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SEL-148721

Development stage: Preclinical
Lead developer: Sharp Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

SEL-148721 is an orally available small molecule drug developed by Sharp Therapeutics for the treatment of Gaucher disease and potentially Parkinson disease. It acts as a modulator of beta-glucocerebrosidase (GBA1), aiming to restore or enhance the enzymatic activity of mutant GBA, which is deficient in patients with Gaucher disease. Unlike current enzyme replacement therapies that require regular infusions and may cause allergic reactions, this candidate offers a more convenient oral administration route. As of June 2025, it remains in preclinical development, with IND-enabling studies planned and Phase I clinical trials anticipated to begin in 2026[1][3].

Other names: SEL-148721SEL148721SEL 148721

Targets

GBA1 (Glucosylceramidase)

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