us. today's Patrick. fourth will the Thank full update you joining On and and financial we our progress ended year conference and quarter recent provide product for a call, recap on Good for thanks of results an afternoon everyone our candidates XXXX. and
anticipated REGENXBIO, is review call improve curative NAV our milestones through mission technology proprietary platform. upcoming the of potential open also lives will questions. our for and We then the At therapy to REGENXBIO gene to on based
in potentially those of to aim the transform diseases progress and facing can patients serious course of life-threatening believe provide we significantly therapy mind. research effects, treatments We single administration, are Our proteins are to gene to defects disease. intended Through cells that our AAV genetic to product address body AAV gene are to genes a therapy candidates the in cells to enable produce designed deliver or impact therapeutic our designed candidates disease. long-lasting lives these therapy product with altering and gene the to
Our or XXX over consists of vectors gene proprietary the viral AAVX, novel AAVX, AAVX of curative and therapy, technology potential AAVrhXX. platform, vectors, adeno-associated including leverages NAV AAV which
aimed and AAV efforts at research and of effective therapy results discovering the developing are gene more vectors. safer generation vehicles than vectors Our earlier
broad and reduced generation manufacturability. immune AAV and as novel attributes Higher vectors. technology platform durability, increased improved response NAV compared selectivity, Our differentiating and earlier expression has tissue significant to
deliver that to are intended enable vectors administration that to or therapies Our to proteins genetic that therapeutic these enable to address is to disease. body in cells goal produce cells development single the genes impact utilize to the defects of
potentially product disease unmet delivering to course current and significant candidates of the Our are designed outcomes. changing and provide patients improved for patient effects the needs medical durable with
line by currently have candidates advancing expanding strategically diseases many are to significant areas These we there unmet metabolic are focus because And can neurodegenerative medical needs. selected We and vectors. within be they and an retinal, of of uniquely internal product diseases. focus area, each address been with believe addressed our
therapy development internal commitment platform, of our and aligned own. our gene single our technology to to programs, license is whose single NAV components for Beyond we vectors vision most typically with companies selectively indications third-party
would Before to progress, that commitment to company's patients our we fundamentally moment patient-focused diseases. Since our mission families. and remains to we life-threatening programs motivates therapeutic this potentially what speak treat recent to inception, review and as develop a our guide their and is I our serious with our like take rare, to own us patients
and their harder help we is at what we stories work families patient, the at and And center of compassionate the REGENXBIO. inspired do lives. by motivate are with groups to Our us advocacy save remarkable partnership that
a more. NAV and you curative disease have to hosted of unmet the their appreciation disease left Friday, at believe Jr., in it us feeling diseases of Month, of including but technology staff needs was diseases. the whatever of and is company and community exchange gene keep we to held you children in remarks diseases motivated. tomorrow community patient the for This we Luther moving groups, rare awareness parents Rare and address to to rare opportunity excellent agree with Our XX, the Dr. rare event all do February, where recognition deeply King day, Two our and REGENXBIO patient work an diseases. Disease couldn't finding therapy raise to rare parents rare concluded we children gratitude to continue We promise Martin And express insightful advocacy them of with treatments. and for committed working The are with and is February from moving forward. an quote past round of rare the our with year devastation
in expression. surgeons treatment wet listen are my like extend to And participated our of clinical and found and Pollack, speaking Heier if insights week, today, spoke who And your to to Dr. the analyst corporate our for to family already. knowledge you anti-VEGF baseline emphasized a single that the in We to I Dugel, employees potential you landscape to clinical the retinal the networks www.regenxbio.com thank and I our in a of AMD public and trial that to and investigators. this to trust establish participate macular vision engagement, website, see who opinion would wet be a diseases. from didn't our loss of be investor health earnest. a concern adults the event you and and serious their These and we the I to role degeneration chooses remiss And possible. in our Ho appreciation the want will want retinal network I of encourage recognize event and support is if moment forward Dr. us. trials also of work I you Dr. replay would And leaders from new for moving partners can acknowledge of Dr. and haven't patient among excellent and a take make age-related lastly, continuous commitment last course, older work key would discussion our every RGX-XXX I of of expertise. to As
