US FDA tau tso cai CTX110 Regenerative Medicine Advanced Therapy (RMAT) tsim nyog!

Lub tuam txhab kev kho noob caj noob ces CRISPR Therapeutics tsis ntev los no tau tshaj tawm tias US Food and Drug Administration (FDA) tau tso cai CTX110 Regenerative Medicine Advanced Therapy (RMAT) kev tsim nyog: qhov no yog gene editing allogeneic CAR-T cell tag nrho los ntawm lub tuam txhab Kev Kho Mob tau siv los ua. kho relapsed los yog refractory CD19+B cell malignancies. CRISPR Therapeutics siv CRISPR/Cas9 gene editing thev naus laus zis los ua peb lub noob hloov kho ntawm T hlwb los ntawm cov neeg pub noj qab haus huv, tso cai rau nws cov CAR-T hlwb ua" off-the-shelf".

Regenerative Medicine Advanced Therapy (RMAT) yog ib txoj kev ceev ceev (fast track) tsim los ua kom txoj kev loj hlob sai thiab kev pom zoo ntawm cov kev kho tshiab tshiab thaum Tebchaws Meskas tau hloov kho cov tshuaj kho dua tshiab ntawm" 21st Century Cures Act" ; nyob rau lub Kaum Ob Hlis 2016. Channel) system. RMAT tuaj yeem yog kev kho ntawm tes, kho cov ntaub so ntswg engineering, tib neeg lub hlwb thiab cov ntaub so ntswg, lossis lwm yam kev kho mob sib xyaw ua ke uas suav nrog cov khoom siv tshuaj kho dua tshiab.

Yuav kom tau txais RMAT tsim nyog rau cov tshuaj nyob rau hauv kev tshawb fawb, yuav tsum muaj cov ntaub ntawv tshawb fawb ua ntej los ua pov thawj tias cov tshuaj muaj txiaj ntsig zoo hauv kev kho mob, ncua sijhawm, thim rov qab lossis kho tus kab mob hnyav lossis kev phom sij rau lub neej lossis tsis ua raws li kev xav tau kev kho mob. RMAT kev tsim nyog suav nrog tag nrho cov kev nyiam tshaj plaws ntawm Fast Track Qualification (FTD) thiab Breakthrough Drug Qualification (BTD), suav nrog kev muaj peev xwm ntawm kev cuam tshuam ntxov nrog FDA, kev tshuaj xyuas ua ntej, thiab kev pom zoo nrawm.

Dr. Samarth Kulkarni, CEO ntawm CRISPR Therapeutics, tau hais tias:" Qhov kev tsim nyog RMAT no yog ua raws li cov ntaub ntawv kho mob uas txhawb nqa peb tau muab rau tam sim no. Qhov no yog lub sijhawm tseem ceeb, qhia tias FDA tau lees paub CTX110 hauv kev kho mob hematological malignancies. Transformational peev xwm. Peb tos ntsoov yuav ua haujlwm ze nrog FDA los txuas ntxiv peb cov kev siv zog los coj qhov kev kho tshiab tseem ceeb no rau cov neeg mob."

Autologous CAR-T thiab allogeneic CAR-T (Duab qhov chaw: crisprtx.com)

CTX110 yog gene-edited allogeneic CAR-T cell therapy muab los ntawm cov neeg noj qab haus huv pub dawb, tsom rau CD19, uas tam sim no yog lub hom phiaj nrov rau CAR-T txoj kev loj hlob. CD19 yog ib hom ntawm pawg sib txawv antigen thiab ib qho tseem ceeb membrane antigen ntsig txog B cell proliferation, sib txawv, ua kom muaj zog thiab cov tshuaj tiv thaiv kab mob.

Tam sim no, ib sab caj npab, ntau qhov chaw, qhib-daim ntawv qhia theem 1 kev sim tshuaj CARBON yog ntsuas qhov kev nyab xeeb thiab kev ua tau zoo ntawm ntau qib ntawm CTX110 hauv kev kho mob ntawm refractory B-cell malignancies. Qhov kev sim tau tso npe rau cov neeg laus uas tau rov ua dua lossis rov ua dua tshiab B-cell CD19+ malignancies uas yav dhau los tau txais tsawg kawg 2 kab kev kho mob.

Cov ntaub ntawv tshaj tawm thaum Lub Kaum Hli xyoo no tau pom tias nyob rau hauv kev npaj-rau-kev kho mob (ITT) cov pej xeem, tag nrho cov kev tso tawm hauv cov neeg mob uas muaj B-cell lymphoma loj (LBCL) ntawm koob 2 (DL2: 100x10E6) thiab ntau dua ntawm ib koob tshuaj. CTX110 kev kho mob Tus nqi ORR tau mus txog 58%, thiab qhov ua tiav kev tshem tawm (CR) mus txog 38%. Tsis tas li ntawd, nyob rau hauv cov neeg mob LBCL, qhov kev tshem tawm tau ntev ntev, nrog rau 6-hli CR tus nqi ntawm 21%, thiab kev tshem tawm ntev tshaj plaws tau khaws cia rau 18 lub hlis tom qab thawj zaug infusion.

Hauv qhov kev sim no, tus nqi tso tawm thiab kav ntev ntawm CTX110 tau zoo ib yam li kev kho mob autologous CD19 CAR-T pom zoo raws li ITT. CTX110 muaj cov yam ntxwv zoo thiab sib txawv ntawm kev nyab xeeb, tsis muaj cytokine release syndrome (CRS) ntawm qib 3 lossis siab dua, qis tus kab mob thiab qhov tshwm sim ntawm lub cev tiv thaiv kab mob cuam tshuam nrog cov kab mob neurotoxic (ICANS).