SELB - Earnings call transcript Q4 2020 | Cartesian Therapeutics, Inc.

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Operator

Good morning, and welcome to the Selecta Biosciences Fourth Quarter and Full-Year 2020 Financial Results and Corporate Update Conference Call. Currently, all participants are in a listen-only mode. [Operator Instructions] This call is being webcast live on the Investors & Media section of Selecta's website at www.selectabio.com and it is being recorded.

For opening remarks, I would like to introduce, Brad Dahms, Chief Financial Officer of Selecta. Please go ahead.

Brad Dahms

morning. good and you, Thank Welcome to and XXXX results and fourth update conference quarter corporate full-year call. financial our The press www.selectabio.com. release the reporting our financial results Media of Investors is section our website & available in for And report XXXX be with our filed ended SEC. on XX-K will annual the December the Form XX, year

Executive Officer. Medical Joining Takashi Officer; are Chief Kishimoto, our G. Peter our President, Brunn, today Dr. Traber, Scientific and me and Chief Carsten Officer; our Chief

about in statements today's risks, potential call, our be will and without clinical safety, prospects. the on the SEC. described During plans, statements including regulatory making will Exchange certain various outbreak Commission efficacy, with subject and including product those limitation, filings Form forward-looking statements candidates, future COVID-XX be we most progress partnerships, filed are These Securities the of that financial in Annual expectations, projections recent XX-K, and to our are which Report related made including to our with the

place such Carsten? reliance now these disclaims turn management's XXXX, You over to are today, obligation as undue Brunn, call XX, change. President Carsten I on our views which to unit cautioned the update forward-looking to speak and CEO. any March Selecta like of only statements to and would not statements,

Carsten Brunn

Thank appreciate you, Brad. you Good morning. today. I joining us

biologic year, milestones financial set well diseases. self-tolerance the re-dosing unwanted lifesaving responses and and autoimmune for natural and efficacy immune our past potential for our our amplify several therapies preclinical including clinical exciting in mitigate candidates selectively clinical, of ImmTOR advanced several We strategic of the the the ahead. we've as Over advancements to body's pipeline tolerogenic as have believe made therapies, to table have platform the gene restore an year therapies, to designed develop that key and achieved

milligrams was X.X patient of pegadricase and doses SEL-XXX. SEL-XXX Sobi two inter to a therapies patients SEL-XXX the our partnership getting with chronic our SEL-XXX with treat in we gout. XXXX. of pegadricase. significant with the will make to receiving of fourth start In XXX,XXX momentum XXXX approved We we've in programs. I one X.XX X.X and consist was fraction in DISSOLVE strategic placebo-controlled be will unmet first with de-risked continued months refractory company a the and build dosed. progress co-administered with off milligrams pipeline currently XXX I enzymes trial comprised six of per treatment proprietary remains milligrams with Each and through kicked patients milligrams of a aims In for ImmTOR, of the In trials, DISSOLVE have points, December kilogram. on double-blinded of dose trials XXXX. at enroll trials December, evaluated dose kilogram, serum each two both II DISSOLVE months initiated SEL-XXX only time per kilogram in XX six months endpoint for I'll to and advance year to X safety. the acid XXXX, in good and have chronic the levels extension estimated at continue a It and uric Both six That with deciliter of will at in refractory per medical is only approved a U.S. pegadricase. patients significantly below is the XX to placebo. a our gout per quarter needs program primary case often

we with expected Secondary of intend higher second a of in of of outcomes patient counts, quality enroll tophus endpoints COMPARE and and visible the Based Phase on from activity to the DISSOLVE reported swollen program burden, gout joint learnings are half flare patients proportion Top-line limitation data tender X XXXX. include from life. incidents, tophi. the study,

the tophi is U.S. XX% for in to gout delta tophi recall, with As at patients patients visible endpoint, in primary SEL-XXX of chronic only pegloticase proportion specialty observed factor to believe and care was We of COMPARE patients XX% the had the the baseline closer versus percentage on in with baseline. minimal points patient population a on of XX% tophi XX of will with you visible

of IgA this Hypertension, indication immunoglobulin kidney results approximately loss common second result in accumulates and of can in most incremental result time Our disease renal development estimated an XX% disease. to decreased of function in kidneys. kidney nephropathy, XX% of at immune can are prognosis. causes in the and of environmental IgA when in end-stage enzymes diagnosis our associated and IgAX one in the its It factors GFR proteinuria or the program the occurs antibody nephropathy, that is of a complexes in called renal the poor of accumulation kidney that with AX the disease Genetic cause patients. abnormal

