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1. WO2022006332 - GENE THERAPY FOR IMMUNO-ONCOLOGY APPLICATIONS

Publication Number WO/2022/006332
Publication Date 06.01.2022
International Application No. PCT/US2021/039983
International Filing Date 30.06.2021
IPC
A61K 35/28 2015.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
A61K 35/15 2015.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
14Blood; Artificial blood
15Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cells; Myeloid precursor cells; Antigen-presenting cells, e.g. dendritic cells
A61K 38/21 2006.1
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
17from animals; from humans
19Cytokines; Lymphokines; Interferons
21Interferons
CPC
A61K 38/215
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
17from animals; from humans
19Cytokines; Lymphokines; Interferons
21Interferons ; [IFN]
215IFN-beta
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
A61K 9/0085
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
0085Brain, e.g. brain implants; Spinal cord
A61K 9/5184
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 ; having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
51Nanocapsules; Nanoparticles;
5107Excipients; Inactive ingredients
5176Compounds of unknown constitution, e.g. material from plants or animals
5184Virus capsids or envelopes enclosing drugs
A61P 35/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
C12N 15/00
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
Applicants
  • CHAN ZUCKERBERG BIOHUB, INC. [US]/[US]
  • THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US]/[US]
Inventors
  • PAULK, Nicole K.
  • DERISI, Joseph L.
  • RALEIGH, David R.
Agents
  • LOCKYER, Jean M.
  • APPLE, Randolph T.
Priority Data
63/046,21130.06.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) GENE THERAPY FOR IMMUNO-ONCOLOGY APPLICATIONS
(FR) THÉRAPIE GÉNIQUE POUR APPLICATIONS D'IMMUNO-ONCOLOGIE
Abstract
(EN) The invention provides compositions and methods for treatment of glioblastoma and other conditions. In particular, the invention provides a recombinant adeno-associated virus (AAV) vector comprising a transgene encoding one or more interferon polypeptides and a CAG promoter which directs the expression of the transgene. The compositions are particularly suitable for intratumoral administration in gene therapy applications.
(FR) L'invention concerne des compositions et des méthodes pour le traitement du glioblastome et d'autres pathologies. En particulier, l'invention concerne un vecteur du type virus adéno-associé (VAA) recombinant comprenant un transgène codant pour un ou plusieurs polypeptides d'interféron et un promoteur CAG qui dirige l'expression du transgène. Les compositions sont particulièrement appropriées pour une administration intratumorale dans des applications de thérapie génique.
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