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PBCM06

Development stage: Discontinued
Lead developer: Passage Bio
Modality: Gene Therapies
Administration: Intra-cisterna Magna

Overview

PBCM06 is an investigational **gene therapy** program developed by Passage Bio and originally advanced from the University of Pennsylvania for **Charcot-Marie-Tooth disease type 2A**. The program has been described as an **MFN2 modulator** intended for direct delivery to the **central nervous system**, with therapeutic rationale centered on modifying mitofusin 2 biology to address disease caused by dysfunction of the **MFN2** pathway. Publicly available pipeline summaries indicate that PBCM06 remained in **preclinical development** and that its overall R&D status was later reported as **discontinued**.

Targets

MFN2 (Mitofusin-2)

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