Abstract: Provided herein are compounds that modulate glucocerebrosidase (GCase), an enzyme whose activity is associated with neurological diseases and disorders (e.g., Gaucher's disease, Parkinson's disease). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating GCase-related diseases and disorders (e.g., Gaucher's disease, Parkinson's disease) with the compounds in a subject, by administering the compounds and/or compositions described herein.
Abstract: Provided herein are compounds that modulate glucocerebrosidase (GCase), an enzyme whose activity is associated with neurological diseases and disorders (e.g., Gaucher's disease, Parkinson's disease). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating GCase-related diseases and disorders (e.g., Gaucher's disease, Parkinson's disease) with the compounds in a subject, by administering the compounds and/or compositions described herein.
Abstract: In some embodiments, the present disclosure provides compositions of antisense oligonucleotides (ASOs) such as gapmer ASOs for inhibition of complement C5 expression. Further, in some embodiments, such ASOs are useful for treating diseases associated with complement C5 dysfunction. Where a sequence is complementary to an intron of a complement 05 pre-mRNA.
Type:
Application
Filed:
October 12, 2022
Publication date:
December 19, 2024
Applicant:
Vanqua Bio, Inc.
Inventors:
Kevin Hunt, Maria Nguyen, Daniel Ysselstein, Carole Deyts
Abstract: Provided herein are compounds that modulate glucocerebrosidase (GCase), an enzyme whose activity is associated with neurological diseases and disorders (e.g., Gaucher's disease. Parkinson's disease). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating GCase-related diseases and disorders (e.g., Gaucher's disease. Parkinson's disease) with the compounds in a subject, by administering the compounds and/or compositions described herein.
Abstract: Provided herein are compounds that modulate glucocerebrosidase (GCase), an enzyme whose activity is associated with neurological diseases and disorders (e.g., Gaucher's disease, Parkinson's disease). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating GCase-related diseases and disorders (e.g., Gaucher's disease, Parkinson's disease) with the compounds in a subject, by administering the compounds and/or compositions described herein.