Drug intelligence / Profile preview

trehalose

Development stage
Phase 4
Lead developer
Seelos Therapeutics
Modality
Drug Delivery Systems, DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Intravenous, Oral, Subcutaneous, Ophthalmic, Intramuscular
01

Overview

Trehalose is a naturally occurring non-reducing disaccharide composed of two glucose molecules linked by an α,α-1,1 glycosidic bond. It is widely found in nature and is known for its ability to stabilize proteins, lipids, and carbohydrates during freeze-thaw or dehydration-rehydration cycles[1][3][5]. In the pharmaceutical industry, trehalose is primarily used as an excipient to enhance the stability of biologics such as monoclonal antibodies, antibody-drug conjugates, fusion proteins, peptides, stem cells, and vaccines[3][5]. Trehalose has also been investigated for its neuroprotective properties in models of neurodegenerative diseases like Parkinson's and Huntington's disease. Its proposed mechanisms include modulation of autophagy (potentially promoting clearance of protein aggregates), stabilization of proteins to prevent misfolding and aggregation, reduction of oxidative stress-induced neuronal damage by stabilizing cellular membranes and modulating mitochondrial function[6][8]. Trehalose has been studied under the code name "Cabaletta" for oculopharyngeal muscular dystrophy[2]. It is approved as a food ingredient in all major markets and included in several commercial therapeutic formulations as a stabilizer/excipient[4].

Brand names
Cabaletta
Other names
trehaloseCabaletta
02

Targets

LpqY-SugABC (LpqY-SugABC trehalose transporter)

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