WO2021150969 LEUCINE-RICH REPEAT KINASE 2 (LRRK2) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF

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Publication Number WO/2021/150969

Publication Date 29.07.2021

International Application No. PCT/US2021/014729

International Filing Date 22.01.2021

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Applicants

ALNYLAM PHARMACEUTICALS, INC. [US]/[US]

Inventors

MCININCH, James D.
GILBERT, Jason
CASTORENO, Adam
DANG, Lan Thi Hoang
LEBLANC, Sarah
PENG, Haiyan
KAITTANIS, Charalambos
SOUNDARAPANDIAN, Mangala Meenakshi

Agents

MEIGS, Julie Broadus
SEETHARAMAN, Sai Venkatesh
RAY, Denise M.
DONAHOE, Casey D.
OKADA, Seiko

Priority Data

62/965,452		24.01.2020		US
63/138,717		18.01.2021		US

Publication Language English (en)

Filing Language English (EN)

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Title

(EN) LEUCINE-RICH REPEAT KINASE 2 (LRRK2) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF

(FR) COMPOSITIONS D'AGENT D'ARNI À KINASE 2 À RÉPÉTITION RICHE EN LEUCINE (LRRK2) ET UTILISATION ASSOCIÉE

Abstract

(EN) The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a leucine-rich repeat kinase 2 (LRRK2) gene, as well as methods of inhibiting expression of a LRRK2 gene and methods of treating subjects having a LRRK2-associated disease or disorder, e.g., Parkinson's disease, using such dsRNAi agents and compositions.

(FR) L'invention concerne des agents et des compositions à base d'acide ribonucléique à double brins ciblant un gène de kinase 2 à répétition riche en leucine (LRRK2), ainsi que des procédés d'inhibition de l'expression d'un gène LRRK2 et des procédés de traitement de sujets ayant une maladie ou un trouble associé à LRRK2, par exemple la maladie de Parkinson, à l'aide de tels agents et compositions à base d'ARNi double brin.

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C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
11	DNA or RNA fragments; Modified forms thereof
113	Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31	Medicinal preparations containing organic active ingredients
70	Carbohydrates; Sugars; Derivatives thereof
7088	Compounds having three or more nucleosides or nucleotides
712	Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31	Medicinal preparations containing organic active ingredients
70	Carbohydrates; Sugars; Derivatives thereof
7088	Compounds having three or more nucleosides or nucleotides
7125	Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48	Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
11	DNA or RNA fragments; Modified forms thereof
113	Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

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1. WO2021150969 - LEUCINE-RICH REPEAT KINASE 2 (LRRK2) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
P	SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25	Drugs for disorders of the nervous system
28	for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47	Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50	the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51	the non-active ingredient being a modifying agent
54	the modifying agent being an organic compound
549	Sugars, nucleosides, nucleotides or nucleic acids

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47	Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50	the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51	the non-active ingredient being a modifying agent
54	the modifying agent being an organic compound
554	the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47	Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50	the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51	the non-active ingredient being a modifying agent
56	the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47	Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50	the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
51	the non-active ingredient being a modifying agent
54	the modifying agent being an organic compound
549	Sugars, nucleosides, nucleotides or nucleic acids