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1. WO2021252895 - COMPOSITIONS AND METHODS FOR THE TREATMENT AND PREVENTION OF NEUROLOGICAL DISORDERS

Publication Number WO/2021/252895
Publication Date 16.12.2021
International Application No. PCT/US2021/037008
International Filing Date 11.06.2021
IPC
C07D 239/00 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
239Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
C07D 239/48 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
239Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
02not condensed with other rings
24having three or more double bonds between ring members or between ring members and non-ring members
28with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
46Two or more oxygen, sulfur or nitrogen atoms
48Two nitrogen atoms
C07D 413/04 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
413Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
02containing two hetero rings
04directly linked by a ring-member-to-ring- member bond
CPC
A61K 31/5377
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
535having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
53751,4-Oxazines, e.g. morpholine
5377not condensed and containing further heterocyclic rings, e.g. timolol
Applicants
  • YUMANITY THERAPEUTICS, INC. [US]/[US]
Inventors
  • TARDIFF, Daniel
  • SCANNEVIN, Robert
  • RHODES, Kenneth
Agents
  • BELLIVEAU, Michael J.
Priority Data
63/038,05611.06.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR THE TREATMENT AND PREVENTION OF NEUROLOGICAL DISORDERS
(FR) COMPOSITIONS ET MÉTHODES DE TRAITEMENT ET DE PRÉVENTION DE TROUBLES NEUROLOGIQUES
Abstract
(EN) The invention provides compositions and methods for treating neurological disorders, such as amyotrophic lateral sclerosis, frontotemporal degeneration, and Alzheimer's disease, among others. Using the compositions and methods described herein, a patient having a neurological disorder, such as a neurological disorder associated with TAR-DNA binding protein (TDP)-43 aggregation, may be administered an inhibitor of FYVE-type zinc finger containing phosphoinositide kinase (PIKfyve) so as to treat an underlying etiology of the disorder and/or to alleviate one or more symptoms of the disease. The inhibitor of PIKfyve may be a small molecule, an anti-PIKfyve antibody or antigen-binding fragment thereof, or a compound, such as an interfering RNA molecule, that attenuates PIKfyve expression. Patients that may be treated using the compositions and methods described herein include those that express are susceptible to developing TDP-43- mediated aggregation and toxicity.
(FR) L'invention concerne des compositions et des méthodes de traitement de troubles neurologiques, tels que la sclérose latérale amyotrophique, la dégénérescence frontotemporale et la maladie d'Alzheimer, entre autres. Les compositions et les méthodes selon l'invention permettent à un patient souffrant d'un trouble neurologique, tel qu'un trouble neurologique associé à l'agrégation de la protéine de liaison TAR-ADN (TDP)-43, de recevoir un inhibiteur de doigt de zinc de type FYVE contenant une phosphoinositide kinase (PIKfyve), de façon à traiter une étiologie sous-jacente du trouble et/ou à atténuer un ou plusieurs symptômes de la maladie. L'inhibiteur de PIKfyve peut être une petite molécule, un anticorps anti-PIKfyve ou son fragment de liaison à l'antigène ou un composé, tel qu'une molécule d'ARN interférant, qui atténue l'expression de PIKfyve. Les patients qui peuvent être traités à l'aide des compositions et des méthodes selon l'invention comprennent les patients qui expriment ou sont susceptibles de développer une agrégation et une toxicité médiées par TDP-43.
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