Abstract: The application is directed to compounds of formula (I) and their salts and solvates, wherein B, R1, R2, R3, R3’, R4, R4’ and R5 are as set forth in the specification, as well as to methods for their preparation, pharmaceutical compositions comprising the same, and use thereof for the treatment and/or prevention of, e.g., MPS1, optionally in combination with ?-L-iduronidase or an analog or variant thereof, e.g., laronidase.
Type:
Application
Filed:
February 3, 2021
Publication date:
May 4, 2023
Applicant:
GAIN THERAPEUTICS SA
Inventors:
Ana Maria GARCÍA COLLAZO, Elena CUBERO JORDÀ, Xavier BARRIL ALONSO, Roberto MAJ, Natalia PEREZ CARMONA
Abstract: The application is directed to compounds of formulae (IA) and (IB) and their salts and solvates, wherein R1a, R2a, R3a, A1, A2, A3, R1b, R2b, R3b, B1, and B2 are as set forth in the specification, as well as to methods for their preparation, pharmaceutical compositions comprising the same, and use thereof for the treatment and/or prevention of, e.g., lysosomal storage diseases, such as Gaucher's disease, and ?-synucleinopathies, such as Parkinson's disease.
Type:
Application
Filed:
November 25, 2020
Publication date:
April 6, 2023
Applicant:
GAIN THERAPEUTICS SA
Inventors:
Ana Maria GARCÍA COLLAZO, Elena CUBERO JORDÀ, Manolo BELLOTTO, Enrique FERNÁNDEZ IGLESIAS
Abstract: The application is directed to compounds of formulae (IA) and (IB): (IA) and (IB), and their salts and solvates, wherein R1a, R2a, A1, A2, A3, A4, R1b, R2b, B1, B2, B3, and G are as set forth in the specification, as well as to methods for their preparation, N pharmaceutical compositions comprising the same, and use thereof for the treatment and/or prevention of, e.g., lysosomal storage diseases, such as Krabbe's disease, and ?-synucleinopathies, such as Parkinson's disease.
Type:
Application
Filed:
November 25, 2020
Publication date:
February 23, 2023
Applicant:
GAIN THERAPEUTICS SA
Inventors:
Ana Maria GARCÍA COLLAZO, Elena CUBERO JORDÀ, Xavier BARRIL ALONSO, Manolo BELLOTTO