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1. WO2020069461 - FRATAXIN EXPRESSION CONSTRUCTS HAVING ENGINEERED PROMOTERS AND METHODS OF USE THEREOF

Publication Number WO/2020/069461
Publication Date 02.04.2020
International Application No. PCT/US2019/053681
International Filing Date 27.09.2019
IPC
C07K 14/47 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
A61K 31/7088 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
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
CPC
A61K 47/02
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
02Inorganic compounds
A61K 47/10
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
06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
08containing oxygen, ; e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
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
A61K 48/0075
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
0075characterised by an aspect of the delivery route, e.g. oral, subcutaneous
A61K 9/0019
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
0012Galenical forms characterised by the site of application
0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
Applicants
  • VOYAGER THERAPEUTICS, INC. [US]/[US]
Inventors
  • PATZKE, Holger
  • HOU, Jinzhao
  • WANG, Hongxing
  • SHU, Yanqun
  • GOULET, Martin
  • SAH, Dinah Wen-Yee
Agents
  • WARD, Donna T.
  • HANSON, Mark T.
Priority Data
62/738,51928.09.2018US
62/901,76917.09.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) FRATAXIN EXPRESSION CONSTRUCTS HAVING ENGINEERED PROMOTERS AND METHODS OF USE THEREOF
(FR) CONSTRUCTIONS D'EXPRESSION DE FRATAXINE COMPRENANT DES PROMOTEURS MODIFIÉS ET LEURS MÉTHODES D'UTILISATION
Abstract
(EN)
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of frataxin (FXN), whether in vitro and/or in vivo including, but not limited to, the exploitation of engineered promoters. Such compositions include delivery via administration of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Friedreich's ataxia or another neuromuscular or neurological condition resulting from a deficiency in the quantity and/or function of frataxin or associated with decreased expression or protein levels of frataxin.
(FR)
L'invention concerne des compositions et des méthodes permettant de modifier, par exemple, d'améliorer, l'expression de la frataxine (FXN), que ce soit in vitro et/ou in vivo, comprenant sans caractère limitatif, l'exploitation de promoteurs modifiés. De telles compositions comprennent l'apport par l'intermédiaire de l'administration d'une particule virale adéno-associée (VAA). Les compositions et les méthodes selon la présente invention sont utiles dans le traitement de sujets chez qui on a diagnostiqué ou suspectés d'avoir une ataxie de Friedreich ou une autre affection neuromusculaire ou neurologique résultant d'une déficience de la quantité et/ou de la fonction de la frataxine ou associée à une expression réduite ou à des niveaux protéiques réduits de la frataxine.
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