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1. WO2020132659 - GENETIC MODIFICATION OF THE HYDROXYACID OXIDASE 1 GENE FOR TREATMENT OF PRIMARY HYPEROXALURIA

Publication Number WO/2020/132659
Publication Date 25.06.2020
International Application No. PCT/US2019/068186
International Filing Date 20.12.2019
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 38/46 2006.01
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)
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
C12N 9/22 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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
C12N 15/10 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
10Processes for the isolation, preparation or purification of DNA or RNA
C12N 9/04 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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
02Oxidoreductases (1.), e.g. luciferase
04acting on CHOH groups as donors, e.g. glucose oxidase, lactate dehydrogenase (1.1)
Applicants
  • PRECISION BIOSCIENCES, INC. [US]/[US]
Inventors
  • DAVEY, Roshni
  • JANTZ, Derek
  • SMITH, James Jefferson
  • OWENS, Gary
Agents
  • GREENWALD, Eric, P.
  • BAKER, C., Hunter
  • ALAM, Saad
  • AMUNDSEN, Eric, L.
  • ATTISHA, Michael, J.
Priority Data
62/783,96921.12.2018US
62/833,57412.04.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) GENETIC MODIFICATION OF THE HYDROXYACID OXIDASE 1 GENE FOR TREATMENT OF PRIMARY HYPEROXALURIA
(FR) MODIFICATION GÉNÉTIQUE DU GÈNE DE L'HYDROXYACIDE OXYDASE 1 POUR LE TRAITEMENT DE L'HYPEROXALURIE PRIMAIRE
Abstract
(EN)
Disclosed are engineered nucleases that bind and cleave a recognition sequence within a hydroxyacid oxidase 1 (HAO1 ) gene. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells. Further, the invention encompasses pharmaceutical compositions comprising engineered nuclease proteins or nucleic acids encoding engineered nucleases of the invention, and the use of such compositions for treatment of primary hyperoxaluria type I.
(FR)
L'invention concerne des nucléases modifiées qui se lient à une séquence de reconnaissance et clivent une séquence de reconnaissance au sein d'un gène d'hydroxyacide oxydase 1 (HAO1). La présente invention concerne également des procédés d'utilisation de ces nucléases modifiées pour fabriquer des cellules génétiquement modifiées. En outre, l'invention concerne des compositions pharmaceutiques comprenant des protéines nucléases modifiées ou des acides nucléiques codant pour des nucléases modifiées selon l'invention et l'utilisation de telles compositions pour le traitement de l'hyperoxalurie primaire de type I.
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