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1. WO2022006436 - COMPOSITIONS AND METHODS FOR CELLULAR DELIVERY OF RNA

Publication Number WO/2022/006436
Publication Date 06.01.2022
International Application No. PCT/US2021/040152
International Filing Date 01.07.2021
IPC
A61K 38/17 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
A61K 31/714 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7135Compounds containing heavy metals
714Cobalamins, e.g. cyanocobalamin, vitamin B12
C12N 15/113 2010.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Applicants
  • BIOAFFINITY TECHNOLOGIES, INC. [US]/[US]
Inventors
  • BAUTA, William E.
  • ELZI, David J.
  • REBEL, Vivienne I.
Agents
  • VILVEN, Janeen
Priority Data
63/047,02401.07.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR CELLULAR DELIVERY OF RNA
(FR) COMPOSITIONS ET MÉTHODES D'ADMINISTRATION CELLULAIRE D'ARN
Abstract
(EN) One embodiment of the present invention provides for a cobalamin complex or the pharmaceutically acceptable salt, stereoisomers, solvate or polymorph thereof comprising a siRNA sequence that is complementary to a nucleic acid sequence that codes for a target protein within a cell wherein the siRNA sequence is bound to a cobalamin and wherein the cobalamin is complexed with a cobalamin binding protein, a pharmaceutical composition formed with the complex and a method of using the complex to treat a disease associated with the target protein.
(FR) Un mode de réalisation de la présente invention concerne un complexe de cobalamine ou le sel pharmaceutiquement acceptable, les stéréoisomères, le solvate ou son polymorphe comprenant une séquence d'ARNsi complémentaire d'une séquence d'acide nucléique qui code pour une protéine cible à l'intérieur d'une cellule, la séquence d'ARNsi étant liée à une cobalamine et la cobalamine étant complexée avec une protéine de liaison à la cobalamine, une composition pharmaceutique formée avec le complexe et une méthode d'utilisation du complexe pour traiter une maladie associée à la protéine cible.
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