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DLX313

Development stage: Unknown
Lead developer: Novartis
Modality: Small Molecules
Administration: Oral

Overview

DLX313 is an investigational small molecule glucocerebrosidase (GBA) activator developed by Novartis for the treatment of Parkinson's disease. Mutations in the GBA1 gene, which result in reduced GBA enzyme activity, are a significant genetic risk factor for Parkinson's and are associated with the accumulation of alpha-synuclein aggregates. DLX313 is designed to enhance the activity of the GBA enzyme, thereby improving lysosomal function and promoting the clearance of toxic alpha-synuclein species. The drug is currently being evaluated in Phase 1 clinical trials to assess its safety, tolerability, and pharmacokinetics in both healthy volunteers and patients with Parkinson's disease.

Targets

GBA1 (Glucosylceramidase)

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