Drug intelligence / Profile preview

4D-710

Development stage
Phase 2
Lead developer
4D Molecular Therapeutics
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
Administration
Inhalation
01

Overview

4D-710 is an investigational gene therapy designed for the treatment of cystic fibrosis (CF). It utilizes a customized adeno-associated virus (AAV) vector, specifically the A101 serotype, to deliver a functional copy of the CFTR gene directly to lung cells via aerosol inhalation. This approach enables lung cells to produce normal CFTR protein regardless of the patient’s specific underlying CFTR mutation. The therapy aims to restore proper regulation of water and salt transport in airway epithelial cells, thereby reducing mucus buildup and alleviating symptoms associated with CF. 4D-710 is being developed by 4D Molecular Therapeutics and is currently in Phase II clinical trials for cystic fibrosis.

02

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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