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1. WO2022055351 - CONJUGATE OF SAPONIN, OLIGONUCLEOTIDE AND GALNAC

Publication Number WO/2022/055351
Publication Date 17.03.2022
International Application No. PCT/NL2021/050549
International Filing Date 09.09.2021
Chapter 2 Demand Filed 10.05.2022
IPC
A61K 47/54 2017.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
A61K 47/55 2017.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
55the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
A61P 35/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
CPC
A61K 47/54
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
A61K 47/55
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51the non-active ingredient being a modifying agent
54the modifying agent being an organic compound
55the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
Applicants
  • SAPREME TECHNOLOGIES B.V. [NL]/[NL]
Inventors
  • HERMANS, Guy
  • POSTEL, Ruben
  • VAN DE LANGEMHEEN, Helmus
  • ASADIAN BIRJAND, Mazdak
Agents
  • NEDERLANDSCH OCTROOIBUREAU
Priority Data
202644210.09.2020NL
202740526.01.2021NL
PCT/NL2021/05038418.06.2021NL
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) CONJUGATE OF SAPONIN, OLIGONUCLEOTIDE AND GALNAC
(FR) CONJUGUÉ DE SAPONINE, D'OLIGONUCLÉOTIDE ET DE GALNAC
Abstract
(EN) The invention relates to a conjugate comprising a saponin covalently linked to a ligand for ASGPR, the ligand comprising at least one GalNAc moiety, and comprising an oligonucleotide covalently linked to the saponin and the ligand for ASGPR. In addition, the invention relates to a pharmaceutical composition comprising the conjugate of the invention. Furthermore, the invention relates to a pharmaceutical composition of the invention, for use as a medicament. The invention also relates to a pharmaceutical composition of the invention, for use in the treatment or prophylaxis of a disease or health problem in which an expression product is involved of for example genes: apoB, HSP17, TTR, PCSK9, ALAS1, AT3, GO, CC5, X gene of HBV, S gene of HBV, AAT and LDH, and for use in the treatment or prophylaxis of for example a cancer, an infectious disease, a viral infection, hypercholesterolemia, primary hyperoxaluria, haemophilia A, haemophilia B, AAT related liver disease, acute hepatic porphyria, TTR amyloidosis, complement-mediated disease, hepatitis B infection, or an auto-immune disease. The invention also relates to a method for producing an oligonucleotide conjugate of the invention. Finally, the invention relates to an in vitro or ex vivo method for transferring the conjugate of the invention from outside a cell to inside said cell.
(FR) L'invention concerne un conjugué comprenant une saponine liée de manière covalente à un ligand pour ASGPR, le ligand comprenant au moins une fraction GalNAc, et comprenant un oligonucléotide lié de manière covalente à la saponine et au ligand pour ASGPR. L'invention concerne également une composition pharmaceutique comprenant le conjugué selon l'invention. En outre, l'invention concerne une composition pharmaceutique selon l'invention, destinée à être utilisée en tant que médicament. L'invention concerne également une composition pharmaceutique selon l'invention, pour une utilisation dans le traitement ou la prophylaxie d'une maladie ou d'un problème de santé dans lequel est mis en cause un produit d'expression des gènes, par exemple : apoB, HSP17, TTR, PCSK9, ALAS1, AT3, GO, CC5, le gène X du VHB, le gène S du VHB, AAT et LDH, et pour une utilisation dans le traitement ou la prophylaxie, par exemple d'un cancer, d'une maladie infectieuse, d'une infection virale, d'une hypercholestérolémie, de l'hyperoxalurie primaire, de l'hémophilie A, de l'hémophilie B, d'une maladie du foie liée à l'AAT, de la porphyrie hépatique aiguë, d'une amyloïdose de TTR, d'une maladie médiée par le complément, d'une infection par l'hépatite B ou d'une maladie auto-immune. La présente invention concerne en outre un procédé de production d'un conjugué d'oligonucléotide selon l'invention. L'invention concerne enfin un procédé in vitro ou ex vivo permettant de transférer le conjugué selon l'invention de l'extérieur d'une cellule vers l'intérieur de ladite cellule.
Latest bibliographic data on file with the International Bureau