Drug Profile

PBGM 01

Alternative Names: PBGM-01

Latest Information Update: 07 Aug 2024

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At a glance

Originator University of Pennsylvania
Developer Passage Bio
Class Gene therapies
Mechanism of Action Beta-galactosidase replacements; Gene transference

Orphan Drug Status

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

Yes - Gangliosidoses
New Molecular Entity No

Highest Development Phases

Phase I/II Gangliosidoses

Most Recent Events

01 Aug 2024 GEMMA Biotherapeutics in-licenses PBGM 01 from Passage Bio
07 Aug 2023 Interim pharmacodynamics and adverse events data from a phase I/II Imagine-1 trial in Gangliosidoses released by Passage Bio
07 Aug 2023 Interim pharmacodynamics and adverse events data from a phase I/II trial in Gangliosidoses released by Passage Bio

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