Drug Profile

PBKR 03

Alternative Names: PBKR-03

Latest Information Update: 07 Aug 2024

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At a glance

Originator University of Pennsylvania
Developer Passage Bio
Class Antidementias; Gene therapies
Mechanism of Action Galactosylceramidase replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Globoid cell leukodystrophy
New Molecular Entity No

Highest Development Phases

Suspended Globoid cell leukodystrophy

Most Recent Events

01 Aug 2024 GEMMA Biotherapeutics in-licenses PBKR 03 from Passage Bio
10 Nov 2022 Suspended - Phase-I/II for Globoid cell leukodystrophy (In infants) in United Kingdom (Intracisternal) (NCT04771416)
10 Nov 2022 Suspended - Phase-I/II for Globoid cell leukodystrophy (In infants) in USA, Israel, Canada, Netherlands (Intracisternal)

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