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

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Approved Drug(s)
Clinical Trial Drug(s)
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]
⏷ Show the Full List of 10 Drug(s)

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

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.