Gene therapy

AAV vectors are the holy grail of gene therapy.  These viral tools introduce "payload genes" into different tissues often with great precision.  They do not cause disease and have a proven safety record in clinical trials. Thus, their use has the potential to provide safe and long-term relief for genetic and/or chronic diseases.

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Strong healthy muscle

The AAVogen pipeline includes gene therapeutics for muscle wasting diseases, especially muscular dystrophy and cancer cachexia. In pre-clinical studies, our gene therapeutics stabilize muscle structure, increase muscle mass and strength and prevent muscle wasting.

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Strong healthy hearts

Cardiac and skeletal muscle wasting go hand-in-hand.  Indeed, addressing cardiac and respiratory failure is key to preventing mortality in many muscle wasting diseases. In pre-clinical studies, our gene therapeutics correct deficits in cardiac muscle structure and in addition, enhance exercise capacity.

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At AAVogen, it's personal

Our mission is to make you stronger. The company was founded by a family directly impacted by two muscle wasting diseases: Duchenne muscular dystrophy and cancer cachexia. Our fight is personal and is shared by affected families across the globe and our commitment to developing better and more effective therapies is undaunted.

AVGN7 prevents muscle wasting in animals with cancer and can restore muscle mass and strength even after wasting has already occurred. AVGNF stabilizes muscle and prevents its degeneration in animal models of LGMD2i, a rare form of muscular dystrophy.

Both drugs act only in muscle and are potentially more safe than alternative approaches being developed by other companies. They could also be used in combination or with other drugs.



Patient outreach

We understand the challenges of fighting a muscle wasting disease. We also understand the role education plays in empowering patients.

If you have questions about AAVogen or our drug pipeline, about gene therapy in general or even different muscle wasting diseases, please contact us. You can also learn more about investing and collaborating with AAVogen by visiting the link to the right.

Whether you're curious, scared or intrigued, we want to hear from you.