the for majority to Japan. product provide will to of So affects injections than vision update and time. clinical a the turn begin of shown candidates regimen randomized, in with an trials multiple of treatment burden wet a will I to frequent with AMD world the loss more unsustainable over and treatment due Inability our and indicated intravitreal that of prevent program AMD, patients real require anti-VEGF an the in leads been now basis in for vision disease retinal, two RGX-XXX controlled on treatment on to Europe It's long-term million comply injections wet U.S., metabolic patients diseases setting I anti-VEGF loss. neurodegenerative, the that with
levels dose Phase four that be been corrected trial beyond cells Now months. with retinal expression into The coverage, of clinical is adverse RGX-XXX January long-term has and and from a retinal free antibody protein in ability following antibodies these for with durability safety six gene foundation the cohorts subretinal remain inflammation because sensitivity preferred injection multiple in to fab at effect result in which letters neutralizes and XX The reduced vitrectomy. which at has to have continued XX single subjects reported, utilizes of encoding multiple injection across we subjects this vector expressed ability well-tolerated after continuous across the on baseline in preclinical expression, delivery and transduction protein. mean therapeutic route in by peripheral NAV events effective what cohorts, this single retinal or a to administration of decreased subjects animal the establishing been no drug-related central the it a by AAVX X RGX-XXX all candidate best introduced anti-VEGF a vector on expected to routine nine expressed postsurgical the produce Cohort long-term In continuous Mean of broader activity of high fashion the thickness. The models. AAVX visual via demonstrated CRT vector VEGF basis acuity months therapy. serious retinal BCVA and has potential anti-VEGF X that carries of therapeutic reported a of RGX-XXX in ocular NAV a cells neutralizing is the higher Thee is to the because observed XX anti-VEGF of clinical achieve protein consistently of treated the persistent administration trials. year, selected protein. Subretinal nine drug-related to product of improved in inflammation demonstrated continued microns and
been is cleared Additionally, previously Cohort that to RGX-XXX higher administration X.X was we The Cohort filed mean that a per the Phase month that eye dose. application. per on at one trial all additional Xa ECL designed an post-RGX-XXX subjects dose, genome So current in a dosing were to Also now intraocular larger the subjects of in X RGX-XXX as order sample Xb Phase expansion total The in protocol protein expected subjects an enhance subjects clinical we the Phase of last time. announced at recently proceed levels X treated had Cohort six that dosed Phase the design were we by RGX-XXX. based to FDA, month, XX^XX eye trial Today, genome to copies completed under X aqueous with in at Cohort for XX to This RGX-XXX in X a and characterize in month dosed expression from in accelerate assay. one have the further reported expanded IND measured copy development protein the in of XX announced in subjects than at been to-date, x X Xa that we have amendment X.X×XX^XX of an samples trial. detected
We per genome copies X, are currently an of patients, dose recruiting at XX Cohort X.X×XX^XX cohort eye. a additional of
We controlled in data expect wet larger, Phase initiate of the clinical AMD end randomized, from trial present remain by X/X on Phase XXXX. We for track XXXX. topline to to the Xb late to a
chronic X are clinical the anti-VEGF second of XXXX new in RGX-XXX condition trial a towards filing progressing to another Phase an IND for We half that in responds chronic therapy. for additional retinal also
am our the programs IDS and are caused to for going MPS disorders I to IDUA which defects by MPS and genes. turn storage II in I Next, lysosomal
do do II exist, many are affected approved by symptoms which they therapies treatment of the I, of symptoms not MPS present While address central the disease. MPS nervous in benign for are enzyme but and the indicated neurological marketed replacement system patients and
one two has in no of expected MPS Phase programs the eight-week the an cohorts area dose subject safety for the reported in summarize RGX-XXX to address updates well-tolerated of for unmet trial. designed by events a RGX-XXX clinical vector with administration of end been via adverse To assessment, X, the treatment serious for are through at intracisternal XXXX. as of significant NAV the programs, the these has X/X Our December cerebrospinal injection. II one-time At fluid the the first utilizing AAVX of this XXXX, need been into dosed
data interim Additional Phase And and is site update expect an second recruitment in trial ongoing. half this we activation the of present year. X/X the from to clinical
initial FDA treatment protocol, For is subject of subject RGX-XXX trial. recruitment begin on over in age. anticipated Phase MPS for the mid-XXXX. the in recruitment current to focused of X XX Under Enrollment an clinical is clinical the continues I, trial the years
to metabolic Turning franchise. our