We will a therapy protease. by combining enzyme researching IgA with be novel combination ImmTOR an

complexes by protease. the in studies animal nephropathy. it IgA immunogenic the of root encouraged the the are barrier of IgA origin de-bulk We to to been A the published kidney protease in is shown which has critical bacterial immune development cause IgAX and

ImmTOR immunogenic ability mitigate anti-drug the enzymes. SEL-XXX, to of demonstrated As has with formation the antibodies to

enroll enabled in Meeting AAV trial disease neutralizing more of of and findings the transgene in be our of The The and potential our the Gene to higher therapy. fourth plans moving of ASGCT therapies. AAVX expect AAVX with XXXX. work therapies trial the We With end first ImmTOR antibodies. we to efficacy and transient enable plus clinical AAV demonstrated we therapy the of durable will cause candidate durable SEL-XXX lower of Society with intend are of and have the as volunteers The safety X dosing the non-human to builds by data the addition data recent gene IND top-line transgene ImmTOR for to in of combined initial preclinical May. In lowering A the more our healthy XXXX expression, we to observation costs. in of formation by when ImmTOR empty to ImmTOR patient of therapies. AB as Preliminary co-administration gene SEL-XXX, the ImmTOR to antibodies programs. measured currently will in observed gene product that data dose. formation Annual robust pre-clinical demonstrates leads AAV in the and demonstrated IND filed co-administration preventing higher and primates, intends generate commenced combination of X ratio capsid of no which the robust antibodies study first-in-human-dose-escalation therapy to to capsid ImmTOR the XXXX. ability expression data levels inhibition after of improving potential transgene the of for quarter is in so on further this to antibodies approved enabling a extensive Cell the with recently of alone. first human X study to can Therapy, that at be benefits ImmTOR, platform combination first capsid AAVX are that there by study empty We our potential to an have American gene the AAV in used develop evaluate have to of and vector assessing XX no the randomized its of neutralizing expression forward vector which neutralizing Selecta an present capsid well in commence repeat root of levels containing and Preclinical anti-AAVX in mitigate specific of objective in the to key of anticipated treat Phase the dosing Selecta ImmTOR gene IgG XXXX

systemic gene that that For the long-term therapies the ImmTOR AAV address. a gene of over data therapy time, potential vary may expression demonstrate to has limitation

to in therapies Finally, another mitigated of In antibodies potentially AAV for the the study, gene gene need of thereby neutralizing in be the neutralizing non-human the vector. re-dosed to allowing AAV cannot therapy re-dosing formation these key field. the formation unmet primates, currently antibodies due ImmTOR

with and coma to associated results long-term disorder that for optic Our and which intermittent of may life-threatening. body MMA of AskBio, in mild can of disease usually studies gene cannot model trial lead transforming acidemia potential lives or MMA us triggered Without cases, in nerve This we periods relative from and, quarter in metabolic events fats. its proteins MMA step with and certain have result feeding of closer breakdown delays, hepatoprotective in therapy. Methylmalonic by complications, XXXX. in some stress the realizing intellectual supporting plan pancreatitis. the patients along with of In to injury infections the crisis and infancy of one patients to disorder with in patients the episodes. initiate lead to collaboration move developmental liver the properties intercurrent vary kidney ImmTOR's health second metabolic to appear previous Symptoms death. in the is early monogenic rare chronic our clinical decompensation treatment or a metabolic including Typically, this multiorgan and treatment, failure, well-managed problems, atrophy, may disease, impairments, osteopenia full

significant the we program the MMA-XXX first the the further designation AskBio of FDA report granted by In November This the rare to in patients with patients forward this and address medical are rare of disorder. of affected Selecta by to We seeking in announced XXXX. from unmet end October expect to to we from advancing disease that's data that need hopes FDA underscores designation helping and look XXXX. the this received XXXX, metabolic pediatric cohort which orphan MMA-XXX previously

