decision John This by is and breadth into as research to novel modality development. we are robust I has The Intellia which of the Intellia for using system time leader positioned to systemic advance clinical the progress delivery to evaluate you, based good chemical from a a established genome our was is our to therapeutic morning. pursue a programs drawn as programs the Thank LNP pipeline. modular exciting and in us initial to build clinic. Intellia very the multiple platform we editing.
our in Similarly, vivo cell ex of editing engineered setting differentiated, positions based therapies. the forth bring capabilities us application to
been you and of I've As the on the the updating of into the with to with R&D the and enthusiasm to science, vivo side. be grown quality in my pipeline Today, has talent on I opportunity My had got our team beginning science. in impressed I'll progress expectations work exceeded. only and the and the team the deal
know, transthyretin TTR organs of ATTR, the disease to cardiomyopathy. NTLA-XXXX you and/or reduce in by to caused deposition goal in in other of disease the first As diverse knocking the peripheral nerve, The neuropathy by fibro peripheral manifestations vivo of of is or protein leading protein candidate, TTR heart, including and is NTLA-XXXX a our TTR gene. development expression out for amyloidosis treatment
more a from baseline reference, TTR or the level. disease XX% clinical a is with of in delay For the of progression seen reduction
present TTR at protein XXX reduction months primates recall, durability than ongoing of pleased observation As days demonstrated study. you an circulating single data a of six nonhuman in LNP showing formulation additional post lead TTR. Today, may of circulating our reductions sustained TTR to average we XX% through administration in [indiscernible] we're of targeting greater
We reduction significant very of these by they to that be clinically achieve expected protein results TTR as levels can show encouraged we are efficacious.
As to genome This CRISPR/CasX protein reach XX data a with recent underscore anticipated by editing most single single The dose. liver of observation the in we patients. is studies once and through for continues observed editing effect power consistent earlier NHP reduction administration, which is concomitant CRISPR/CasX, months following these TTR of sustained. levels to we with the of knockdown, curative develop data durable treatment
steps to are as the the of that John course this To to eager taken move We end, have as program NTLA-XXXX. clinic evaluation several possible. important of soon we clinical towards mentioned, as
package conducted will Most both pre-IND IND form studies, our with of of X toxicology the we recently, our the meeting FDA our enabling Phase study. which shortly design IND and initiated and thereafter
In NTLA-XXXX addition, we X refine All as commence established application support submit supply to together, the IND guidance expected in the our our prior Phase expect to this of chain end mid we year. manufacturing material by XXXX. enables for now operations to us to
XX-XX of commercialization have we with are agreement development lead co-development Regeneron. a party program and and the reminder, a which for co-commercialization this the for As we
turn now insertion efforts our Let's in targeted to the liver.
demonstrated the transgene in quarter's into call, of ASGCT as and recall, Factor locus. using first at IX on albumin inserted nonhuman in as insertion the last April, the discussed we mediated may You CRISPR/CasX targeted primates liver presented
A hybrid Factor proprietary patients. reminder, that's vehicle delivery a The our our LNP vector missing B clotting Factor combines blood As hemophilia in the IX study the delivery CRISPR IX used with B [indiscernible] including an protein or system defective gene. which
in nice our same the change ATTR demonstration of random delivery being program Targeted a insertion should used in guide approach safety vectors. efficacy. of both clear and We of modularity provides our of the our the LNP targeted In retroviral with the mutagenesis CRISPR is the the one RNA. system the reduce risk integration insertion believe due as to platform, sole advantages
and efficacy of insertion cure a the targeted single durable disease. addition, should In with course provide treatment potentially
were now administration through the period. complete study two single Today, we to Factor showing NHP results XX that from months IX levels of share circulating the study day durable are pleased human at protein observed the the
of consistent reported these humans. IX rodent circulating Importantly, levels were protein [Indiscernible] within sustained the is observation. the months circulating levels levels Factor with in in protein XX NHPs throughout normal IX range of protein study Factor were in which
our in initial working Our platform indications. targeted the knockout of beyond actively Intellia’s and genetic setting Factor insertion approaches scope continue the program. addressable in We're to to expanding well IX diseases the progress Regeneron with significantly evaluate CRISPR/CasX vivo
additional present also progress upcoming on you we're in vivo look forward of scientific parallel, and wholly-owned of as other our updating interest transgenes several We development our insertion on targeted efforts. expect conferences. therapeutic we part to to evaluating In at independently data
strategy. therapy move Let's cell to engineered our
Here, range of to similarly solid platform a based design hematological cells a treat leveraging and we tumors. are modular approach to engineered
Our will myeloid on focus for TCR program. owned receptor or be T our or acute replacement leukemia cell wholly our approach today AML
inserts over As locus. knocks by key out believe provide TCR the both TCR and the eliminating CAR-T We alpha replacement a safety endogenous approach genes in therapeutic beta replacement reminder, TCR should the Intellia’s approach our and advantages and in efficacy. TCR
from enhancing reducing First, inserted the inserted physiology, miss versus [indiscernible] normal the the TCR, by by preserving between and and and [indiscernible] second stabilizing result by third, could T TCRs. cell of therapeutic expression [indiscernible] endogenous that
immediate less type because in This recognize therapy there is than broaden XX,XXX for they medical initial TCRs marrow new without CAR-T. overall bone approximately significant range and cell U.S. need. replacement cancer alone. the In rapidly is and unmet engineered Further, for tumor [indiscernible] our can rate. with with is TCR cases XX% of treatment addressable antigens approach AML. the were The blood AML basis fatal the of tumor five-year disease program XXXX survival
only Over the in three for there treatment decades options patients. limited two have to been AML the past advances
blast for are is in X our lead XX% than or as WTX. other AML hematological therapy TCR solid types. based targeting numerous overexpressed AML Tumor as WTX Wilms and of greater in For tumor well we
of call, candidates multiple WTX lead WTX As outlined recognize last on we identified that primary the TCR quarter's epitope interest.
testing we data have candidate, collaborators vitro announce of inform functional derived and have and nomination simultaneous panel of previously therapy remain TCRs guided of lead development our endogenous XX our for and showing successful that Every TCR year were in fully patient studies the These functional in to at blast. xenograft including be insertion of track. studies cell capable first Raffaele. on by killing models we Ospedale are of this initiated engineered we a of derived these end San which candidates. specifically and WTX we efficiently which generated engineered of the AML will We patient with will TCR Today with various knockout pleased also cells
evaluation. clinical we In establishing for manufacturing are capabilities support addition, relationships contractor to
numerous our significant our in approach, overexpressed is tumor we other pursuing WTX broad believe to expect replacement solid tumors. unlock array guidelines. a of TCR types, immuno-oncology be to will we program including the As foundation will Furthermore, [ph] enhance opportunities our further we AML evaluate cultures if
I quarter's financial With second would turn that, go will statement. Glenn? over the who the like call to through to Glenn,
