AR126207A1 - Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogénicos - Google Patents

Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogénicos

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Publication number
AR126207A1
AR126207A1 ARP220101641A ARP220101641A AR126207A1 AR 126207 A1 AR126207 A1 AR 126207A1 AR P220101641 A ARP220101641 A AR P220101641A AR P220101641 A ARP220101641 A AR P220101641A AR 126207 A1 AR126207 A1 AR 126207A1
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Argentina
Prior art keywords
antisense strand
dsrna
strand
nucleotides
sense strand
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ARP220101641A
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English (en)
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Anastasia Khvorova
Sarah Davis
Annabelle Biscans
Ananth Karumanchi
Vignesh Narayan Hariharan
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Univ Massachusetts
Beth Israel Deaconess Medical Ct Inc
Vignesh Narayan Hariharan
Ananth Karumanchi
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Application filed by Univ Massachusetts, Beth Israel Deaconess Medical Ct Inc, Vignesh Narayan Hariharan, Ananth Karumanchi filed Critical Univ Massachusetts
Publication of AR126207A1 publication Critical patent/AR126207A1/es

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Abstract

Esta divulgación se refiere a objetivos novedosos para trastornos angiogénicos. También se proporcionan nuevos oligonucleótidos. También se proporcionan métodos para usar los nuevos oligonucleótidos para el tratamiento de trastornos angiogénicos (por ejemplo, preeclampsia). Reivindicación 1: Una molécula de ARN de doble cadena (ARNdc), comprendiendo dicho ARNdc una cadena antisentido y una cadena sentido, cada cadena con un extremo 5’ y un extremo 3’, en donde: (1) la cadena antisentido comprende una secuencia sustancialmente complementaria a una secuencia de ácido nucleico de 5’ CTCTCGGATCTCCAAATTTA 3’ (SEQ ID Nº 1) o 5’ CATCATAGCTACCATTTATT 3’ (SEQ ID Nº 2); (2) la cadena antisentido tiene una longitud de al menos 20 nucleótidos; (3) la cadena antisentido comprende al menos un 50% de modificaciones 2’-O-metilo; (4) los nucleótidos en cualquier una o más de las posiciones 2, 4, 5, 6, 8, 10, 12, 14, 16 y 20 desde el extremo 5’ de la cadena antisentido no son 2’-metoxi-ribonucleótidos; (5) los nucleótidos en las posiciones 1 - 2 a 1 - 8 desde el extremo 3’ de la cadena antisentido están conectados entre sí a través de enlaces internucleotídicos de fosforotioato; (6) una porción de la cadena antisentido es complementaria a una porción de la cadena sentido; (7) la cadena sentido tiene una longitud de al menos nucleótidos; (8) la cadena sentido comprende al menos 65% de modificaciones 2’-O-metilo; (9) los nucleótidos en cualquiera una o más de las posiciones 4, 6, 8, 10 y 14 desde el extremo 5’ de la cadena sentido no son 2’-metoxi-ribonucleótidos; y (10) los nucleótidos en las posiciones 1 - 2 desde el extremo 5’ de la cadena sentido están conectados entre sí a través de enlaces internucleotídicos de fosforotioato. Reivindicación 56: Una composición farmacéutica que comprende: un primer ARNdc que comprende una primera cadena sentido y una primera cadena antisentido, en donde la primera cadena antisentido comprende una región de complementariedad que es sustancialmente complementaria a SEQ ID Nº 1, en donde el primer ARNdc comprende el ARNdc de cualquiera de las reivindicaciones 1 - 46; un segundo ARNdc que comprende una segunda cadena sentido y una segunda cadena antisentido, en donde la segunda cadena antisentido comprende una región de complementariedad que es sustancialmente complementaria a SEQ ID Nº 2, en donde el segundo ARNdc comprende el ARNdc de cualquiera de las reivindicaciones 1 - 46; y un vehículo farmacéuticamente aceptable.
ARP220101641A 2021-06-23 2022-06-22 Compuestos de oligonucleotidos anti-flt1 optimizados para el tratamiento de la preeclampsia y otros desordenes angiogénicos AR126207A1 (es)

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US (2) US11702659B2 (es)
EP (1) EP4359539A1 (es)
KR (1) KR20240040729A (es)
CN (1) CN117677699A (es)
AR (1) AR126207A1 (es)
AU (1) AU2022299169A1 (es)
BR (1) BR112023026862A2 (es)
CA (1) CA3174095A1 (es)
IL (1) IL309334A (es)
TW (1) TW202315943A (es)
WO (1) WO2022271786A1 (es)

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CA2995110A1 (en) * 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
WO2017132669A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides

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