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SNCA-TARGETING SIRNA COMPOSITIONS FOR TREATING SNCA-ASSOCIATED DISEASE

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SNCA-TARGETING SIRNA COMPOSITIONS FOR TREATING SNCA-ASSOCIATED DISEASE

PCT NATIONAL PHASE APPLICATION

Published

date

Filed on 1 November 2024

Abstract

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SNCA gene, particularly in a CNS tissue, as well as methods of inhibiting expression of a SNCA gene and methods of treating subjects having a SNCA-associated neurodegenerative disease or disorder, e.g., Parkinson's Disease (PD), multiple system atrophy (MSA), Lewy body dementia (LBD), among other synucleinopathies, using such dsRNAi agents and compositions.

Patent Information

Application ID202447083688
Invention FieldBIOTECHNOLOGY
Date of Application01/11/2024
Publication Number45/2024

Inventors

NameAddressCountryNationality
HOANG DANG, Lan Thi675 West Kendall Street Henri A. Termeer Square Cambridge, Massachusetts 02142U.S.A.U.S.A.
SCHLEGEL, Mark K.675 West Kendall Street Henri A. Termeer Square Cambridge, Massachusetts 02142U.S.A.U.S.A.
BARRY, Joseph675 West Kendall Street Henri A. Termeer Square Cambridge, Massachusetts 02142U.S.A.U.S.A.
NGUYEN, Tuyen675 West Kendall Street Henri A. Termeer Square Cambridge, Massachusetts 02142U.S.A.U.S.A.
CASTORENO, Adam675 West Kendall Street Henri A. Termeer Square Cambridge, Massachusetts 02142U.S.A.U.S.A.
SO, Kawai675 West Kendall Street Henri A. Termeer Square Cambridge, Massachusetts 02142U.S.A.U.S.A.

Applicants

NameAddressCountryNationality
ALNYLAM PHARMACEUTICALS, INC.675 West Kendall Street Henri A. Termeer Square Cambridge, Massachusetts 02142U.S.A.U.S.A.

Documents

NameDate
202447083688-CLAIMS UNDER RULE 1 (PROVISIO) OF RULE 20 [01-11-2024(online)].pdf01/11/2024
202447083688-COMPLETE SPECIFICATION [01-11-2024(online)].pdf01/11/2024
202447083688-DECLARATION OF INVENTORSHIP (FORM 5) [01-11-2024(online)].pdf01/11/2024
202447083688-DRAWINGS [01-11-2024(online)].pdf01/11/2024
202447083688-FIGURE OF ABSTRACT [01-11-2024(online)].pdf01/11/2024
202447083688-FORM 1 [01-11-2024(online)].pdf01/11/2024
202447083688-FORM 18 [01-11-2024(online)].pdf01/11/2024
202447083688-POWER OF AUTHORITY [01-11-2024(online)].pdf01/11/2024
202447083688-Sequence Listing in PDF [01-11-2024(online)].pdf01/11/2024
202447083688-Sequence Listing in txt [01-11-2024(online)].txt01/11/2024

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