Clinical Application Scenarios
Cov Kab Mob Neuromuscular
Txhua tus tib neeg cov ntaub ntawv kho mob rau FS344 yog los ntawm kev sim tshuaj kho noob. Hauv Phase I/IIa kev sim ua nyob rau hauv Nationwide Children's Hospital, rau tus neeg mob uas muaj Becker leeg nqaij dystrophy (BMD) tau txais ob sab quadriceps direct txhaj ntawm AAV1.CMV.FS344. Hauv Cohort 1 (3 × 10¹¹ vg/kg/leg), ob tus neeg mob tau pom tias muaj kev txhim kho ntawm 58 meters thiab 125 meters, feem, hauv 6-feeb taug kev xeem; hauv Cohort 2 (6 × 10¹¹ vg/kg/leg), ob tus neeg mob tau pom tias muaj kev txhim kho ntawm 108 meters thiab 29 meters, raws li. Kev ntsuam xyuas histological pom tau tias txo qis fibrosis, txo qis hauv nruab nrab nucleation, thiab myofibril hypertrophy. Tsis muaj qhov tshwm sim tsis zoo tshwm sim.
Tib pab neeg no kuj tau soj ntsuam cov noob kev kho mob hauv cov neeg mob uas muaj kev sib koom ua ke ntawm lub cev myositis (sIBM), muab txoj hauv kev cuam tshuam rau cov kab mob refractory no raws li kev cuam tshuam ntawm ob qho tib si ntawm follistatin- ua kom cov leeg muaj zog thiab txo cov kab mob mononuclear cell infiltration.
Preclinical Translational Studies: Hauv cov qauv nas, ib qho kev txhaj tshuaj intramuscular ntawm AAV1-FS344 ua rau muaj kev nce ntxiv ntawm ntau dua 20% hauv cov leeg nqaij hauv cov tsiaj qus- hom C57Bl/6 nas, ua tau zoo dua lwm cov tshuaj myostatin inhibitors xws li GASP-1 thiab FLRG. Hauv mdx nas (Duchenne leeg nqaij dystrophy qauv), pawg koob tshuaj siab (1.5 × 10¹² vg / kg) pom qhov nce 15-fold hauv cov ntshav follistatin, nrog kev txhim kho koob tshuaj hauv cov leeg nqaij ntev dua 560 hnub.
Hauv cov kev tshawb fawb uas tsis yog -tib neeg primate (macaque), AAV1-FS344 tau ua tiav qhov loj ntawm cov leeg nqaij thiab lub zog raws li kev tswj hwm ntawm cov leeg nqaij tshwj xeeb (MCK) thiab kev txhawb nqa thoob ntiaj teb (CMV), yam tsis tau soj ntsuam cov kab mob lossis kev ua haujlwm hloov pauv hauv lub cev xeeb tub.
Kev Ua Si Cov Tshuaj Kho Mob thiab Lub Cev Kom Zoo Tshaj Plaws: Raws li cov txheej txheem ntawm myostatin inhibition txoj hauv kev, FS344 tau siv rau hauv cov tshuaj ua haujlwm thiab cov tshuaj kis las kom kov yeej cov leeg nqaij loj. Cov pej xeem lub hom phiaj suav nrog cov kws tshawb fawb qib siab uas tau ua tiav kev cob qhia thiab kev noj zaub mov kom zoo thiab tab tom nrhiav kev txhawb nqa lub cev hnyav ntxiv. Thaum siv ua ke nrog GLP-1 receptor agonist poob phaus tswj, FS344 tuaj yeem tiv thaiv cov teebmeem catabolic thiab tiv thaiv cov leeg nqaij.
Nws yog ib qho tseem ceeb kom paub meej tias cov ntaub ntawv preclinical tau hais los saum toj no yog ua raws li AAV tus kab mob vector xa tawm - tus kab mob txuas ntxiv nthuav tawm FS344 hauv cov leeg rau lub lis piam mus rau xyoo, uas yog qhov txawv ntawm cov tshuaj pharmacokinetics ntawm kev txhaj tshuaj ncaj qha ntawm recombinant FS344 protein. Qhov ib nrab-lub neej ntawm cov proteins sib xyaw yog ntsuas hauv cov xuab moos; Yog li ntawd, cov tshuaj txhaj tshuaj hauv zej zog tsis tuaj yeem rov ua dua qhov cuam tshuam ntev ntawm kev kho noob.
Cov Lus Qhia Txog Kev Siv thiab Siv
Dosage and Route of Administration
Tib neeg cov ntaub ntawv pharmacokinetic rau kev txhaj tshuaj ncaj qha ntawm recombinant FS344 yuav luag tsis muaj; tag nrho cov txheej txheem hauv zej zog yog cov ntaub ntawv ntxiv los ntawm cov ntaub ntawv tsiaj thiab kev txiav tawm theoretical. Tam sim no, muaj peb txoj cai tseem ceeb:
Routine 1 (Intramuscular Txhaj rau Hnub Kev Kawm): 50-100 mcg rau ib qho kev txhaj tshuaj, intramuscularly 30 feeb ua ntej kev cob qhia (IM), tsuas yog siv rau hnub kev cob qhia, txiav tawm rau hnub so. Lub sijhawm siv 8 lub lis piam tom qab 8 lub lis piam tshem tawm. Cov chaw txhaj tshuaj muaj xws li deltoid, vastus lateralis, lossis gluteus medius.
