and Thank you, us was The financial ImmTOR across business therapies programs. therapy joining advance with diseases. clinically appreciate Brad. to I start our autoimmune XXXX therapies and and I’ll as enzyme of validated milestones, platform in morning. several strategic well substantial our gene you marked as with therapies, outset our pipe today. particularly Good progress, that Enzyme gene continue
continued treat significant which chronic progress getting a to unmet gout, comprised pegadricase. that Sobi proprietary of make is ImmTOR is need. SEL-XXX in We've ministers Our are to refractory which patients was court uricase SEL-XXX approved with licensed program, to
X.X dose have and and I double-blinded milligrams will trials levels December dose DISSOLVE of endpoint with DISSOLVE was clinical one have kilogram. deciliter DISSOLVE two X controlled be milligrams on trials uric the kicked of the at time placebo dosed. consists Both months XX In doses As trials per two six kilogram of third per both months milligrams DISSOLVE aims SEL-XXX to per XXX serum only of quarter XXXX. primary below X a ImmTOR, will the as first Each initiated patients kilogram at placebo. each a pegadricase in six SEL-XXX. a points, patient of safety. in and of was and enroll XX September, II for X.XX I X.X XXXX and In months trial milligrams the extension reminder, off per evaluated our acid program
Secondary the result indication a is second of IgA XXXX. when antibody immune kidney or disease in application end occurs the that endpoints enzyme therapies. the and no IgAX causes complexes a immunoglobulin or for There by of with approved development of proactively new leverage from in file DISSOLVE to of program second impact called cause are most drug expected to kidneys activity ongoing kidneys. success program in and Top patient the schedule, of the the intend proteases potential half procedures environmental COVID the of pace include an currently the and put IgA outcomes have IgA as incidents, Genetic nephropathy its that accumulated gout of kidney in burden, ImmTOR of and is limitation counts, are AX tender nephropathy, can ID that joint progressing life. We're reported XXXX. pleased tophus an accumulation of several factors We the pandemic. the place combines we of which SEL-XXX into flare investigation common quality an disease. one this minimize line of abnormal on with swollen enrollment, and the data in
intend we development. treat therapy, overcome However, of to IgA previous with root our proteases disease novel cause limitations the combination and associated the with
In turning February. combined our X with dose-escalation first-in-human initiated ImmTOR we the optimal AAVX AskBio, trial phase DNA determine the to to therapies. therapy of capsid The in the and programs. collaboration is empty being mitigate healthy with conducted gene no vector aims capsids of trial used volunteers in Now AAVX containing ImmTOR to dose to SEL-XXX, in of an gene formation antibodies
of made We remain are with We and report the pleased with expect fourth date. to data AskBio to together track quarter the on XXXX. in topline progress
down program. our fats. gene Moving break gene rights to and or treatment methylmalonic to strengthened MMA-XXX the by monogenic portfolio of proteins the body separate in therapy a which candidate for ImmTOR recently acidemia and We for disorder MMA-XXX an MMA, wholly-owned obtaining certain update cannot the rare our exclusive
MMA-XXX the and give the biomarker the tolerability. genetic related our program to in of preclinical Due to study decision production platform] demonstrated We're of XXXX that & The independently Selecta’s review IND look for further of ImmTOR third [ImmTOR on which gene the is benefits And therapy will there's the to to their AskBio ImmTOR which smoothly. enabling Selecta’s to In an American and fund acquired manufacturing by exercise vivo. strategic to potential XXXX. fourth IND more durable clinic studies to and will Selecta proceed well recent team advancement programs. the primates, in disorders. manufacturing is antibodies all prioritization for the therapy in we advancing co-administration portfolio ImmTOR. Therapy internal findings MMA-XXX of non-human redosing expression expected to any progressing evaluate meeting utilize MMA-XXX based in immunogenicity of continues observed the of disease, various delay, potential party of vector leads week, rights ImmTOR explore and the and and of therapy grateful through of gene antibodies and a in in the robust quarter preclinical for these commence clinical forward as in The mitigate XXXX to phase and an of Selecta of annual neutralizing data program, expect combination Gene therapy this AskBio’s neutralizing platform specific conducted the have Bayer, to development. antibodies. prevent AAV no with programs X/X higher to partnership impact as presented gene demonstrating inhibition extensive gene AAV to transgene AAV Earlier into enable AAV ImmTOR in the that Society we the at of safety Cell builds we treatments on AskBio
in OTC which proper is proprietary excellent genetic our function the being is urea OTC OTC critical deficiency ahead, developed transcarbamylase cycle. treat SEL-XXX, therapy genetic the an of gene gene, Looking for ornithine candidate, caused by or mutation deficiency. is product to disorder
We clinical XXXX. trial application expect or and/or to file CTA in a IND an
our Plan or Pediatric to was SEL-XXX XXXX. We the will an also on February submitted PIP European Medicines Agency which in provide Investigation for pediatric update committee
at the potential under holds to gene therapy This LGMD with ImmTOR gene including work payment the completion Sarepta. Before muscular Sarepta a X DMD agreement dystrophies significant and ImmTOR’s the received combination dystrophy research looking the Therapeutics continues certain therapy, conduct to Overall, and in subtypes. of million validates [fields]. license has promise option neuromuscular ImmTOR our to We related disorders the further and recently preclinical or revolutionary limb-girdle preclinical a platform. potential on be study for we of wrap-up of Duchenne muscular or milestone
generated to of repeat antibodies durable data addition date, encouraged by formation by the of potential dosing expression enable to and dose. to in the transgene preventing the demonstrating more after robust ImmTOR We’re neutralizing the of first
to unmet primary damage, of suited biliary inflammation, focus scarring well and on is well target significant IND of it's studies defined with enabling ducts. ImmTOR program need cholangitis. is liver advancing through a platform. initial is an autoimmune the immune the Our that antigen, tolerance autoimmune bile chronic, It to leads small progressive medical our application disorder PBC a and
and in find We this expect additional we PBC on in half an to look later second updates to program year. the providing XXXX forward IND this of
turn over XXXX. first ended our through Brad March I'll quarter run the Brad? results Now, XX, the call to for financial to