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3DNA-based gene therapy for Duchenne Muscular Dystrophy

Development stage
Preclinical
Lead developer
Code Biotherapeutics
Modality
Gene Therapies
Administration
Intravenous, Intramuscular
01

Overview

The 3DNA-based gene therapy for Duchenne Muscular Dystrophy (DMD) is a non-viral gene therapy candidate being developed by **Code Biotherapeutics** in collaboration with **Takeda**. The therapy utilizes the proprietary **3DNA platform**, which consists of a synthetic, multivalent DNA scaffold designed to deliver large genetic payloads. Unlike traditional viral-based gene therapies (such as those using AAV vectors) that are limited by payload capacity and pre-existing immunity, the 3DNA technology is capable of delivering the **full-length dystrophin gene**. This approach aims to restore the production of the functional dystrophin protein in both skeletal and cardiac muscle tissues. Because it is non-viral, the therapy is designed to be non-immunogenic, potentially allowing for redosing and treatment of patients regardless of their specific genetic mutation or pre-existing antibodies to viral vectors.

Other names
3DNA DMD gene therapyCode Bio DMD program3DNA-DMD
02

Targets

DMD (Dystrophin)

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