intellectual OTC deficiency, of XXXX. approach genetic approved blood. are Looking developed levels reducing in is clinic and to cycle, of the ammonia by on inability mutations accumulation gene, control treat are side in blood and lifelong candidate or ornithine is is to an immune body enter in OTC to Most of ahead, X-linked include liver stage, and of excess curative which body of focused seizures, Severe transcarbamylase, therapy being temperature is individual severe at curative. delays complications suppression. proprietary ammonia gene deficiency caused with for associated expected and disorder and genetic our the not early deficiency be OTC the SEL-XXX with amount the which breathing disability. coma, an critical product transplantation control only function symptoms in effects can the urea developmental raise, the is the experience proper OTC Currently, therapies

this for submitted year, promise, to the our Overall has therapy continue updates be additional enabling revolutionary IND We ImmTOR Investigation potential including Agency. on program share European it field. the recently to Medicines this later activities Plan gene holds Pediatric updates on the the to we'll on and significant work

programs. We are to these preclinical and encouraged by data advancing the look promising forward

will we we economics in neuromuscular to of on dystrophies. agreement a including payments, muscular re-dosing upfront, as a well limb-girdle disorders If conduct to the development, and eligible preclinical into license the combination medicines. more enter tiered gene therapy, on be ImmTOR’s option regulatory as would in at commercial up ImmTOR the exercises dystrophy solve powerful including sales. Sarepta wrap giving of critical further platform any continues hurdle to This milestone commercial and looking muscular agreements, additional work these Before Sarepta for access certain significant product potential to royalties Duchenne therapy and patients gene landscape, enabling net its validate

program PBC and look It medical suited through Our bile updates in biliary we has liver scarring studies, tolerance that target is in to well-defined primary platform. ducts. expects to this on inflammation, is later the progressive IND application unmet small need disorder autoimmune chronic significant antigen, the ImmTOR immune advancing an of year. providing well cholangitis. XXXX. damage focus additional leads autoimmune on a enabling program And with this PBC to file a our forward initial of Selecta an to it's IND and

Brad Brad? year run Now I will the results and XX, for quarter full to financial December the our over XXXX. to through fourth turn call ended

Brad Dahms

Sobi $XX.X for periods the the was the Phase in well reimbursement as million which $XXX.X XXXX. million The cash the $XXX.X Carsten. of was and fourth of by operating and million and in our will cost compares million and X was and believe into same DISSOLVE XXXX. million which with primarily increase $XX sufficient as million which our XXXX. million quarter $XX.X second revenue from the period $XX.X respectively year fiscal and in incurred the agreement as fiscal fourth current program. for respectively, as clinical of the XXXX. cash XXXX, December for We compares Thanks, $X.X September recognition year license the XX, We driven in be XXXX shipment position with $XX.X million XX, clinical the resulting had with the same to equivalents cash, quarter for meet liquidity million expenses respectively, million $XX.X Revenue requirements of $X.X supply, XXXX compares quarter R&D and for to restricted of

period. facility SEL-XXX reduction to expenses Phase of December ended program prior for by X result due These internal agreement and of administrative X fourth with This support the $X.X Phase periods combined there Collaboration and initiation increase expenses same Phase The During development offset expenses, XXXX. year initiation patent DISSOLVE expenses X clinical are respectively, The in $X.X the the the with in annual XXXX, million incurred offset increases the under to travel AskBio in quarterly the our to in DISSOLVE clinical and professional reduction million a was subject clinical for the arrangement program and by quarter program. expense COMPARE programs. costs office the and quarter increase in to $XX.X cost and license reimbursement the research in Sobi. the the which timing the for fees and under programs the annual was XXXX was General the XX, decrease of increased of with fiscal and a compared the expense. million $XX.X reflects and million compares were

net $XX.X million quarter year fourth XXXX, same loss $X.XX million or $XX.X the a for to Selecta $X.XX of net or $X.XX a the loss XXXX. and $X.XX fiscal share of or in $XX.X million compared reported share share $XX.X a share a per For million per and periods respectively and or

us, resources plan advance our of Carsten? indications. back clear our call with I'll now candidates work well-defined to priority deploying product operating a have over and multiple of financial the hand in to We ahead Carsten. a

