Research programme: Krabbe disease therapeutics - Gain Therapeutics
Alternative Names: GALC programme - Gain Therapeutics; Krabbe disease therapeutics - Gain TherapeuticsLatest Information Update: 18 Apr 2024
At a glance
- Originator Gain Therapeutics
- Class Neuroprotectants; Pharmacological chaperones; Small molecules
- Mechanism of Action Enzyme stimulants; Galactosylceramidase modulators
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Preclinical Globoid cell leukodystrophy
Most Recent Events
- 18 Apr 2024 Preclinical trials in Globoid cell leukodystrophy in USA (PO) (Gain Therapeutics Pipeline, April 2024)
- 15 Dec 2021 Gain Therapeutics plans to initiate animal proof of concept study in Globoid cell leukodystrophy in 2022
- 15 Dec 2021 Gain Therapeutics intends to file IND application for Globoid cell leukodystrophy in 2023