Drug Information
Drug General Information | Top | |||
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Drug ID |
D8FX9H
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Drug Name |
PBFT02
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Drug Type |
Gene therapy
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Indication | Frontotemporal dementia [ICD-11: 6D83] | Phase 1/2 | [1] | |
Company |
Passage Bio
|
Target and Pathway | Top | |||
---|---|---|---|---|
Target(s) | Progranulin (PGRN) | Target Info | . | [2] |
References | Top | |||
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REF 1 | ClinicalTrials.gov (NCT04747431) A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered Into the Cisterna Magna of Adult Subjects With Frontotemporal Dementia and Mutations in the Progranulin Gene. U.S.National Institutes of Health. | |||
REF 2 | Clinical pipeline report, company report or official report of Passage Bio |
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