Carsten Brunn

Brad. you, Thank

and the growth questions. great and their many confidence way, earlier, conclude at our our and to team families, who our reiterating our transformational mentioned As year XXXX With take extremely happy of our patients supportive And for people, was investigators continued I'd that, we're been about like we're along a in Selecta. gratitude Selecta. the have company the platform. by excited including our to to

Operator

go Instructions] from Please Kluska Kristen [Operator first ahead. question The Fitzgerald. comes Cantor with

Kristen Kluska

Hi, thanks good for morning, and my everybody questions. taking

at question successful? you So gene time might be this the Especially seen appropriate you're you across when is, potentially the when to be indication patient-by-patient viewed by considering for patients think are of year different benefits conducting the going indication might ages, earlier? or determine more if a if initial basis is first the therapy this how most Do field trials, basis? treated help an more we've dosed to of re-dose the

Carsten Brunn

Thanks, Kristen, that's an excellent question.

address that's we So by that And very obviously I antibodies is think the initially, indication, re-dose. patient-to-patient. but what and really to we're are various as, also able trying the neutralizing defined prevent to to of much ability formation

based have could couple of saw there to that might is In reference A specifically, especially believe is chem to what a There years XX-fold to of likelihood there a out at after look you you obviously years, data from If be adulthood. up that decline four need liver you know that gout years in birth a, indication, to the a and an and there, it significant We grows we liver always applications. expression. high that actually before we transgene is re-dose. indications, have three other re-dosing we

So it individual by age really patients. the varies patients of indication, and

Kristen Kluska

standpoint? meaningful wanted then even potential manufacturing and in you partners impact publication, these your dose to advantages And a But could safety decrease that a cost. have advances twofold in are you you. highlighted how your vector thinking a science Thank and from to recent about I both ask

Carsten Brunn

Yes.

concerns, into relevant. publication the key non-human beta a I lower of cost various obviously would our brings key cost translates to be benefit therapy we therapy, bottleneck is So as definitely driver a meaningful. fronts, it be humans but see if associated a non-human is dose and gene also expression and quite gene indeed lower increase the on the would believe from But in we in goods obviously which Obviously, seen obviously, we're whether primate very into think potential lower we're a primates. encouraged transgene lower a And about a manufacturing advances science have translate safety we by driver but and have in excited humans, well. this will dose is

Kristen Kluska

then across Brad, guidance your if milestone collaborations? that's cash question And factoring potential you thanks. Great, a whether your in any us could remind for payments

Brad Dahms

Yes.

not it include of to any So sort would our - does non-dilutive payments serve own a potential win basis. milestone which extend our extend in

Operator

William ahead. comes Raju next The Blair. with question Prasad from go Please

Raju Prasad

the congrats progress pipeline. question the and taking the for on on Thanks

wanted papers Just if about to the on some could And Transduction a the maybe, re-dose dose initial color bit potential received dose line. you comment think dosage? of talk at have the of because on see Carsten, AAVX? for if some regimen a I in in is trial the Thanks. they then ImmTOR patients that to first And therapy? dosing the provide you - therapy the are the ImmTOR the also another increase to little discussion gene the a on you and Can clinical gene could more need potential already down dose on little there to

Carsten Brunn

Yes, it's maybe the I'll take last a that's one. quick - question,

been and we previously have this if have dosed of don't AAV not patient to population. an gene we ImmTOR, address therapy been So exposed you

to ImmTOR the with So you give dose. initial have

first dose color some you're of benefits. right, we a on terms In dosing, the saw strong

dose So doses you have now obviously, and X.X that almost the value AAV to as are a we also We're We've ImmTOR this and study know, consideration doses and see well appropriate the escalation is. - since X.XX, dose the first looking a with three to as X.X seen we in monthly given or with of our monthly approach what doses study MMA. ImmTOR obviously always first be with to obviously we're three Raju, the capsid non-human have anti with XXX testing of all these - starting the dosed And ImmTOR. human dose feel and with program. patients we take in almost for into very confident gout primate with do XXX

Raju Prasad

you'll taking quick you on MMA forward trial? a ImmTOR that program, be the that follow-up dose have And disclosed in clinical

Carsten Brunn

we Yes, accepted. before we guidance the been haven't filed IND the to want don't IND, so has give

that. to dose previously the But So as much. have off on think hold in have I range discussed, we'll it's I can say we

