TSHA-101 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病1

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04798235
(ClinicalTrials.gov)
March 12, 202122/2/2021First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 GangliosidosisPhase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 GangliosidosisInfantile GM2 Gangliosidosis (Disorder)Biological: TSHA-101Dr. Anupam SehgalTaysha Gene Therapies, Inc.;GlycoNetActive, not recruitingN/A15 MonthsAll3Phase 1/Phase 2Canada