Drug intelligence / Profile preview

PACS2

Development stage
Preclinical
Lead developer
Southwest Medical University
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Intramuscular
01

Overview

PACS2 (Phosphofurin acidic cluster sorting protein 2) is a multifunctional sorting protein localized at mitochondria-associated endoplasmic reticulum membranes (MAMs) that plays a critical role in maintaining ER homeostasis and MAM structural integrity. In sepsis-induced myopathy (SIM), PACS2 levels are significantly depleted, leading to MAM disruption and the activation of excessive FAM134B-mediated ER-phagy (reticulophagy), which contributes to muscle wasting. Therapeutic intervention using adeno-associated virus (AAV)-mediated overexpression of PACS2 has demonstrated the ability to restore MAM integrity and activate the ERK–MAPK signaling axis. This activation prevents the nuclear translocation of Transcription Factor EB (TFEB), thereby downregulating FAM134B and ameliorating skeletal muscle atrophy and dysfunction in preclinical models.

Other names
Phosphofurin acidic cluster sorting protein 2
02

Targets

FAM134B (Reticulophagy regulator 1)ERKHDAC6 (Histone deacetylase 6)MAM (Mitochondria-associated endoplasmic reticulum membrane)

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