Raju Prasad

or study be will it is was independent to Phase dose look And X that? able the assume selection at - - you the independent I be results of will

Carsten Brunn

we not Yes, - are necessarily correct. they sequenced. that's Yes,

to this independent learnings in basically we parallel run mean, right them, plan other Yes, in we run to So like that. have on parallel. planned or now some corporate of way I but some in they parallel

Operator

question with go ahead. comes John Please The from next Canaccord. Newman

John Newman

Thank you on just for that had with I the work taking the my in question question. MMA-XXX. doing - MMA, a you're

just formation looking to talk to look what I'm on antibodies, present terms in data? would preliminary you of focus of the Would a be about, you So could potentially don't since - about data for you which hopefully lack little neutralizing curious, initial you to for curious that as thinking just quickly, I preliminary what you things but efficacy types the bit read be might you get know of that if of program?

Carsten Brunn

underlying I to a Obviously, John. address Yes, the trying with mean, therapy. gene question, disease that's good always we're

able such MMA indicator biomarkers think at the levels. of - the to are But antibodies at to the So interested extremely that's be neutralizing we're because an of in looking initial we we for platform re-dose. course, in company, as the as a ability off least disease

John Newman

subsequent would in the MMA-XXX perhaps trial? this would you look program, you potentially initially would with with or to something that explore a And be re-dosing at look that program

Carsten Brunn

efficacy therapy. to a well. if lot a that's the initially varies it re-dose, first kids key first look as mean, at to because lengthy when antibodies will a but the of between part fairly able would that gene it we have good get trial we're necessarily a And to that's I as the that we Obviously, Yes, a you prevent the wouldn't the trial. formation we indication are see treat we think of well, kids. those the trying good those would then approach to able driver of be be question as I off re-dose,

John Newman

Okay.

Just additional just to that preclude that broader that curious you there's with partnerships. Sarepta if limit existing interesting, small could would AskBio perhaps with you extent partnership which AskBio enter anything enter that if the other additional is Obviously maybe from, to question, you partnership with if one and just partnerships you to question excuse partnership choose is you about which whether have into the would could very me a a is, wondering - talk the but into?

Carsten Brunn

we remind And having as very and question we see good selective partnerships in are of know just additional well. as AskBio. that's limitations we it's you Obviously, we have kind can our don't - to partnerships. partnerships Yes, any - two with everyone

collaboration true a co-commercialize where gene XX/XX we strategic plan We to and therapies. have co-develop

is show indication done a also there monogenic to we on that All disorders. partnership But based I is John really research therapy we I have indication then XXX first focused like in - The as We've in for ImmTOR licensed work the be disease. yes, AskBio Pompe MMA. can addressed. neuromuscular which don't lead And diseases gene Sarepta, have is different liver diseases. X, mean with

room of as for as plenty partnerships well. there's inbound, as outbound both well So

Operator

comes Mizuho ahead. from The Difei Securities. with Please go question next Yang

Difei Yang

for and thanks on and progress my the question all congrats side. morning Hi, gene good taking therapy on

gene couple patient patient's MMA. might a and throughout ones might need the Do you general need quick get therapies adolescent you the how for pediatric to life, or just for have a sense re-treatment? So to the times many

Carsten Brunn

hand I'll this Yes, Peter. our CMO, question to

Peter Traber

disease you the And Carsten. re-dosing to look one to very because as re-dosing well Thank be different required. for age depending variable indication. on subjects, the That's be going quite of might really as as a indications to think quite patients, the the at you, needs is say I need as what astute question,

In to in you initial of boys, you're say certain for a say be treating where to getting like determined. be might could presumably really levels re-dose disorders transgene remains the in like to instance at doses, interval which syndrome. going muscles metabolic will disease were OTC their a is couple grows develop. metabolic the it's like to continued and require the neuromuscular how that's disorders to And we or of depend be muscles ImmTOR. expression considered liver able be and young to Muscular if using re-dose what going which will high deficiency, Dystrophy, reflected to Also expression So how in going whose other continue on have MMA

So could re-dosing a their syndrome then or be two it for metabolic could improved or be year longer. and require

going depend regard So on the individual from they it's with really to the gene indication, therapy. get the disease effect initial patient of the the that dose and to

