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1. WO2021041997 - CO-POTENTIATORS FOR THERAPY OF CYSTIC FIBROSIS CAUSED BY MINIMAL FUNCTION CFTR MUTANTS

Publication Number WO/2021/041997
Publication Date 04.03.2021
International Application No. PCT/US2020/048645
International Filing Date 28.08.2020
IPC
A61K 31/437 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
4353ortho- or peri-condensed with heterocyclic ring systems
437the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/4155 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
41having five-membered rings with two or more ring hetero atoms, at least one of which is nitrogen, e.g. tetrazole
4151,2-Diazoles
4155not condensed and containing further heterocyclic rings
A61P 11/00 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
A61K 31/4353 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
4353ortho- or peri-condensed with heterocyclic ring systems
A61K 31/47 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
A61K 31/4709 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
4709Non-condensed quinolines containing further heterocyclic rings
CPC
A61K 31/4245
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
41having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
4245Oxadiazoles
A61K 31/435
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
A61K 31/437
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
4353ortho- or peri-condensed with heterocyclic ring systems
437the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/47
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
A61K 31/4745
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
4738ortho- or peri-condensed with heterocyclic ring systems
4745condensed with ring systems having nitrogen as a ring hetero atom, e.g. phenantrolines
A61K 31/551
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
55having seven-membered rings, e.g. azelastine, pentylenetetrazole
551having two nitrogen atoms, e.g. dilazep
Applicants
  • THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US]/[US]
Inventors
  • VERKMAN, Alan S.
  • PHUAN, Puay-Wah
  • HAGGIE, Peter M.
Agents
  • HAN, Hai
  • OBEIDAT, Baha, A.
  • KUMABE, Blake, K.
  • SOLTANI, Bobby, B.
  • ZENTZ, Bradley, J.
  • SARGEANT, Brooke
  • QUIST, Brooke, W.
  • ROTH, Carol, J.
  • EIDT, Chandra, E.
  • WITTKOPP, Cristina, J.
  • O'BRIEN, Daniel
  • CARLSON, David, V.
  • CHEN, David W.
  • CONLEE, David C.
  • STARK, Duncan
  • TARLETON, E., Russell
  • SUN, Eileen, S.
  • HARWOOD, Eric, A.
  • GERTZ, Glenda, A.
  • TALBERT, Hayley, J.
  • BARRETT, Jared, M.
  • DANLEY, Jeffrey E.
  • SAKOI, Jeffrey, M.
  • SEO, Jeong Hee
  • BAUNACH, Jeremiah, J.
  • ZHANG, Jianping
  • MORGAN, John, A.
  • WAKELEY, John, J.
  • COE, Justin, E.
  • HENCKEL, Karen, M.
  • HEFTER, Karl, A.
  • HERMANNS, Karl, R.
  • MORGAN, Kevan, L.
  • COSTANZA, Kevin, S.
  • COOPER, Michael, P.
  • BENDEMIRE, Paul D.
  • LIN, Qing
  • HALLER, Rachel, A.
  • IANNUCCI, Robert
  • WEBB, Samuel, E.
  • LEEK, Shoko, I.
  • ROSENMAN, Stephen, J.
  • ABEDI, Syed
  • BOLLER, Timothy, L.
  • SHEWMAKE, Thomas, A.
  • CANTRELL, Tyler, C.
  • MIXCO, Javier, M.
  • LIU, Shi
  • LAWRENZ, Steven, D.
  • CLAYTON, Kenneth, W.
  • LI, Yiheng
  • SAKOI, Zachary, M.
Priority Data
62/893,10728.08.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CO-POTENTIATORS FOR THERAPY OF CYSTIC FIBROSIS CAUSED BY MINIMAL FUNCTION CFTR MUTANTS
(FR) CO-POTENTIALISATEURS POUR THÉRAPIE DE LA FIBROSE KYSTIQUE PROVOQUÉE PAR DES MUTANTS CFTR À FONCTION MINIMALE
Abstract
(EN)
Provided herein are combination-potentiator ("co-potentiator") therapeutic regimens, which can be used to modulate cystic fibrosis transmembrane conductance regulator (CTFR) mutant proteins. Co-potentiators have potential utility for treatment of many loss-of-function mutations of the CFTR chloride channel (e.g., N1303K).
(FR)
L'invention concerne des régimes de traitement de potentialisateur de combinaison (« co-potentialisateur »), qui peuvent être utilisés pour moduler des protéines mutantes de régulateur de conductance transmembranaire de fibrose kystique (CTFR). Les co-potentialisateurs ont une utilité potentielle pour le traitement de nombreuses mutations de perte de fonction du canal chlorure de CFTR (par exemple, N1303K).
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