β-glucocerebrosidase

Compound 28, also known as β-Glucocerebrosidase modulator 1, is an allosteric modulator of β-Glucocerebrosidase with an EC50 of 9.0 μM and a Kd of 0.050 μM.

Glucocerebrosidase (Glucosylceramidase; GBA), a lysosomal enzyme, catalyzes the hydrolysis of the β-glucosidic linkage in glucocerebroside (GC) to yield glucose and ceramide [1].

Glucocerebrosidase-IN-1 (compound 11a) is a potent and selective GCase (glucocerebrosidase) inhibitor, with an IC50 of 29.3 μM and a Ki of 18.5 μM. Due to GCase involvement in pathological disorders consequent to enzyme deficiency, Glucocerebrosidase-IN-1 can be used for research of Gaucher disease (GD) and Parkinson's disease (PD)[1].

Glucocerebrosidase-IN-1 (compound 11a) hydrochloride is a potent and selective glucocerebrosidase (GCase) inhibitor with IC50 of 29.3 μM and Ki of 18.5 μM, used in the research of Gaucher disease (GD) and Parkinson's disease (PD) [1].

NCGC00092410 (ML008) is a glucocerebrosidase (GC) inhibitor with IC50 value of 31 nM. It can be used for the study of Gaucher disease caused by mutation of glucocerebrase gene.

Ambroxol (NA-872) is a secretolytic agent used in the treatment of respiratory diseases associated with viscid or excessive mucus. It is the active ingredient of Mucosolvan, Lasolvan or Mucoangin. The substance is a mucoactive drug with several properties including secretolytic and secretomotoric actions that restore the physiological clearance mechanisms of the respiratory tract which play an important role in the body's natural defense mechanisms. It stimulates synthesis and release of surfactant by type II pneumocytes. Surfactants act as an anti-glue factor by reducing the adhesion of mucus to the bronchial wall, in improving its transport and in providing protection against infection and irritating agents.

Ambroxol hydrochloride (Mucosolvan) is a metabolite of BROMHEXINE that stimulates mucociliary action and clears the air passages in the respiratory tract. It is usually administered as the hydrochloride.

WAY-640509 is a glucocerebrosidase inhibitor.

ML198 is a novel activator and non-inhibitory chaperone of glucocerebrosidase. ML198 serves as a starting point to develop a novel approach towards small molecule treatment for patients suffering from Gaucher disease.

ML266, a glucocerebrosidase (GCase) chaperone with an IC50 of 2.5 µM, facilitates the transport of the mutant protein to the lysosome, thereby restoring GCase activity without inhibiting the enzyme's function. This compound shows promise for Gaucher disease research.

Conduritol B epoxide is an irreversible inhibitor of β-glucosidase (GCase).

Gcase activator 2 is a β-glucocerebrosidase activator that induces dimerization of GCase at the endoplasmic reticulum (ER) and lysosomal pHs, increases lysosomal substrate metabolism, and has therapeutic potential for the treatment of Parkinson's and Gosheimer's diseases.

N-Boc-3-hydroxy-1,2,3,6-tetrahydropyridine enhances β-glucocerebrosidase (β-Glucocerebrosidase) activity by 20%. It is used in research on lysosomal storage disorders and other protein accumulation diseases.

13C6 Glucosylsphingosine (d18:1) is an isotopically enriched form of 1-β-D-glucosylsphingosine (d18:1) that is intended for use as an internal standard for the quantification of 1-β-D-glucosylsphingosine by GC- or LC-MS. 1-β-D-Glucosylsphingosine is a lysolipid derivative of glucosylcerebroside that decreases activity of glucocerebrosidase in LA-N-2 cells in a dose-dependent manner.

1-β-D-Glucosylsphingadienine is a glucosylsphingosine, which are deacetylated lysolipid derivatives of glucosylcerebrosides . They are formed when sphingosines undergo glucosidation by UDP-glucose. Glucosylsphingosines completely reduce neurite outgrowth and induce death of LA-N-2 cells at concentrations of 10 and 50 μM, respectively. They also decrease the activity of glucocerebrosidase in LA-N-2 cells in a dose-dependent manner. Glucosylsphingosine levels are elevated in patients with Gaucher's disease, both in the spleen (in types 1, 2, and 3) and brain (type 2 and 3); thus, glucosylsphingosine has been used as a key biomarker of the disease. This product contains 1-β-D-glucosylsphingadienine isolated from plants.

Glucosylsphingosine (Lyso-Gb1) is a naturally occurring glycosyl ceramide, a metabolite of GBA (acid β-glucosidase), involved in cellular recognition, signaling, and intercellular interactions.Glucopsychosine accumulates in certain neurodegenerative diseases (e.g., Gaucher disease), disrupts lysosomal function through activation of mammalian target of rapamycin complex 1 (mTORC1), and is a noncompetitive inhibitor of glucocerebrosidase.

HPSE1-IN-1 (compound 16) functions as a selective Heparanase-1 (HPSE1) inhibitor, displaying moderate activity against exo-β-d-glucuronidase (GUSβ) and glucocerebrosidase (GBA) [1].

HPSE1-IN-2 (compound 2) functions as an inhibitor of Heparanase-1 (HPSE1) while concurrently inhibiting exo-β-d-glucuronidase (GUSβ) and glucocerebrosidase (GBA) activities [1].