Option 2 (Subcutaneous Txhaj Cycle): 100 mcg / hnub subcutaneous txhaj (SC) rau 10-hnub thauj khoom, ua raws li kev saib xyuas ntawm 100 mcg txhua hnub, rau tag nrho lub voj voog ntawm 30 hnub.
Option 3 (Low-Frequency Subcutaneous): 100-300 mcg subcutaneous txhaj, 2-3 zaug hauv ib lub lis piam.
Kev txhaj tshuaj intramuscular yog suav tias yog qhov zoo dua rau kev xa cov tshuaj mus rau cov leeg nqaij, tab sis raws li cov khoom noj muaj protein ntau, qhov chaw txhaj tshuaj thiab qhov chaw effector tsis tas yuav tsum nruj me ntsis. Siv cov tshuaj insulin 29-31G; ib qho kev txhaj tshuaj yog feem ntau 0.05-0.2 mL.
Reconstitution Procedure: FS344 yog muab los ua lyophilized hmoov, feem ntau hauv 1 mg vials. Kev kho dua tshiab yuav tsum tsuas yog siv cov dej tsis huv rau kev txhaj tshuaj uas muaj 0.9% benzyl cawv.
Standard Reconstitution Protocol: 1 mg ntawm lyophilized hmoov yog ntxiv rau 2 mL ntawm sterile dej, ua rau qhov kawg concentration ntawm 500 mcg / mL. Ntawm qhov concentration no, 50 mcg sib haum rau 10 units ntawm insulin syringe, thiab 100 mcg sib haum rau 20 units.
Kev khiav hauj lwm txheej txheem: Cia cov hmoov lyophilized vial kom ncav cuag chav sov li 10-15 feeb; disinfect lub stopper nrog ib tug cawv swab; kos lub ntim ntim ntawm cov tshuaj tua kab mob; ntxig lub koob obliquely rau hauv lub stopper thiab maj mam txhaj cov tshuaj nyob rau hauv lub vial phab ntsa, tsis txhob sib cuag nrog cov hmoov; maj mam dov lub vial rau hauv koj lub xib teg los pab kev tawg, tsis txhob co-FS344 yog cov protein uas tsis muaj zog, muaj zog co tuaj yeem ua rau denaturation; cov tshuaj yuav tsum tau meej thiab pob tshab tom qab tiav dissolution. Muab pov tseg yog tias nws dhau los ua huab, muaj cov khoom, lossis tsis muaj xim. Cov txheej txheem tawg yuav siv sijhawm 2-5 feeb.
FAQ
Q: Qhov kev txiav txim yam tsawg kawg nkaus yog dab tsi?
A: Yam tsawg kawg nkaus kom muaj nuj nqis yog 10 vials / teeb.
Q: Nws siv sijhawm ntev npaum li cas rau nkoj?
A: Hauv - cov khoom xaj yuav raug xa tuaj hauv 24 teev.
Q: Dab tsi purity theem koj tuaj yeem muab tau?
A: Txheem purity 99%.
Q: Qhov kev pabcuam courier koj siv? A: Koj tuaj yeem xaiv los ntawm FedEx, DHL, UPS, thiab kev pabcuam xa ntawv.
Q: Ntev npaum li cas shipping?
A: Kwv yees li 10-15 hnub.
Q: Koj puas tuaj yeem muab COA (Certificate of Account) daim ntawv tshaj tawm kev soj ntsuam zoo thiab HPLC (High-Kev Ua Haujlwm Kev Ua Haujlwm) qhia?
A: Yog lawm, tag nrho cov khoom raug kuaj xyuas ua ntej ua zaub mov xa tuaj.
Q: Koj puas txhawb OEM hom?
A: OEM branding tau txais kev txhawb nqa, raws li qhov yuav tsum tau ua kom tsawg kawg nkaus.
Q: Yuav ua li cas tom qab- kev pabcuam muag khoom?
A: Tom qab cov neeg siv khoom kos npe rau cov khoom, yog tias nws tau lees paub tias qhov teeb meem zoo yog tshwm sim los ntawm peb cov khoom, lawv tuaj yeem txaus siab rau kev hloov pauv dawb lossis nyiaj rov qab. Peb ua raws li lub hauv paus ntsiab lus ntawm kev ua haujlwm thiab lub luag haujlwm, ua tib zoo teb thiab daws txhua yam teeb meem zoo, tsis muaj lub luag haujlwm lossis zam teeb meem, thiab ua qhov zoo tshaj plaws los tiv thaiv cov cai thiab kev nyiam ntawm peb cov neeg siv khoom.
Q: Txoj kev them nyiaj twg muaj?
A: Peb txhawb nqa kev hloov pauv hauv txhab nyiaj, Bitcoin, Hloov pauv, thiab Xoom.
Cim npe nrov: kev tshawb fawb-qib khov- cov hmoov qhuav rau kev siv lub cev: follistatin 344 cas: 80449-31-6, Tuam Tshoj kev tshawb fawb-qib khov-qhuav hmoov rau lub cev siv lub cev: follistatin 344 cas: 80449-31-31 manufacturers, Hoobkas-6
