HRP20211910T1 - Mnd promotor himerni antigen receptori - Google Patents

Mnd promotor himerni antigen receptori Download PDF

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Publication number
HRP20211910T1
HRP20211910T1 HRP20211910TT HRP20211910T HRP20211910T1 HR P20211910 T1 HRP20211910 T1 HR P20211910T1 HR P20211910T T HRP20211910T T HR P20211910TT HR P20211910 T HRP20211910 T HR P20211910T HR P20211910 T1 HRP20211910 T1 HR P20211910T1
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HR
Croatia
Prior art keywords
paragraph
ltr
cell
lentiviral vector
promoter
Prior art date
Application number
HRP20211910TT
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English (en)
Inventor
Richard Morgan
Kevin FRIEDMAN
Byoung RYU
Original Assignee
2Seventy Bio, Inc.
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Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=54333432&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HRP20211910(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 2Seventy Bio, Inc. filed Critical 2Seventy Bio, Inc.
Publication of HRP20211910T1 publication Critical patent/HRP20211910T1/hr

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    • C07K2317/00—Immunoglobulins specific features
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    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Claims (15)

1. Lentivirusni vektor sadrži polinukleotid koji sadrži pojačivač mijeloproliferativnog sarkoma virusa, negativno kontrolno područje izbrisano, vezno mjesto početnice dl587rev (MND) zamijenjeno, promotor operativno vezan na nukleinsku kiselinu koja kodira himerski antigenski receptor (CAR), pri čemu CAR sadrži: (a) scFv koji veže antigen sazrijevanja B-stanica (BCMA); (b) CD8α zglobnu regiju; (c) CD8α transmembransku domenu; (d) CD137 kostimulatornu signalizacijsku domenu; i (e) CD3ζ primarnu signalizacijsku domenu.
2. Lentivirusni vektor iz stavka 1 u kojem CAR dalje sadrži signalni peptid
3. Lentivirusni vektor iz stavka 1, pri čemu lentivirus je odabran iz skupine koju čine: virus humane imunodeficijencije (HIV); i HIV-2.
4. Lentivirusni vektor prema bilo kojem od stavaka od 1 do 3, pri čemu sadrži lijevi (5') lentivirusni LTR, psi (ψ) signal za pakiranje, središnji polipurinski trakt / DNA preklop (cPPT / FLAP), retrovirusni izvozni element; MND promotor operativno povezan sa CAR-om iz stavke 1; i desni (3') lentivirusni LTR.
5. Lentivirusni vektor iz stavka 4, koji nadalje sadrži: a) heterolognu poliadenilacijsku sekvencu; b) heterolognu poliadenilacijsku sekvencu koja je poliadenilacijski signal goveđeg hormona rasta ili sekvencu poliadenilacije zečjeg beta-globina; ili c) element za posttranskripcijsku regulaciju virusa hepatitisa B (HPRE) ili element za posttranskripcijsku regulaciju virusa hepatitisa svisca (WPRE).
6. Lentivirusni vektor iz stavka 4 ili 5, pri čemu: (a) je promotor 5' LTR-a zamijenjen s heterolognim promotorom; (b) je promotor 5' LTR-a zamijenjen s citomegalovirusnim promotorom (CMV), Rous sarkoma virusnim (RSV) promotorom, ili Simian Virus 40 (SV40) promotorom; (c) 3' LTR sadrži jednu ili više modifikacija; (d) 3' LTR sadrži jednu ili više brisanja; ili (e) 3 'LTR (f) auto-deaktivacijski (SIN) LTR.
7. Stanica imunološkog efektora koja sadrži lentivirusni vektor iz bilo kojeg stavka od 1 do 6.
8. Stanica imunološkog efektora iz stavka 7, pri čemu je stanica imunološkog efektora T-limfocit.
9. Stanica imunološkog efektora iz stavka 7, pri čemu je stanica imunološkog efektora prirodna stanica ubojica (NK stanica).
10. Sastav koji sadrži stanicu imunološkog efektora iz stavka 8 ili 9 i fiziološki prihvatljiv ekscipijens.
11. Sastav prema stavku 10 za upotrebu u postupku liječenja raka kod subjekta kojem je to potrebno.
12. Sastav prema stavku 10 za upotrebu u postupku liječenja hematološkog maligniteta kod subjekta kojem je to potrebno
13. Sastav za upotrebu prema stavku 12, pri čemu je hematološki malignitet malignitet B-stanica
14. Sastav za upotrebu prema stavku 13, pri čemu je malignitet B-stanica izabran iz skupine koju čine: multipli mijelom (MM), ne-Hodgkinov limfom (NHL) i kronična limfocitna leukemija (CLL).
15. Sastav za upotrebu prema stavku 14, pri čemu MM izabran iz skupine koju čine: simptomatski multipli mijelom, tinjajući multipli mijelom, plazma stanična leukemija, nesekretorni mijelom, IgD mijelom, osteosklerotski mijelom, solitarni plazmacitom kosti i ekstramedularni plazmacitom..
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