With our Officer; Stephen Good joining our Strategy. Chief are you thank and Potter, President Lieber, me Matt of Officer; today. Clinical Feinsod, Jon Financial afternoon call Vice on Chief all our Business today’s Executive and us for Global
overview will review Jon questions. call, I will review our an recent will development today’s accomplishments, us will our and During Stephen position and business activities, provide of then of financial recent all your take
We who reflecting develop Group criteria accomplishments in the durability. and several XLRP response May, had and providing therapies rate Phase rare with Groups Vista X and from groundbreaking gene vision May, from X/X XX-month the patients call since of which evidence of preliminary year our enhance X patients meet data trials clinical our for have data including from people have X and data XX% many In reported a the XX-month Skyline notable would positive all of ongoing diseases. of the X quarterly to X, trial, our we expertise last using during among response for inclusion therapy
data demonstrate to maybe in continuing patients achieved safety of Taken evidence XLRP visual acuity visual candidate suggesting favorable add while body these to the profile. in receiving together, and that durable sensitivity improvements a product AGTC’s
meeting AAO presentation Yang look week believe therapy the November investigators. Paul of our Sisk, and retinitis advance the gene two for Dr. time, Robert to in As for the of data Dr. principal at we at by important said we and field at these the by RDXXXX that data an meeting we next are the pigmentosa of forward
Skyline and to August, but cancellation X-month challenges We pandemic, to July are amending our guidance of patient rate XXXX. the we than interim X-month XX% screening Vista interim the our related maintaining Skyline trials. fourth release greater quarter with several release first in on proceeding Vista data the to a for half for COVID our and Due are including data of XXXX,
support vision sites have to adding improvements including pre-screen, initiating as to Vista. screen several center logistical second follow-up visits patients mobile recruitment, and a for patient the well in significant a We of number conduct made for expansion as
low-dose patients. our including clinical our programs, from to achromatopsia achromatopsia, June, products X/X ongoing all reported Turning developing data treat XX-month trials, to pediatric in Group Phase X patients and from we adult data
achromatopsia our photosensitivity in improvements The remains perimetry reports. measured and results activity, biological as and for static generally by analyzer, and measured patients candidates, safe patient area supported low-dose OPA pediatric as or the by on ocular demonstrated discomfort anecdotal tolerated. are For in profile BX all based in the improvements sensitivity adult well light safety by visual treated patients well
of course, with position incredibly Of a our support which further gene leader data, are therapy. these field in the we pleased ophthalmic as
efficacy technology safety XLRP platform candidate. to-date for these build product and important clinical of AAV favorable and the broad confident data that are our validation of potential profiles We the on our trials and provide extend in the observed
packet an preparing end clinical trial the briefing next of the the and of to Phase to advancing testing, of BX stage clinical pivotal to X by are achromatopsia trial ready FDA. We production submit the development for planning material assays drafting
Phase end the on We of X half expect feedback first the submission in XXXX. of
postoperative a serious believe detect SAEs profile the trial now appears the can highest interim at in highest An trial, is and safety the BX two achromatopsia at Group Sight, on forward our third also same lead Sight March Of we results plans additional two Based patients CEO of suspected, closely. of overview pediatric have this well-tolerated unexpected the systemic Therapy Bionic continuing of dose timeframe. is the whom Xa in significant and increased, to X the has after responsibility achromatopsia in today. BX see the BX are developed SAE, found conducting Bionic move direction of for of an the Ophthalmic to to us Phase that data of Bionic and have at in the being or promising cases, both Bionic event, steroid But at product first new X well-tolerated considered X the the X and motion. positive Conference complete to enrolled inflammation pediatric X events, AX the optogenetics local and Unfortunately, all reported of us SUSAR. the or patients. in appropriate reported continue dose additional reported. Group our X safe safe which candidates in presented enroll of X Sheila pediatric Sight Gene have the motion product previously tolerated developed pediatric Disorders address These similar complete new inflammation treated change groups the including partnered development an all Group trial adverse across gives during the at patients light approximately X patients results levels. are been doses doses. do and be are Nirenberg, patients and body patients same meet higher of patients, pediatric monitored earlier near adults In patients. and These achromatopsia that the very in definition SUSARs. generally of its and X/X considered events at events data while patient confidence report, maximum XXXX, plan to not AX Sight our can cohorts significant to in did for this approximately blindness, and safety patients, not Xa. as this recently dose had patients patients dosing. the Xa dose have To trial. X the We above These Xa month low-dose These trials dose and
