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1. WO2021050991 - COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA

Publication Number WO/2021/050991
Publication Date 18.03.2021
International Application No. PCT/US2020/050551
International Filing Date 11.09.2020
IPC
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
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
CPC
A61K 31/7088
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
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 14/47
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
C07K 2319/40
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
40containing a tag for immunodetection, or an epitope for immunisation
Applicants
  • LACERTA THERAPEUTICS, INC [US]/[US]
  • FALK, Darin [US]/[US]
Inventors
  • FALK, Darin
  • RODRIGUEZ, Edgardo
  • SAHA, Madhurima
Agents
  • FERNANDEZ, Lizette M.
  • GRAHAM, Jamie L.
Priority Data
62/899,95313.09.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA
(FR) COMPOSITIONS ET MÉTHODES POUR LE TRAITEMENT DE L'ATAXIE DE FRIEDREICH
Abstract
(EN)
The present application provides compositions for treatment of Friedreich's Ataxia (FA). These include, but are not limited to, nucleic acid constructs and recombinant vectors comprising a human frataxin 5' untranslated region (5'UTR FXN) and a human frataxin (FXN) nucleotide sequence are provided herein. Also provided are methods for treatment of FA.
(FR)
La présente invention concerne des compositions pour le traitement de l'ataxie de Friedreich (FA). Celles-ci comprennent, sans caractère limitatif, des constructions d'acide nucléique et des vecteurs recombinés comprenant une région 5' non traduite de la frataxine humaine (FXN à 5'UTR) et une séquence nucléotidique de la frataxine humaine (FXN). La présente invention concerne également des méthodes de traitement de la FA.
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