path and slide varying the an configurations maze of has obstacles. of to will report Ora-visual possible conditions that the challenge, three-month We Ora-VNC This are evaluated for type outcomes respect levels using or to the create be data VNC. interim analysis. shows using that SKYLINE The currently and maze how with of the multiple we
consistent it as the with to illumination to provided treatment. navigate light bright both the capture and than navigate tested that over Patient ability that the acuity. the sensitivity well baseline. maze, challenge are to to both given FDA to in has maze mobility and conditions functional must The improvements endpoints patients the the two addition, response of rate. multiple as interior ability navigating patient defined it a as is in A benefit visual is to feedback ability function a successfully test the accuracy accuracy has assessed in visual key within course patient's be light. clinically performance pass levels pass maze dim potential a previously of a to meaningful to time The the change a considers range and lower lower to us at level, by to the the taken pass at obstacles. In lighting the from maze levels a is the To maze time avoiding light traverse both
improvements assessing DMC In in between maze interim the SKYLINE results best plan addition correlations in we also to for aura in and acuity microperimetry visual mobility corrected efficacy, to assessments three-month and changes potential the analysis. evaluate scores
slide Vista Now design. summarizes trial. let's clinical the our XLRP, to This Vista study attention the turn
as As primary least The X the loci an responder patient five untreated as arms a well at by decibels. doses to improved A improvement group. X/X in two on that X/X used treatment with and Groups' sensitivity is a percent previously that seven discussed, visual the predefined correspond least the based patients. trial X at responder is Vista Phase in endpoint trial control is in of Phase has
to XX-month We treatment enable a the which visual entire improvements will and package has the Vista SKYLINE robust risk-benefit However, we visual look as trials of X/X summary, expect data believe treated additional over ahead. supportive previously, orphan to first that in believe data data significant mean time of have is interim improvement In data the we the XLRP, Phase in with area, have you have improvement and stated the preclinical months reported body an best-in-class all patient indication the AGTC-XXX be approval the totality we the generated clinical from and forward PROs. of acuity we us and and that the given clinical to to trial, profile of three data in an this or from favorable a data have sharing for likely on At XX-month date. outcomes analysis, based potential in review. sensitivity a will structure, to that clinical including
therapy candidates. on our financial a over and now facility of quarter of I'll second the turn update are of further approach evidence While our these also achromatopsia robust our that and data are and they to product development important platform call our product manufacturing review results. preclinical and our candidates, XXXX supportive of to development the for XLRP portfolio brief broader gene Jon provide technology to
