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1. WO2022107853 - LOW-DOSE HEPATOCYTE GROWTH FACTOR GENE THERAPY FOR DIABETES

Publication Number WO/2022/107853
Publication Date 27.05.2022
International Application No. PCT/JP2021/042463
International Filing Date 18.11.2021
IPC
A61K 48/00 2006.1
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
A61K 35/76 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
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
A61K 35/761 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
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
761Adenovirus
A61K 38/22 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
22Hormones
A61P 1/18 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
1Drugs for disorders of the alimentary tract or the digestive system
18for pancreatic disorders, e.g. pancreatic enzymes
A61P 3/10 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
08for glucose homeostasis
10for hyperglycaemia, e.g. antidiabetics
CPC
A61K 35/76
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
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
A61K 35/761
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
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
761Adenovirus
A61K 38/22
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
22Hormones
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
A61P 1/18
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
1Drugs for disorders of the alimentary tract or the digestive system
18for pancreatic disorders, e.g. pancreatic enzymes
A61P 3/10
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
08for glucose homeostasis
10for hyperglycaemia, e.g. antidiabetics
Applicants
  • 国立大学法人 鹿児島大学 KAGOSHIMA UNIVERSITY [JP]/[JP]
Inventors
  • 小戝 健一郎 KOSAI, Ken-ichiro
  • 松田 恵理子 MATSUDA, Eriko
Agents
  • 高島 一 TAKASHIMA, Hajime
Priority Data
2020-19284419.11.2020JP
2021-14579507.09.2021JP
Publication Language Japanese (ja)
Filing Language Japanese (JA)
Designated States
Title
(EN) LOW-DOSE HEPATOCYTE GROWTH FACTOR GENE THERAPY FOR DIABETES
(FR) THÉRAPIE GÉNIQUE À FAIBLE DOSE PAR FACTEUR DE CROISSANCE DES HÉPATOCYTES CONTRE LE DIABÈTE
(JA) 糖尿病に対する低用量の肝細胞増殖因子遺伝子治療
Abstract
(EN) The present invention provides an agent for protecting and regenerating pancreatic beta cells in a mammal having diabetes, the agent containing a recombinant viral vector that expresses hepatocyte growth factor (HGF), wherein the agent is characterized by being administered in a dose of 1010-1012 virus particles (vp)/kg body weight, and by the virus vector including a nucleic acid that encodes HGF downstream of a promoter having transcription activity capable of yielding a therapeutically effective HGF level in the blood at said dose.
(FR) La présente invention concerne un agent de protection et de régénération des cellules bêta pancréatiques chez un mammifère atteint de diabète, l'agent contenant un vecteur viral recombinant qui exprime le facteur de croissance des hépatocytes (HGF), l'agent étant caractérisé en ce qu'il est administré en dose de 10 10-1012 particules de virus (vp)/kg de poids corporel, et en ce que le vecteur viral comprend un acide nucléique qui code pour HGF en aval d'un promoteur ayant une activité de transcription pouvant produire un taux de HGF thérapeutiquement efficace dans le sang à ladite dose.
(JA) 本発明は、肝細胞増殖因子(HGF)を発現する組換えウイルスベクターを含有してなる、糖尿病を有する哺乳動物における膵β細胞の保護・再生剤であって、1010~1012ウイルス粒子(vp)/kg体重の用量で投与され、かつ該ウイルスベクターが、当該用量において、治療上有効な血中HGFレベルを与え得る転写活性を有するプロモーターの下流にHGFをコードする核酸を含むことを特徴とする、剤を提供する。
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