Rhonda. Thanks,
to Turning XX. Slide
gene the rare what owned attractive. regulator and why is GOLD key In diseases. this I findings gene, producing of for safety that a platform. to iron. consistency sRNA wholly These Craig cardiovascular As XX% SLNXXX potentially tolerability breadth hepcidin, approach of risk the SLNXXX observed and consistent hematological generated both effect using demonstrate knockdown a target the of and sRNA world's genetic disease a to which of profile. modality we've after saw Phase our targeting TMPRSSX, of strong of in a clinical programs the studies, robust separate targeting natural high of We're data durability now dose single approach a Lp(a), we that we good factor up a so liver address range population we've preclinically prevents are with and the two affecting mentioned, from the is believe
people effective. high for is lowering independent like our levels Lp(a) health risk There and increases worldwide. this specific with program, aortic drugs unmet factor significantly On are diet that affecting Lp(a) determined existing can not stenosis to an high approved option Lp(a) heart XX, cardiovascular five huge Lp(a) Turning no a Slide not and public for cardiovascular and a long XX, SLNXXX modifiable currently need. Clearly, is and changes. major for in lifestyle disease you or are heart Lp(a) through see attack, treatment serious issue cholesterol Slide failure. one events is genetically risk
ascending evaluated double-blind, our with X dose ongoing, multiple includes Turning with high to both part, looking placebo-controlled Lp(a). at a The single and reported positive program SLNXXX data or the Lp(a) dose and adults XX we above and randomized, program. XX that's at study In single cohorts XX and ascending atherosclerotic is adults Slide top-line dose high that a deciliter. cardiovascular stable per healthy milligrams February, disease This in studies. multiple Phase dose
to Slide the a look top-line reported. SLNXXX here's now data Turning at we XX,
safety important a was an to XXX persisting in assess of clinically Longer-term were up concerns of XXX the XX% reduction SLNXXX tolerated, ongoing manner follow-up is identified. there mentioned, I to days. action. As no from dose-dependent Lp(a) days duration maximum at to XX% a reduced XX%, SLNXXX and with well further
we X Steve breaker embargo lead study our at Dr. Clinic. what Cleveland policy, ACC can in from by investigator, detailed disclose due a April right will in results be the limited now we're presented to While late ACC on Nissen
where program hepcidin, preclinical a Moving our now is SLNXXX ability SLNXXX hemochromatosis. regulation generated and on the of in see also thalassemia, potential thalassemia, to to for the of healthy for broad known has regulate disease orphan iron hepcidin master therapeutic we vera as MDS body. This strong in program. based volunteers. We've of polycythemia regulator data models a number and PV. designations mechanism rare including established in and pediatric designation disease hereditary SLNXXX drug proof beta-thalassemia now We've
SLNXXX the gene SLNXXX cell X.X clinically mentioned, can production. is X.X significant. hepcidin. of we per expression, In grams increased improving red targets and normalizes turn, thalassemia I in that increase As see model, can TMPRSSX's deciliter robust raise An the in systemic This reducing TMPRSSX iron endogenous a levels lowers liver. considered distribution, hemoglobin preclinical per By by grams deciliter. this you
XX, a was dose-dependent per study single milligram induced of doses. Slide persisting last healthy XX that dose This fourfold at presented X.X can hepcidin double-blind in SLNXXX day volunteer was that XX up and full iron. effects now out with results we the a XX after the X single-dose Slide May. see you approximately our throughout adults. Meeting placebo-controlled reported December. shows Hematology last randomized increased in average you to to milligram durable around that Percentage healthy manner Society XX% from Slide We XX reductions study change study a in Annual with of kilogram period. American SLNXXX Turning and baseline serum at
patients. expect proof in XX, an for was iron establish Slide We durable and on and of and to safety ongoing mechanism Turning from showed was of study serum strong GOLD the encouraged thalassemia long these first transferrin reductions summary, the clinical SLNXXX remain important study saturation, duration because this SLNXXX. results study it our and this in a profile by build with action. to and in platform
Slide to the you Turning see ongoing here program our design XX, Phase can of in X thalassemia.
in the includes which enrolled and expect top-line fully cohorts, quarter of dose now single year. third ascending have XX We the data patients this
Phase Craig entry particularly due a disease discontinue is is the criteria mentioned, to enroll MDS highly we and is patient decided to of low-risk subgroup MDS challenging cohorts. required to for As low, very specific MDS study MDS have rare the This a patients. X. small
in plan see the derive thalassemia we we is for and MDS, need most high where near-term. indications opportunity PV While PV can an area we SLNXXX of prioritizing believe we address. and unmet are study one value that Phase particularly hand we is back I'll the well to year. this believe X We later suited SLNXXX With to to Craig. over start the call that,