AAVogen, an early-stage biotech company, is developing a new treatment for inclusion body myositis (IBM). AVGN7 is a novel gene therapy treatment that is designed to treat IBM and other muscle wasting conditions. AAVogen has raised $2.3 million to date, mostly from NIH grants, and is now seeking another $2-3 million in seed funding. This money would be used to complete the preclinical preparation for an IND (Investigation New Drug) filing with the FDA. AAVogen hopes to start a clinical trial in 2 years.
No approved treatments exist for IBM or most other muscle wasting diseases. AVGN7 has the potential to restore the size and strength of muscles. It works by blocking the actions of myostatin and other proteins that limit muscle growth. Animal studies have shown that this indeed “takes the brakes off” muscle growth, increasing muscle size, strength, and exercise capacity.
AVGN7 has an advantage over other similar products that have been investigated, because it blocks several inhibitory proteins, not just myostatin. This broader scope of action results in a greater beneficial effect on muscles.
AVGN7 uses genetic therapy to increase the levels of a protein called Smad7 inside muscle cells. Smad7 is the molecule that does the work, blocking several metabolic pathways, resulting in muscle growth.
Rather than put Smad7 itself into the body, AVGN7 puts the gene for Smad7 into the body using a special type of virus called adeno-associated virus, or AAV. The gene for Smad7 is loaded into the virus, which then unloads the gene into the target cell where it is used to produce Smad7 protein, providing long-lasting benefit.
The adeno-associated virus does not cause disease and is not harmful to humans. It has already been used in other gene therapy products which have been approved by the FDA.
AVGN7 uses a specific version of AAV, called AAV6, which specifically targets muscle cells. The virus will not enter other types of cells. This targeting decreases the possibility of side-effects.
Many readers may be aware of the small clinical trial of follistatin gene therapy for IBM that was completed a few years ago. AVGN7 is similar, but AAVogen expects that AVGN7 will be an improved version of this treatment, with stronger benefits and less risks of side-effects.
The initial clinical trial of AVGN7 will involve direct local injection into a particular muscle, such as perhaps the quadriceps muscle of the thigh. The ultimate goal, however, will be to give AVGN7 through an IV line, allowing it to spread throughout the body, potentially benefiting all of the muscles with one treatment. If successful, AVGN7 might benefit leg strength, hand strength, and swallowing.
AAVogen needs additional investments to continue its work developing AVGN7.
Equity investments and convertible notes are available. The minimum investment amount is $50,000.
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For more information about AAVogen, please visit the company’s website.
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