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Myomaker Bio

Los Angeles, California, United States

Ownership: Private
Development stage: Other

Overview

MyoGene Bio is a pre-clinical stage biotechnology company focused on developing cutting-edge gene-editing therapies for muscle-wasting diseases. The company's primary focus is on Duchenne muscular dystrophy (DMD), utilizing a proprietary CRISPR/Cas9 platform to permanently delete specific exons and restore the reading frame of the dystrophin gene. By targeting the underlying genetic cause of DMD, MyoGene Bio aims to provide a long-term solution that improves muscle function and quality of life for patients. The company is also exploring the use of muscle-specific proteins like Myomaker to enhance the delivery and efficacy of its therapeutic candidates.

Therapeutic areas: Rare Diseases
Modalities: Gene therapyCRISPR-based therapy
Industry: Biotech

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