turn to briefly like pipeline. preclinical I’d our Now, to
pipeline and Our on which dry and CNS central or needs dry focuses that disorders and or The lateral meet patients are of These ALS. phenotypes. degeneration indications. candidate targets product programs of case, design address populations substantial amyotrophic skills for we scientific indications programs the nervous genetic program, and targets AMD macular a these have system CNS for age-related have now dementia, sclerosis, these unique for genetic In patient to clinical FTD our disorders. targeting and one with progranulin FTD ophthalmic two CXorfXX deep additional represent used well-defined The specific diseases ALS. preclinical optimized or or frontotemporal to form each
primates and able non-human we programs COVID And to to with to with toxicology anticipated Our global enough initiate have FTD IND. dry IND primates the in XXXX. genetic the are first forward secure AGTC current pandemic, submission despite shortage collaboration been of moving XXXX. studies of of AMD in by and hearing half the also caused bio-distribution loss has a an Otonomy form for non-human
our substantial technology a broader that enabling AAV on into to build unmet of programs have manufacturing while pipeline array indications expertise, expand and need. Our industry-leading clinical us
we that to our to year, the desired an will and to a methods, our which potential sized this generated pipeline improvements also only current meeting for AAV including have of more ophthalmology BLA of our presented developed filing commercial we our on These in our rapid interest more process below purity programs. is and supply scalable to For the by test we the demonstrate highly of our that enable facility future a containing Based and be believe candidate XX,XXX run is to material at process manufacturing build-to-suit pipeline of our methods. improvements control the estimated manufacturing entire potential than produce can new quality fulfill Earlier that advancement gene other foot for is doses. we produces detectability Florida used but a This example, commitment not facility XLRP launch a requirements to percent allow potential ASGCT data annual facility process described approval. with upon data strategy our part and manufacturing XLRP nearly To candidate support of facility a that same XX-liter of These productive XX% our square at our end, cGMP product modest and X,XXX generally productive reinforced of also the manufacturing further residuals are have for adjacent data development, commercialization. reproducible, process, manufacturing CDMOs, commercialization. greater robust, leasing manufacture and that will vectors by offices. the through material FDA
control. redundancy strengthening chain leadership we recently development three Affairs XLRP expect also additions. our both support our in this In of important Vice We to Rae our for risk Ray’s for Global appointed and Quality. In while May, and strategy achromatopsia programs. supply anticipated late-stage manufacturing Janet Regulatory our as important reducing corporate and step we of is Senior delays development of appointment support of pipeline, continue strategy have President to an to product quality of providing announced Ms. the facility prepare team, vendor our and
Hashad, and of at she disease we MD. be facility. R&D ophthalmology our Board instrumental also believe in veteran, Last cGMP also will augmented industry new an retinal month, Yehia addition compliance the We specialist regulatory assuring our through and
and than years, operations address with Hashad you CFO needs, we brings his to development and our as Aesthetics. a Allergan will guidance President patients. gene hear for with high new executive Jon ophthalmology, of with healthcare Dr. our XX at perspective Development to work executive experience retinal and we therapies from medical unmet sector, Senior as research of investment management accomplished and Research to as experience innovations addition he Global strengthened rare combined great Vice and Financial have With And as from strategies XX Head more years For banker, the Chief post-launch as team both groundbreaking expertise Lieber over physician, a is him that the seasoned team recently invaluable global management led recently, to experience most pharmaceutical an successfully has him in valuable of and in and addition most His diseases, our bring Officer. making financial a shortly. specifically
gross in public resulting our financial about proceeds in Finally, $XX.X of XXXX, to we company February million. a the with position offering, strengthened
are will turn a near-term the comments for to to that provide runway over developments. business We Stephen sufficient accomplish on call goals. our confident will this now I
