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1. WO2020100089 - COMPOSITIONS AND METHODS FOR THE TREATMENT OF SMOOTH MUSCLE DYSFUNCTION

Publication Number WO/2020/100089
Publication Date 22.05.2020
International Application No. PCT/IB2019/059805
International Filing Date 14.11.2019
IPC
A61K 38/17 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
17from animals; from humans
A61P 1/00 2006.01
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
A61P 9/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9Drugs for disorders of the cardiovascular system
A61P 11/06 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
06Antiasthmatics
A61P 13/02 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13Drugs for disorders of the urinary system
02of urine or of the urinary tract, e.g. urine acidifiers
A61P 13/08 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13Drugs for disorders of the urinary system
08of the prostate
Applicants
  • UROVANT SCIENCES GMBH [CH]/[CH]
Inventors
  • MELMAN, Arnold
Priority Data
62/767,45714.11.2018US
62/873,06411.07.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR THE TREATMENT OF SMOOTH MUSCLE DYSFUNCTION
(FR) COMPOSITIONS ET MÉTHODES POUR LE TRAITEMENT D'UN DYSFONCTIONNEMENT DES MUSCLES LISSES
Abstract
(EN)
The present disclosure provides compositions and methods to treat diseases and conditions related to smooth muscle dysfunction, or to ameliorate symptoms thereof comprising gene therapy, wherein one or more polynucleotides encoding one or more subunits of the Maxi-K channel, or mutants, variants, functional fragments, or derivatives thereof (e.g., fusions and chimaeras) are administered to a subject in need thereof, and wherein the administration of the polypeptides result in the expression of functional Maxi-K channels in the targeted smooth muscle. In some aspects, the composition of the disclosure comprise plasmid vectors containing at least one nucleic acid encoding a Maxi-K channel peptide. Also disclosed are pharmaceutical compositions, articles or manufacture, and kits.
(FR)
La présente invention concerne des compositions et des procédés pour traiter des maladies et des affections liées à un dysfonctionnement des muscles lisses, ou pour faire régresser les symptômes de celles-ci, comprenant une thérapie génique, un ou plusieurs polynucléotides codant pour une ou plusieurs sous-unités du canal Maxi-K, ou des mutants, des variants, des fragments fonctionnels ou des dérivés de ceux-ci (par exemple, des fusions et des chimères) étant administrés à un sujet qui en a besoin, et l'administration des polypeptides conduisant à l'expression de canaux Maxi-K fonctionnels dans le muscle lisse ciblé. Selon certains aspects, la composition de l'invention comprend des vecteurs plasmidiques contenant au moins un acide nucléique codant pour un peptide de canal Maxi-K. L'invention concerne également des compositions pharmaceutiques, des articles ou la fabrication, et des kits.
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