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AR124223A1 - Células genéticamente diseñadas y usos de las mismas - Google Patents

Células genéticamente diseñadas y usos de las mismas

Info

Publication number
AR124223A1
AR124223A1 ARP210103337A ARP210103337A AR124223A1 AR 124223 A1 AR124223 A1 AR 124223A1 AR P210103337 A ARP210103337 A AR P210103337A AR P210103337 A ARP210103337 A AR P210103337A AR 124223 A1 AR124223 A1 AR 124223A1
Authority
AR
Argentina
Prior art keywords
polynucleotide encoding
exogenous polynucleotide
cell
seq
amino acid
Prior art date
Application number
ARP210103337A
Other languages
English (en)
Inventor
Michael Naso
Mark Wallet
Barry Morse
Luis Borges
Buddha Gurung
Hillary Quinn
Liam Campion
Jill Carton
Heidi Jessup
Kenneth Brasel
Lucas Thompson
John Wheeler
Original Assignee
Century Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Century Therapeutics Inc filed Critical Century Therapeutics Inc
Publication of AR124223A1 publication Critical patent/AR124223A1/es

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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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Abstract

Se proporcionan células madre pluripotentes inducidas por ingeniería genética (iPSC) y células derivadas de las mismas que expresan un receptor de antígeno quimérico (CAR) y métodos para usar las mismas. También se proporcionan composiciones, polipéptidos, vectores y métodos de fabricación. Reivindicación 1: Una célula madre pluripotente inducida (iPSC) o una célula derivada de la misma que comprende: (i) un primer polinucleótido exógeno que codifica un receptor de antígeno quimérico (CAR) dirigido a un antígeno CD19; (ii) un segundo polinucleótido exógeno que codifica un receptor de superficie celular inactivado que comprende un epítopo específico de anticuerpo monoclonal y una interleuquina 15 (IL-15), en donde el receptor de superficie celular inactivado y la IL-15 están unidos operativamente por un péptido autoproteasa; y (iii) una deleción o expresión reducida de uno o más de los genes B2M, TAP 1, TAP 2, Tapasina, RFXANK, CIITA, RFX5 y RFXAP. Reivindicación 29: Una célula madre pluripotente inducida (iPSC), una célula asesina natural (NK) o una célula T que comprende: (i) un primer polinucleótido exógeno que codifica un receptor de antígeno quimérico (CAR) que tiene la secuencia de aminoácidos de SEQ ID Nº 61; (ii) un segundo polinucleótido exógeno que codifica una variante del factor de crecimiento epitelial truncado (tEGFR) que tiene la secuencia de aminoácidos de SEQ ID Nº 71, un péptido de autoproteasa que tiene la secuencia de aminoácidos de SEQ ID Nº 73, e interleuquina 15 (IL-15) que tiene la secuencia de aminoácidos de SEQ ID Nº 72; y (iii) opcionalmente, un tercer polinucleótido exógeno que codifica un antígeno leucocitario humano E (HLA-E) que tiene la secuencia de aminoácidos de SEQ ID Nº 66; en donde el primer, segundo y tercer polinucleótidos exógenos están integrados en los loci de los genes AAVS1, CIITA y B2M, para eliminar o reducir así la expresión de CIITA y B2M. Reivindicación 66: Una célula madre pluripotente inducida (iPSC) o una célula derivada de la misma que comprende: (i) un primer polinucleótido exógeno que codifica un receptor de antígeno quimérico (CAR); (ii) un segundo polinucleótido exógeno que codifica un receptor de superficie celular inactivado que comprende un epítopo específico de anticuerpo monoclonal y una interleuquina 15 (IL-15), en donde el receptor de superficie celular inactivado y la IL-15 están unidos operativamente por un péptido autoproteasa; y (iii) una deleción o expresión reducida de uno o más de los genes B2M, TAP 1, TAP 2, Tapasina, RFXANK, CIITA, RFX5 y RFXAP.
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