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1. WO2020097511 - MESSENGER RNA THERAPY FOR TREATMENT OF OCULAR DISEASES

Publication Number WO/2020/097511
Publication Date 14.05.2020
International Application No. PCT/US2019/060546
International Filing Date 08.11.2019
IPC
A61K 9/51 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
48Preparations in capsules, e.g. of gelatin, of chocolate
50Microcapsules
51Nanocapsules
C12N 15/11 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
11DNA or RNA fragments; Modified forms thereof
A61P 27/02 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
27Drugs for disorders of the senses
02Ophthalmic agents
CPC
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 48/0075
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 9/0048
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
0048Eye, e.g. artificial tears
A61K 9/0051
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
0048Eye, e.g. artificial tears
0051Ocular inserts, ocular implants
A61K 9/127
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
A61K 9/1271
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
Applicants
  • TRANSLATE BIO, INC. [US]/[US]
Inventors
  • ANDROSAVICH, John R.
  • DeROSA, Frank
  • KARVE, Shrirang
Agents
  • KIM, Sang-A
  • CONSALVI, Mary
  • BAUER, Steven
  • SHALEK, James
  • COUSINS, Abigail
Priority Data
62/758,10509.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MESSENGER RNA THERAPY FOR TREATMENT OF OCULAR DISEASES
(FR) THÉRAPIE PAR L'ARN MESSAGER POUR LE TRAITEMENT DES MALADIES OCULAIRES
Abstract
(EN)
The present invention provides, among other things, a method of ocular delivery of messenger RNA (mRNA), comprising administering into an eye of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administration of the composition results in expression of the protein encoded by the mRNA in the eye.
(FR)
Cette invention concerne, entre autres, un procédé d'administration oculaire d'ARN messager (ARNm), comprenant l'administration dans l'œil d'un sujet en ayant besoin d'une composition comprenant un ARNm codant pour une protéine, de façon que l'administration de la composition provoque l'expression de la protéine codée par l'ARNm dans l'œil.
Latest bibliographic data on file with the International Bureau