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4D-710

4D Molecular Therapeutics · Phase 2 active Biologic

4D-710 is a gene therapy that uses an adeno-associated virus to deliver a functional copy of the CFTR gene to the lungs, correcting the underlying genetic defect in cystic fibrosis.

4D-710 is a gene therapy that uses an adeno-associated virus to deliver a functional copy of the CFTR gene to the lungs, correcting the underlying genetic defect in cystic fibrosis. Used for Cystic fibrosis.

At a glance

Generic name4D-710
Sponsor4D Molecular Therapeutics
Drug classGene therapy
TargetCFTR gene
ModalityBiologic
Therapeutic areaCystic Fibrosis
PhasePhase 2

Mechanism of action

This approach aims to restore normal CFTR protein function, which is essential for the proper functioning of chloride channels in the lungs, thereby improving lung function and reducing the risk of pulmonary exacerbations. By delivering the CFTR gene directly to the lungs, 4D-710 seeks to bypass the need for systemic gene therapy and minimize potential off-target effects.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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