UW biotech spinout to develop gene therapy system – Myosana Therapeutics, a Seattle-based company developing gene therapy technology, has raised $5 million to fund the development of an early-stage candidate treatment for Duchenne muscular dystrophy (DMD). Mysona claims that its experimental platform for delivering genes to cells outperforms the standard method, which is limited to smaller pieces of DNA.
The team: The company was founded in 2018 by Stanley Froehner, a University of Washington professor of physiology and biophysics, and Nick Whitehead, a UW research associate professor. Froehner is the company’s chairman, and Whitehead is its chief scientific officer.
Matthew Lumley, a partner at Medicxi who has been on Myosana’s board for over a year, was appointed CEO in January. Lumley, who lives in London, previously worked as the senior director of rare disease clinical development at Moderna and as the medical director of rare diseases at Pfizer; his son also has DMD, which causes muscle weakness and atrophy. [UW biotech spinout to develop gene therapy system]
DMD is caused by mutations in the largest known human gene, which encodes a protein called dystrophin. Several companies, including Pfizer and Sarepta Therapeutics, are working on gene therapies that use modified viruses called AAVs to introduce functional dystrophin DNA into cells (adeno-associated viruses). Because AAVs cannot handle large genes, they are instead loaded with a smaller piece of DNA that produces a truncated form of dystrophin.
Sarepta has requested FDA approval based on data demonstrating that the introduced gene is active in cells; a decision is expected in the spring, and the company has raised $1.2 billion to market the therapy while clinical trials are completed. Sarepta also sells three approved drugs that aim to treat about 30% of DMD patients through a technique known as exon skipping.
Lipid nanoparticles, a technology similar to that used in the COVID-19 RNA vaccines, are another experimental gene therapy delivery approach. [UW biotech spinout to develop gene therapy system]
Myosana has created antibodies that bind to a protein on skeletal and cardiac muscle cells in order to deliver genes into them. The system is capable of delivering large-sized genes and can be used repeatedly, whereas AAV vectors are typically designed for single use due to the risk of developing immunity.
Although no data on Myosana’s system has been published, Whitehead told GeekWire that it has been used to deliver the entire DMD gene to muscles in mice via intravenous injection.
The startup intends to use the new funding to help identify an early development candidate for DMD by 2025, but the platform has broad application potential. “Success in treating Duchenne would open up opportunities for Myosana to target a wide range of neuromuscular and cardiac diseases as proof of principle for the platform,” Lumley said in a statement announcing the funding on Wednesday. [UW biotech spinout to develop gene therapy system]
The investors are as follows: John Ballantyne, co-founder and former chief scientific officer of Fargo, North Dakota-based Aldevron, which provides contract drug development and manufacturing services, led the funding round. Ballantyne also co-founded Agathos Biologics, a Fargo-based startup focused on biomanufacturing and therapeutic molecule delivery into cells, in 2021.
The Muscular Dystrophy Association contributed $650,000 to the seed round, and Parent Project Muscular Dystrophy contributed $500,000, building on a $350,480 investment in Myosana in August 2021. CureDuchenne Ventures was an early investor in Myosana as well.
