GBA1

Lysosomal acid glucosylceramidase · P04062 · GBA1 on Sugi Atlas →

107,577 patent compounds predicted against GBA1, 55,029 high-confidence (nearest known ligand ≥ 0.5)

Top predicted compounds (top 20 by supporting neighbours, then confidence)

CompoundConfidenceSupporting neighbours
SCHEMBL94371 SCHEMBL94371 0.64 12/20
SCHEMBL102757 SCHEMBL102757 0.39 8/20
Miglustat Miglustat (SCHEMBL246893) 1.00 7/20
SCHEMBL247045 SCHEMBL247045 1.00 6/20
SCHEMBL33424 SCHEMBL33424 0.37 6/20
SCHEMBL44781 SCHEMBL44781 0.37 6/20
SCHEMBL129425 SCHEMBL129425 0.37 6/20
SCHEMBL130804 SCHEMBL130804 0.37 6/20
SCHEMBL130805 SCHEMBL130805 0.37 6/20
Miglitol Miglitol (SCHEMBL22593) 1.00 5/20
SCHEMBL61561 SCHEMBL61561 0.59 5/20
SCHEMBL136393 SCHEMBL136393 0.59 5/20
SCHEMBL182764 SCHEMBL182764 0.59 5/20
SCHEMBL191711 SCHEMBL191711 0.59 5/20
SCHEMBL258033 SCHEMBL258033 0.59 5/20
SCHEMBL126159 SCHEMBL126159 0.57 5/20
Hydrochloric Acid Hydrochloric Acid (SCHEMBL249520) 0.44 5/20
SCHEMBL49953 SCHEMBL49953 0.62 4/20
Inositol Inositol (SCHEMBL5831) 0.50 4/20
Scyllitol Scyllitol (SCHEMBL5832) 0.50 4/20

Clinical trials — most relevant to GBA1 by text similarity (a relevance ranking, not an exhaustive list)

TrialPhaseStatus
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease PHASE3COMPLETED
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease PHASE3COMPLETED
High-Dose Ambroxol in GBA1-Related Parkinson PHASE1/PHASE2UNKNOWN
Ambroxol as a Novel Disease Modifying Treatment for Lewy Body Dementia PHASE1/PHASE2NOT_YET_RECRUITING
Ambroxol as a Treatment for Parkinson's Disease Dementia PHASE2UNKNOWN
Efficacy, Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of BIA 28-6156 in GBA-PD PHASE2ACTIVE_NOT_RECRUITING
Ambroxol as a Disease-modifying Treatment in GBA-PD PHASE2COMPLETED
Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease PHASE2WITHDRAWN
Safety and Effect of Oral RVX000222 in Subjects With Fabry Disease PHASE1/PHASE2WITHDRAWN
Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease PHASE2UNKNOWN
A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease EARLY_PHASE1ACTIVE_NOT_RECRUITING
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease EARLY_PHASE1RECRUITING
A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease nanCOMPLETED
Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease nanCOMPLETED
Assessment of Neurological Manifestations in Gaucher Disease Patients nanUNKNOWN
Biomarker Analysis for GBA Associated Parkinson's Disease nanUNKNOWN
Inflammatory Pathways and Cardiac Growth Factors Associated With Fabry Disease Cardiomyopathy nanUNKNOWN
Omics Gaucher Study: Multiomic Approach nanACTIVE_NOT_RECRUITING
Biomarker for Patients With Fabry Disease (BioFabry) nanWITHDRAWN
Genetic Studies of Lysosomal Storage Disorders nanENROLLING_BY_INVITATION
the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease nanRECRUITING
Registry Gangliosidoses nanUNKNOWN
Examining the Immune Response in Patients With Gaucher Disease and Hepatitis C nanUNKNOWN
Biomarkers Related to Bone in Pediatric Gaucher Disease nanUNKNOWN
Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease nanCOMPLETED
A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition nanCOMPLETED
Biomarker for Gaucher Disease: BioGaucher (BioGaucher) nanWITHDRAWN
Prodromal Parkinsonian Features in GBA1 Mutation Carriers nanRECRUITING
Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease nanUNKNOWN
Screening of Fabry Disease in Patients With GI Symptoms nanUNKNOWN

Related proteins — ESM-2 sequence neighbours (a GBA1 binder may also engage these)

ProteinNameSimilarity
Q9BDT0 1.000 Q9BDT0 →
Q5R8E3 1.000 Q5R8E3 →
Q70KH2 1.000 Q70KH2 →
Q2KHZ8 0.995 Q2KHZ8 →
P17439 0.995 P17439 →
Q6YGZ1 0.960 Q6YGZ1 →
Q8WP17 0.958 Q8WP17 →
Q9UEF7 0.957 Q9UEF7 →
Q71RP1 0.957 Q71RP1 →
F1NZI4 0.955 F1NZI4 →
Q9Z2Y9 0.955 Q9Z2Y9 →
HPSE Heparanase 0.952 landscape →
Q8BP56 0.952 Q8BP56 →
O35082 0.952 O35082 →
Q9MYY0 0.951 Q9MYY0 →
P58242 0.950 P58242 →
P53761 0.949 P53761 →
Q5NVF6 0.948 Q5NVF6 →
O89023 0.947 O89023 →
O35795 0.947 O35795 →
Q04519 0.946 Q04519 →
Q0VD19 0.946 Q0VD19 →
Q8BX37 0.946 Q8BX37 →
Q6XPZ3 0.946 Q6XPZ3 →
Q9EQV6 0.946 Q9EQV6 →
ALPG Alkaline phosphatase, germ cell type 0.946 landscape →
O97583 0.945 O97583 →
Q6ZNF0 0.945 Q6ZNF0 →
Q6UWV6 0.945 Q6UWV6 →
Q0P5F0 0.944 Q0P5F0 →