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4D-725

Development stage: Preclinical
Lead developer: 4D Molecular Therapeutics
Modality: Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration: Inhalation

Overview

4D-725 is an investigational genetic medicine developed by 4D Molecular Therapeutics (4DMT). It is an AAV-based gene therapy utilizing the proprietary A101 aerosol vector. Designed for single-dose aerosol treatment, 4D-725 aims to deliver a corrective transgene to patients with alpha-1 antitrypsin deficiency lung disease, thereby addressing the underlying genetic defect.

Targets

SERPINA1 (Alpha-1-antitrypsin)

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