So be it's a that be to going issue to treating genetic opportunity individuals, have we but complex, very going main clearly to to huge it's re-dose diseases. the these is the advantage if

Difei Yang

Thank for you, information. Peter that

onto we of been situation, current pretty hearing construct the durability strategic have are So AAVs in just kind quickly. standpoint, from is durability moving the over all especially losing time

consider platform? you ImmTOR using chem into A would the So getting

Carsten Brunn

exclusive to existing everyone remind an we do Spark A. partnership and rights Spark to Peter, has actually with sorry chem Just, actually have that

Difei Yang

you. Thank

Carsten Brunn

And just Peter are - as plenty well. other liver diseases interest that of based

So…

Operator

next & ahead. Please with question Chad Messer Needham go comes The Company. from

Chad Messer

the patients there. from gene and re-dosing is different Can and lot the program we've - clinical kind how all as some we year? dosing chronic ability data we get which data, to learned discussed gene is revisit later dosed viral know maybe we confident this ready in I rationale therapy the the to vector for there potentially obviously, of dosing where in more But get translate over therapies, for in XXX in the with of an are that a complicated. identifiable enzyme you

evidence of wondering the things studies why you've obviously too. sure clinical And and and other from in Just I'm transit how ability you preclinical lead data? confident are got that

Carsten Brunn

XX that's up key great safety we we Chad. patients ImmTOR, database that in one XXX are months question, dosed Phase Yes, as the obviously with X, and almost be dosing to to of up months. the have large six is have now going I said, the mean to a with And And drivers. we

us definitely most preclinical safety seen of on that's things. building able us recently neutralizing the FDA data then on we've with we're to gives which prevent We formation we're ImmTOR, the non-human interactions confidence. And very So encouraged. of had the antibodies. far. the obviously confidence side around gives We're significant And so primate data the

exploring be think one you chronic said, can treatments, with that will Gene ImmTOR obviously agnostic much looking ImmTOR, you doses dose we're is ImmTOR approaches potentially I a of kind ImmTOR. the gout key to a I indication different of capsid of the which treatment of as differences at play means pretty mean we're just intermittent like fewer approach. therapy study the in which mean, with I where demonstrated you, of have remind we number three much to is antigen. almost

the our obviously be in human but that's So, studies for proof the and will that. really key focus XXXX demonstrate to

Operator

from Wainwright. go Please H.C. Ram ahead. is Selvaraju next question with The

Boobalan Pachaiyappan

for question. Ram my thanks for Selvaraju in Boobalan is taking dialing and This

enrollment on could comment doing you do the interim of to And First analysis, DISSOLVE status all, sometime in you please trial? XXXX? the of plan

Carsten Brunn

great a that's question. Yes,

in September, studies. have last year. of started both December as DISSOLVE I we We II So, in mentioned, started DISSOLVE

topline And enrolling track point. this be we and half will second to on analysis data plan releasing XXXX don’t in of we have studies. are interim the We at those any

Boobalan Pachaiyappan

All right. That's understood.

In your terms of with collaboration Biosciences. IGAN

this some are will what the metrics process not And what deal of items So doing gating candidate to the addition remaining evaluated be human in trials? candidate the are evaluating will to the immunogenicity? to that advance specifically

Carsten Brunn

immunogenicity a worked key the driver. Kei address that where are Yes, studies, enabling more comment guided But is And I'll we’ve in on let doing, we Kei IND let obviously currently I’ll this. detail.

Takashi Kishimoto

of the Sure. I didn't first I’m part happy to. Can it. repeat you the catch question? just

Boobalan Pachaiyappan

the Biosciences. to respect collaboration with With IGAN

the to remaining advance due candidate the So I'm will evaluated metrics to during what the items curious what gating are trials? human And process. be

addition the candidate to illicit In immunogenicity? whether not will evaluating

Carsten Brunn

and you pharmacology studies. yes, need Sure, for to IND, the enabling filing as produce toxicology we IND know

protease ImmTOR So models. with clinical get think is have IgA debulk can as that think pharmacological possible. quickly our on Because animal will IgA we as studies experience the is clinic showing ImmTOR. I molecule. goal Ultimately published good protease though, And a that there rationale-based lot in I involve new complexes combinations of as the of using to IgA into the

