A novel drug delivery route
For the genetic therapies that are revolutionizing medicine, the liver has been the prime target for delivery. As it targets genetically driven neuromuscular disease, Dyne Therapeutics is committing to a different approach.
"We're finally figuring out how to deliver these genetic medicines to the billions of muscle cells, and across the blood-brain barrier to the central nervous system," says John Cox, President and CEO.
He sees this delivery method as disruptive – a culmination of work in genetic medicines known as oligonucleotides that began in the 1970s.
Dyne's FORCE platform uses an antibody fragment (Fab) with the ability to deliver higher doses of therapy than was previously possible, with favorable safety profiles.
In 2027, Dyne expects to launch its first drug, z-rostudirsen, targeting Duchenne muscular dystrophy. A second therapy, for people living with myotonic dystrophy type 1 will follow.
Trials have been promising. "The best anybody has hoped for has been a slowing of decline in the disease. What we've been seeing in both of these diseases is improvement from baseline in function," says Cox.
"We're finally figuring out how to deliver genetic medicines to the billions of muscle cells, and across the blood-brain barrier to the central nervous system."
John Cox, President and CEO, Dyne Therapeutics
Next phase of growth
The development pipeline is pushing Dyne to its own critical transformation. The Massachusetts-based business is set to make the leap from early-stage to a full commercial biotech company.
That has required strengthening the company with new forms of expertise. "We're building out capabilities in patient services, market access, medical affairs, and scaling from a manufacturing and inventory standpoint," says Cox.
He seeks out people who share a passion for targeting unmet medical need. Visitors are struck by how energized the workforce is, says Cox.
"They say, 'My God, it's Friday at 5 o'clock, and the parking lot's full'. It's because everyone's on a mission to make a difference – and this is just the beginning for us."
Poised for investment
New and existing investors have bolstered Dyne's financial firepower: it has over $900 million on the balance sheet.
"The key at this point is to allocate that capital to the right things," Cox says. "Fortunately, in our disease areas, we only need one sales force and one commercial infrastructure."
"In our FORCE platform, we use the same Fab, so we essentially have the same manufacturing and supply chains, and we don't have to build duplicate infrastructures."
Dyne will focus spending on advancing clinical and regulatory processes and preparing for launch.
