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Seattle, WA

Developing a non-viral gene therapy for muscular dystrophy and heart diseases.

• Non-Profit Support: Three major non-profit organizations have invested in Myosana, highlighting the potential impact of their therapy.
• Experienced Team: The Myosana team has over 200 years of combined experience in treating muscular dystrophy.
• Innovative Platform: The platform overcomes limitations of viral vector-delivered gene therapy, offering targeted muscle delivery and immune neutrality.
• Broad Application: The technology has potential applications beyond DMD, including other muscle and heart diseases.
• Clinical Trials: Preparations are underway for early clinical trials to test the safety and efficacy of the treatment.

Myosana Therapeutics is advancing a novel gene therapy platform aimed at treating Duchenne Muscular Dystrophy (DMD) and other muscle and heart diseases. The company has developed a non-viral delivery system that targets muscle cells directly, allowing for the delivery of full-length dystrophin, a crucial protein missing in DMD patients. This approach addresses the limitations of current viral vector-based therapies, which often deliver incomplete proteins and have significant side effects. Myosana’s technology also shows promise in treating other genetic muscle conditions and heart diseases.

The company is currently in the process of preparing for early clinical trials to test the safety and efficacy of their treatment in boys with DMD. With support from major non-profit organizations like Parent Project Muscular Dystrophy, Muscular Dystrophy Association, and Cure Duchenne, Myosana is poised to make significant strides in the field of gene therapy. The team, with over 200 years of combined experience in muscular dystrophy research, is focused on bringing this potentially life-changing treatment to market, offering hope to thousands of families affected by these debilitating conditions.

Company Info

Myosana Therapeutics is developing a non-viral gene therapy platform to treat Duchenne Muscular Dystrophy and other muscle diseases by delivering the full-length dystrophin gene directly to muscle cells.

Myosana Therapeutics is focused on developing a non-viral gene therapy platform aimed at treating Duchenne Muscular Dystrophy (DMD) and other muscle and heart diseases. The company’s innovative approach involves delivering the full-length dystrophin gene directly to muscle cells, including the heart, without using viral vectors. This method is designed to overcome the limitations of current gene therapies, which often involve high costs and partial protein delivery with severe side effects.

The platform utilizes special antibodies to target muscle cells, enabling the delivery of the complete dystrophin gene, which is crucial for muscle function. This approach not only aims to restore dystrophin in muscle tissue but also supports long-term muscle repair by working in muscle stem cells. Myosana’s technology has shown promising results in preclinical studies, reducing muscle damage and improving muscle strength, and is progressing towards human clinical trials.