UW Biotech Firm Raises M To Develop Gene Therapy System For Muscular Dystrophy – GeekWire

UW Biotech Firm Raises $5M To Develop Gene Therapy System For Muscular Dystrophy – GeekWire

From left: Myosana CEO Matthew Lumley with co-founders Stanley Froehner and Nick Whitehead. (Photos by Myosana and UW)

News: Myosana Therapeutics, a Seattle startup developing gene therapy technology, raised $5 million to develop an early-stage treatment candidate for Duchenne muscular dystrophy (DMD). Mysona says his experimental platform for delivering genes to cells has advantages over the standard approach, which is limited to smaller pieces of DNA.

People: Co-founders Stanley Froehner, a University of Washington professor of physiology and biophysics, and Nick Whitehead, a UW research associate professor, launched the company in 2018. Froehner serves as president and Whitehead as chief scientific officer.

Matthew Lumley, a partner at venture firm Medicxi who has served on Myosana’s board for more than a year, was appointed chief executive in January. London-based Lumley was previously senior director of rare disease clinical development at Moderna and medical director of rare diseases at Pfizer; his son also has DMD, which causes muscle weakness and atrophy.

The therapeutic landscape: DMD is caused by mutations in the largest known human gene, called DMD, which codes for a protein called dystrophin. Companies developing gene therapies include Pfizer and Sarepta Therapeutics, which use modified viruses called AAVs (adeno-associated viruses) to produce a truncated form of dystrophin in muscle cells.

Sarepta has applied for US Food and Drug Administration approval based on data showing that the introduced gene is active in cells; That decision is expected in the spring, and the company has raised $1.2 billion to market the therapy while it completes studies evaluating clinical outcomes. Sarepta also markets three approved drugs that together aim to treat about 30% of DMD patients using a method called exon skipping.

Other experimental gene therapy delivery approaches include lipid nanoparticles, technology similar to that behind the COVID-19 RNA vaccines.

The technology: Myosana has developed antibodies that lodge on a protein in cardiac and skeletal muscle cells to deliver genes to them. The system can deliver large genes and can be used repeatedly, whereas AAV vectors are generally designed for single use due to the risk of developing immunity.

Data on the Myosana system is not published, but Whitehead told GeekWire that they have used it to deliver the entire DMD gene to the muscles of mice via an intravenous injection.

The startup aims to use the new funding to help identify an early development candidate for DMD by 2025, but the platform has the potential for wide application. “As a proof-of-principle for the platform, success in treating Duchenne would open up opportunities for Myosana to address a wide range of neuromuscular and cardiac diseases,” Lumley said in a statement Wednesday announcing the funding.

The investors: The funding round was led by John Ballantyne, co-founder and former chief scientific officer of Fargo, North Dakota-based Aldevron, a company that provides contract drug development and manufacturing services. In 2021, Ballantyne also co-founded Fargo-based Agathos Biologics, a start-up focused on biomanufacturing and delivering therapeutic molecules to cells.

Initial round participants are Muscular Dystrophy Association, which contributed $650,000, and Parent Project Muscular Dystrophy, which contributed $500,000, building on a $350,480 investment in Myosana in August 2021. CureDuchenne Ventures was also a previous investor in Myosana.

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