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1. WO2021046169 - METHODS AND COMPOSITIONS FOR THE TREATMENT OF ALS

Publication Number WO/2021/046169
Publication Date 11.03.2021
International Application No. PCT/US2020/049123
International Filing Date 03.09.2020
IPC
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/475 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
475Growth factors; Growth regulators
C07K 14/71 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
71for growth factors; for growth regulators
C12N 15/86 2006.01
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
CPC
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/475
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
475Growth factors; Growth regulators
C07K 14/71
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
71for growth factors; for growth regulators
C12N 15/86
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
C12N 2750/14143
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
2750ssDNA Viruses
00011ssDNA Viruses
14011Parvoviridae
14111Dependovirus, e.g. adenoassociated viruses
14141Use of virus, viral particle or viral elements as a vector
14143viral genome or elements thereof as genetic vector
Applicants
  • UNIVERSITY OF CINCINNATI [US]/[US]
Inventors
  • MACLENNAN, Alexander John
Agents
  • LIVINGSTON, Jennifer L.
  • LORENTZ, Joshua A.
  • BONNER, Anthony F.
  • TENT, Brian A.
  • RERICHA, Nicholaus R.
  • OBERHAUS, Geoffrey L.
  • NEAL, Cynthia A.
  • DERBYSHIRE, Zachary
Priority Data
62/895,05203.09.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS AND COMPOSITIONS FOR THE TREATMENT OF ALS
(FR) MÉTHODES ET COMPOSITIONS POUR LE TRAITEMENT DE LA SCLÉROSE LATÉRALE AMYOTROPHIQUE
Abstract
(EN)
Compositions including modified adeno-associated virus (AAV) vectors comprising a recombinant AAV (rAAV)-based genome are provided herein, wherein the rAAV-based genome includes one or more of: a brain derived neurotrophic factor (BDNF)-encoding cDNA insert; or a neurotrophin-3 (NT-3)-encoding cDNA insert. Also provided are methods of treating motor neuron degenerative disorders, such as amyotrophic lateral sclerosis (ALS), by administering the disclosed compositions.
(FR)
Cette invention concerne des compositions comprenant des vecteurs de virus adéno-associé (AAV) modifié, comprenant un génome à base d'AAV recombinant (rAAV), le génome à base de rAAV comprenant un ou plusieurs des éléments suivants : un insert d'ADN complémentaire codant pour un facteur neurotrophique dérivé du cerveau (BDNF); ou un insert d'ADN complémentaire codant pour la neurotrophine-3 (NT-3). L'invention concerne également des procédés de traitement de troubles dégénératifs des neurones moteurs, telle que la sclérose latérale amyotrophique (SLA), par l'administration des compositions décrites.
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