General Information of DTT (ID: TTKIBKM)

DTT Name Beta-hexosaminidase subunit beta (HEXB) DTT Info
Gene Name HEXB

Molecule-Related Drug & Molecule List

Molecule-Related Drug List
Molecule Type:
DTT
DTP
DME
Drug Status:
Clinical Trial Drug(s)
2 Clinical Trial Drug(s) Transported by This DTP
Drug Name Drug ID Indication ICD 11 Highest Status REF
TSHA-101 DMFTPCT GM2 gangliosidosis 5C56.00 Phase 1/2 [1]
AXO-AAV-GM2 DMISRRC Tay-sachs disease 5C56.00 Phase 1 [2]
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Molecule Interaction Atlas

References

1 ClinicalTrials.gov (NCT04798235) Phase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis. U.S.National Institutes of Health.
2 ClinicalTrials.gov (NCT04669535) A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease. U.S.National Institutes of Health.