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

General Information of Drug (ID: DMIDEKB)

• Drug Name: AAV2-hFIX16 Drug Info
• Synonyms: AAV-F.IX, University of Pennsylvania; AAV-factor IX gene therapy (hemophilia B), University of Pennsylvania

Indication

Disease Entry	ICD 11	Status	REF
Factor IX deficiency	3B11	Phase 1	[1]

• Cross-matching ID: TTD Drug ID: DMIDEKB

Molecule-Related Drug Atlas

Drug(s) Targeting Coagulation factor IX (F9)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Emicizumab	DM0AQ83	Factor VIII deficiency	3B10	Approved	[3]
Recombinant Factor IX	DMY7ESM	Bleeding disorder	GA20-GA21	Approved	[4]
CSL-654	DMZJGVL	Factor IX deficiency	3B11	Phase 3	[5]
RG6013	DMER75Y	Haemophilia A	3B10.0	Phase 3	[6]
AMT-061	DMSO02K	Haemophilia B	3B11.0	Phase 3	[7]
Fidanacogene elaparvovec	DMFZ81T	Haemophilia B	3B11.0	Phase 3	[8]
Mim8	DMX33ZR	Haemophilia A	3B10.0	Phase 3	[9]
TTP889	DMCQARM	Blood forming organ disorder	JB64.1	Phase 2	[10]
AMT-060	DM7RNKM	Factor IX deficiency	3B11	Phase 1/2	[11]
SB-FIX	DMUP52Y	Haemophilia B	3B11.0	Phase 1/2	[12]

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Coagulation factor IX (F9)	TTFEZ5Q	FA9_HUMAN	Modulator	[2]

References

• [1] ClinicalTrials.gov (NCT00515710) LTFU for Gene Transfer Subjects With Hemophilia B. U.S. National Institutes of Health.
• [2] Assessing the potential for AAV vector genotoxicity in a murine model. Correction in: Blood. 2011 June 16; 117(24): 6739.
• [3] 2017 FDA drug approvals.Nat Rev Drug Discov. 2018 Feb;17(2):81-85.
• [4] BAX326 (RIXUBIS): a novel recombinant factor IX for the control and prevention of bleeding episodes in adults and children with hemophilia B. Ther Adv Hematol. 2014 Oct;5(5):168-80.
• [5] Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood. 2012 September 20; 120(12): 2405-2411.
• [6] Anti-factor IXa/X bispecific antibody (ACE910): hemostatic potency against ongoing bleeds in a hemophilia A model and the possibility of routine supplementation. J Thromb Haemost. 2014 Feb;12(2):206-13.
• [7] Clinical pipeline report, company report or official report of uniQure.
• [8] Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua. J Thromb Haemost. 2021 May;19(5):1212-1218.
• [9] ClinicalTrials.gov (NCT05878938) Open-label Safety Study in Adults and Adolescents With Haemophilia A With and Without FVIII Inhibitors Switching Directly From Emicizumab Prophylaxis to NNC0365-3769 (Mim8) Prophylaxis. U.S.National Institutes of Health.
• [10] Partial factor IXa inhibition with TTP889 for prevention of venous thromboembolism: an exploratory study. J Thromb Haemost. 2008 Mar;6(3):457-63.
• [11] Phase I/II clinical trial of AMT-060 for treating hemophilia B. uniQure N.V.
• [12] ClinicalTrials.gov (NCT02695160) Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B. U.S. National Institutes of Health.