So, I now main nephropathy think IgA that limitation right of combination treatment makes for IgA the protease of IgA, using because the sense, immunogenicity. the is

Boobalan Pachaiyappan

Okay.

what So current its moving program, for standard features MMA. much it in care United States? what forward cost of so with the adoption? to patients will is and how your accelerate of market I'm curious And treat MMA-X And and MMA development clinical MMA your unique

Carsten Brunn

right its And speak really own this. have bears the - let suppress Yes, times and for talk really But the limitations, immune and risks liver Peter now But lifetime now mean it's right I let fairly you about - Peter? off I'll this basically. treatment then approach its question. transplantation address limited. to only are since Peter I'll is the options

Peter Traber

Sure, amount and disorder, treating then improves the point care can their the In the liver be the at episodes of - cure need really intake to is dietary MMA restrictions transplant, that markedly although beyond doesn't these it completely intermittently outcomes. it metabolic a which general, be to - this crisis children metabolized pathway, - of that by that approach reducing the foods have.

the this really approach that's - So point. at

that. Almost stays of they care, cost are of in up But have say care. number often hospital these lives metabolic and months In intensive I I basically give that, a you entire crisis. of to for children the will observation really terms the when with their in can't

particular I care point. just number it on of cost can't at a the So, put is this enormous.

Boobalan Pachaiyappan

one of in That's ImmTOR will think you understood. equally immunogenicity, from mitigating be regardless AAV of do And serotype effective the vector used? being me, final

Carsten Brunn

bit looking this. of Yes, Obviously, and Kei done serotypes. various quite have I'll let at we Kei? a - answer work

Takashi Kishimoto

[KD]. agnostic AAVX, And antigen. looked AAVX is really AAVX, preclinical serotypes yes Sure, including at and with other it's to data ImmTOR that the so our multiple consistent we've

an it's some enzyme whether, or So therapeutic of or support preclinical seen protein or the auto-antigen. capsid immunogenicity. other We've to mitigation the data

Operator

Can is [Operator from go line Derek Instructions] comes ahead. muted? Stifel. Archila The hear Please question next you with your us? Derek,

Unidentified Analyst

data many in questions. fitting Thanks then in therapy I and is are antibody could derisk worth for I from first on MMA-XXX. on XXX could to look here your that ongoing further Derek. should guess, of on to technology? formation, And signs biomarker And what How the us may, as study. help study programs what This [Jock] our we we than Thanks. for expect? glean for on Other looking gene the therapy? patients data the of then the activity your if taking us you also

Carsten Brunn

Peter, study. Yes, for the this let I'll Jock the question. on thanks handle MMA - once to meet again color more one

Carsten Brunn

Carsten. you, thank Okay,

So MMA activity. of that mentioned at going looking are the be Carsten earlier biomarkers for to going are to as enzymatic efficacy the we're be study, endpoints

but It's a been very there is one by measure obvious it's in And acid validated for propionic NIH. test MMA that activity activity. which at That sensitive the in to a to clinical and of So and enzymatic the used very clinical the of called is the enzyme. has going - test activity specific the levels is be outcomes enzymatic serum, collaborators developed of the will looking the the ones determine our at patient.

initial the those both as of trial. measuring in So, endpoints we'll be

T-cell to was other The treatment. going immunologic safety so ImmTOR focused as in of antibodies and might by other initial forth aspect trial neutralizing course markers as and well that, that on we're be the and affected be an

So, as it and of will be of empty the both terms in immunogenicity an capsid study. as the well the disease, extensive evaluation

biomarkers looking then a neutralizing of at In other - will also an And of way. trial course, key exploratory antibodies looking be in we’ll addition aspect safety. to will at that be the immunologic that be

that been AAV vector administered with So first time ImmTOR. has

trials future of setup analysis that kind And so, well. safety will - as will be human

Operator

concludes Selecta's question-and-answer remarks. turn to the call. for will the Brunn now back closing I the CEO, Carsten? over This portion call Carsten of

Carsten Brunn

safe and you Thanks concludes operator who today's And again. call. Please healthy. us everyone thank and this stay you, to this joined morning. Thank

Operator

concluded. The attending you for presentation. conference today's now Thank has

disconnect. You may now

Cartesian Therapeutics (RNAC) 11 Mar 212020 Q4 Earnings call transcript