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1. WO2022119974 - RNA-TARGETING COMPOSITIONS AND METHODS FOR TREATING CAG REPEAT DISEASES

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Publication Number WO/2022/119974
Publication Date 09.06.2022
International Application No. PCT/US2021/061482
International Filing Date 01.12.2021
IPC
A61K 38/46 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
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
A61P 21/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
CPC
A01K 2227/105
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2227Animals characterised by species
10Mammal
105Murine
A01K 2267/0318
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2267Animals characterised by purpose
03Animal model, e.g. for test or diseases
0306Animal model for genetic diseases
0318Animal model for neurodegenerative disease, e.g. non- Alzheimer's
A61K 48/005
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
A61K 48/0058
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
A61P 21/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
A61P 25/14
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
14for treating abnormal movements, e.g. chorea, dyskinesia
Applicants
  • LOCANABIO, INC. [US]/[US]
Inventors
  • NELLES, David A.
  • BATRA, Ranjan
  • ROTH, Daniela
  • ZISOULIS, Dimitrios
  • TA, Angeline
Agents
  • ELRIFI, Ivor
  • BROWN, Fraser
  • HENDERSON, Andrew
Priority Data
63/119,97701.12.2020US
63/130,06023.12.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) RNA-TARGETING COMPOSITIONS AND METHODS FOR TREATING CAG REPEAT DISEASES
(FR) COMPOSITIONS DE CIBLAGE D'ARN ET PROCÉDÉS DE TRAITEMENT DE MALADIES À RÉPÉTITION CAG
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Abstract
(EN) Disclosed are RNA-targeting gene therapy compositions and methods for destroying or blocking toxic target CAG repeat RNA and treating CAG repeat disorders such as Huntington's Disease (HD) and Spinocerebellar Ataxia Type 1 (SCA1).
(FR) L'invention concerne des compositions de thérapie génique ciblant l'ARN et des procédés de destruction ou de blocage d'ARN de répétition CAG cible toxique et de traitement de troubles de répétition CAG tels que la maladie de Huntington (HD) et l'ataxie spinocérébelleuse de type 1 (SCA1).
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