Feedback
Settings

Settings

Goto Application

1. WO2020261178 - METHODS OF TREATING DUCHENNE MUSCULAR DYSTROPHY USING AAV MINI-DYSTROPHIN GENE THERAPY

Submit observation
PermaLink
Publication Number WO/2020/261178
Publication Date 30.12.2020
International Application No. PCT/IB2020/056029
International Filing Date 25.06.2020
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 14/47 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
C12Q 1/6851 2018.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6844Nucleic acid amplification reactions
6851Quantitative amplification
C12Q 1/70 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
70involving virus or bacteriophage
CPC
A01K 2217/075
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
2217Genetically modified animals
07Animals genetically altered by homologous recombination
075inducing loss of function, i.e. knock out
A01K 2227/10
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
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 2267/0306
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
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 48/0083
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
0083characterised by an aspect of the administration regime
Applicants
  • PFIZER INC. [US]/[US]
Inventors
  • BALDUS, Phoebe Arnold
  • BEIDLER, David Roger
  • BINKS, Michael
  • DEMARCO, Suzanne C.
  • HUANG, Rong
  • MCDONNELL MOOREHEAD, Tara
  • NEELAKANTAN, Srividya
  • NEUBERT, Hendrik
  • RUNNELS, Herbert
  • SANKAR, Savita
  • SHAPKINA, Tatiana G.
  • SHERLOCK, Sarah Paige
  • WHITELEY, Laurence Oliver
  • YONG, Florence Hiu-Ling
Agents
  • ZIELINSKI, Bryan C.
Priority Data
62/867,86227.06.2019US
63/016,23027.04.2020US
63/025,14114.05.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS OF TREATING DUCHENNE MUSCULAR DYSTROPHY USING AAV MINI-DYSTROPHIN GENE THERAPY
(FR) PROCÉDÉS DE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE À L'AIDE D'UNE THÉRAPIE GÉNIQUE BASÉE SUR L'AAV-MINI-DYSTROPHINE
front page image
Abstract
(EN)
The disclosure describes methods of treating humans with Duchenne muscular dystrophy by providing doses of an AAV9 vector that expresses a mini-dystrophin protein in transduced muscle cells.
(FR)
L'invention concerne des procédés de traitement d'êtres humains atteints d'une dystrophie musculaire de Duchenne par l'administration de doses d'un vecteur AAV9 qui exprime une protéine de mini-dystrophine dans des cellules musculaires transduites.
Latest bibliographic data on file with the International Bureau
# -