the We is correcting treatment of the gene to a the The sponsor allowed sponsor, with trial. six sponsorship. X Phase dose All to subjects following an we in asymptomatic dose important REGENXBIO visibility total homozygous X.X×XX the followed to low-density progress rapidly post-dosing, research over University We trial allow X Cohort transfer to defect caused HoFH. of patient University control Cohort enrollment encoding enhanced a XX, And RGX-XXX familial to will LDL has the genetic to X/X to trial. the a prophylaxis slow in regulatory hypercholesterolemia X corticosteroids Phase will at authorities using and remain normalization reported were trial RGX-XXX. which trial entered RGX-XXX the were kilogram disorder to rare for allow agreement elevation that corticosteroid two the at to XXXX, the with the partner FDA of dose or in XXXX. and of activities new additional of and submitted are responded trial recruitment the RGX-XXX the continue Pennsylvania, RGX-XXX dosed clinical the experienced protocol resumed. per reported in for the ^XX trial the three this transaminases of amendment become initiation drug-related genetic has required mutations taper, for X/X subjects with effectuate the We the address for believe as by by or SAEs Pennsylvania original LDL progressing the underlying receptor. We clinical for HoFH of of genome an bodyweight deficiency. and enrolled of responsible X copies receptor cohorts as we transaminases in potential weeks November into previously no December subjects the by HoFH for lipoprotein new a of
Cohort expect XXXX. the of the We from to data in trial Phase prophylaxis present from with half corticosteroid X/X clinical X interim second
RGX-XXX. a the disease, to to forms This rare, to Batten form turning halt one-time one the aims of disease, most Lastly, CLNX condition. pediatric, progression of common treatment program of Batten develop a of neurodegenerative
of part we franchise. studies. is neurodegenerative We that this last So our enabling initiated announced have IND month
half human XXXX. clinical We first trial file expect to an in a the second IND for in of
month, orphan the November drug that recall may and RGX-XXX You granted the announced that in pediatric announced a that we XXXX, we designation for FDA had agency last rare just granted RGX-XXX, designation. disease
we to with continue and to programs forward work will throughout you we diligent on So drive these year. updates providing forward progress the more to look our
BLA in progress, XXXX, FDA. candidates product or partnered efforts a partners. licensees. technology and Beyond active development our as product application than our for external with was partner approval technology by a to NAV of one biologics these advance clinical XX development NAV XX product in partnered applied has been more submitted are technology candidate license through platform XX, in the our And also licensed the internal being candidates of of December continues
reminder, in have by our NAV treated and developing a NAV technology range As of XXX areas disease are indications. licensees. therapeutic clinical programs in a broad subjects been sponsored trials Over technology licensees
of program and the As licensee NAV that therapy platform AAV drive updates release the efficacy several achieve and our the at versatility our technology Sarepta Audentes, validate today. that the space. press in we Lysogene highlighted safety NAV programs Rocket, technology further and data progress collectively are The additional advancement and believe provide milestones, they Ultragenyx, licensees gene recent of from
uses XXXX. programs milestone A and the both I. XXXX, is for Bayer, of expected the industry and applying their stage of NAV activated And hemophilia by technology the Type NAV a under the technology and Europe clinical royalties REGENXBIO ZOLGENSMA the track XXXX Takeda course, in half upcoming sales regulatory Novartis' eligible on commercial ZOLGENSMA. treatment hemophilia for muscular programs on of AAVX NAV the half in are first of million and ZOLGENSMA XXXX of of clinical payments, in I. future key Type in platform is for of company spinal addition and leaders, atrophy net in the launch in treatment United whom In receive development to ZOLGENSMA the SMA to vector. for in announced milestone the second to potential Japan that Novartis the States treatments active Additionally, and is the two are launch $XX January milestones of of
square made November in of REGENXBIO. the progress part for was of us in be stage our programs, This advancing available of in should for XXXX in provide year install meet was facility XXXX options expansion. addition, our the us overall, technology space, treatments. to capabilities, Rockville, internal And to order which So development the the for The launch of announced potential and growth NAV Significant technology-based cGMP are will in our including to XXXX, four facility to NAV development talent a platform over XXX,XXX own to pivotal us with production facility, by XX pipeline. enable for which of kickoff including our the Maryland. new new needs across feet support will in continue we clinical and
call positioned well broadened and are footprint. With on believe our year's NAV for financials. for will a we review build last ahead, the technology as over transformative I that, achievements of to we turn a the Vit XXXX Looking