Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMC1XHK)

• Drug Name: AVR-RD-01 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
Fabry disease	5C56.01	Phase 1/2	[1]

• Cross-matching ID: TTD Drug ID: DMC1XHK

Molecule-Related Drug Atlas

Drug(s) Targeting Alpha-galactosidase A (GLA)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Pegunigalsidase alfa	DMOUW65	Fabry disease	5C56.01	Approved	[3]
Migalastat	DMFIQ2H	Fabry disease	5C56.01	Approved	[4]
ST-920	DMOJIPG	Fabry disease	5C56.01	Phase 1/2	[5]
4D-310	DMNG59Y	Fabry disease	5C56.01	Phase 1/2	[6]
isaralgagene civaparvovec	DM5SSPZ	Fabry disease	5C56.01	Phase 1/2	[7]
Alpha-D-Mannose	DMF5DLW	Discovery agent	N.A.	Investigative	[8]
Fucose	DMAHMSV	N. A.	N. A.	Investigative	[8]
2,5-dideoxy-2,5-imino-D-altritol	DMI1O54	Discovery agent	N.A.	Investigative	[9]
Beta-1-C-Butyl-1-deoxygalactonojirimycin	DMMKDEZ	Discovery agent	N.A.	Investigative	[9]
Beta-1-C-butenyl-1-deoxygalactonojirimycin	DM6GTBC	Discovery agent	N.A.	Investigative	[9]

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Alpha-galactosidase A (GLA)	TTIS03D	AGAL_HUMAN	Not Available	[2]

References

• [1] ClinicalTrials.gov (NCT03454893) FAB- GT Open-Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease. U.S. National Institutes of Health.
• [2] Clinical pipeline report, company report or official report of Avrobio.
• [3] Characterization of a chemically modified plant cell culture expressed human alpha-Galactosidase-A enzyme for treatment of Fabry disease. Mol Genet Metab. 2015 Feb;114(2):259-67.
• [4] AT-1001: a high-affinity alpha3beta4 nAChR ligand with novel nicotine-suppressive pharmacology. Br J Pharmacol. 2015 Apr;172(7):1834-45.
• [5] AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction. Mol Ther Methods Clin Dev. 2020 Jul 9;18:607-619.
• [6] Clinical pipeline report, company report or official report of 4D Molecular Therapeutics
• [7] ClinicalTrials.gov (NCT04046224) A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease (STAAR). U.S.National Institutes of Health.
• [8] How many drug targets are there Nat Rev Drug Discov. 2006 Dec;5(12):993-6.
• [9] 2,5-Dideoxy-2,5-imino-d-altritol as a new class of pharmacological chaperone for Fabry disease. Bioorg Med Chem. 2010 Jun 1;18(11):3790-4.