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1. WO2020068862 - COMPOSITIONS AND METHODS FOR THE TREATMENT OF NETHERTON SYNDROME

Publication Number WO/2020/068862
Publication Date 02.04.2020
International Application No. PCT/US2019/052779
International Filing Date 24.09.2019
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 38/16 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
CPC
A61K 35/763
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
763Herpes virus
A61K 38/57
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
55Protease inhibitors
57from animals; from humans
A61K 9/0014
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
0014Skin, i.e. galenical aspects of topical compositions
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
A61P 17/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
17Drugs for dermatological disorders
C07K 14/4703
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
4701not used
4702Regulators; Modulating activity
4703Inhibitors; Suppressors
Applicants
  • KRYSTAL BIOTECH, INC. [US]/[US]
Inventors
  • AGARWAL, Pooja
  • KRISHNAN, Suma
  • FREEDMAN, John C.
Agents
  • YIN, Bu
  • MULLEN III, James J.
  • MURASHIGE, Kate H.
  • POTTER, Karen
  • AHN, Sejin
  • CAO, Yuchen
  • O'SULLIVAN, Desmond
  • RICE, Rachel
  • SCHMIDT, Jeffrey W.
Priority Data
62/735,58224.09.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR THE TREATMENT OF NETHERTON SYNDROME
(FR) COMPOSITIONS ET MÉTHODES DE TRAITEMENT DU SYNDROME DE NETHERTON
Abstract
(EN)
The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a Serine Protease Inhibitor Kazal-type (SPINK) polypeptide (e.g., a SPINKS polypeptide); viruses comprising the recombinant nucleic acids; compositions and formulations comprising the recombinant nucleic acids and/or viruses; methods of their use (e.g., for the treatment of Netherton Syndrome); and articles of manufacture or kits thereof.
(FR)
La présente invention concerne des acides nucléiques de recombinaison comprenant un ou plusieurs polynucléotides codant pour un polypeptide de type inhibiteur Kazal de la sérine protéase (SPINK) (par exemple, un polypeptide SPINKS) ; des virus comprenant les acides nucléiques de recombinaison ; des compositions et des formulations comprenant les acides nucléiques de recombinaison et/ou les virus ; des procédés d'utilisation de ceux-ci (par exemple, pour le traitement du syndrome de Netherton) ; ainsi que des articles manufacturés ou des kits associés.
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