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CGT-001

Development stage: Preclinical
Lead developer: Carbon Biosciences
Modality: Gene Therapies
Administration: Inhalation

Overview

CGT-001 is an investigational gene therapy developed by Carbon Biosciences for the treatment of cystic fibrosis. It is designed as a CFTR (cystic fibrosis transmembrane conductance regulator) replacement therapy that uses gene transfer technology to deliver functional copies of the CFTR gene to affected tissues. The therapy specifically targets lung tissue and has demonstrated upper airway tissue tropism and potential for repeatable dosing in preclinical models. As of now, its highest development status is preclinical[1][3][4].

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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