Drug Profile

PBML 04

Alternative Names: PBML-04

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 Cerebroside sulfatase replacements; Gene transference

Orphan Drug Status

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

No
New Molecular Entity No

Highest Development Phases

Suspended Metachromatic leukodystrophy

Most Recent Events

01 Aug 2024 GEMMA Biotherapeutics in-licenses PBML 04 from Passage Bio
04 Aug 2022 Suspended - Preclinical for Metachromatic leukodystrophy in USA (Parenteral)
08 Jun 2022 The US FDA approves Investigational New Drug (IND) application of PBML 04 in Metachromatic leukodystrophy, in USA

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