No AAV? No problem. Code Biotherapeutics joins the search for a non-viral gene therapy
Brian McVeigh’s wake-up call came one morning in the form of a LinkedIn notification congratulating him for 25 years at GlaxoSmithKline.
“I was like, ‘Oh my God, it has been a quarter century at one company, what am I doing?’” he said.
McVeigh quit his job as VP of worldwide business development transactions and investment management in November 2017, spent half a year as the CEO of KBP BioSciences, and eventually ended up on the board of Genisphere, where scientists were working on a drug delivery platform using synthetic DNA.
“As this started to grow and preclinical data started to get generated, we realized that it really needs to be on its own in order to achieve its full potential,” he said.
Last April, he and brother-sister duo Bob and Lori Getts founded Code Biotherapeutics to take the “next major leap forward” in gene therapy. And on Tuesday, they officially launched with $10 million in seed funding.
As CEO, McVeigh is leading the development of a synthetic DNA-based vector, dubbed 3DNA, to overcome the current challenges of AAV. The vector consists of a series of strands of synthetic DNA linked together to form a scaffold, which unlike AAV, doesn’t trigger an immune response.
“That is a big issue with AAV — that folks have either natural immunity to the viral vectors, or once they’re dosed with it, they create an immune response to it so that you can’t re-dose,” McVeigh said.
The ability to re-dose would allow scientists to treat people with a lower dose to start, potentially avoiding some of the safety issues involved with giving higher doses.
Another key bonus to using synthetic DNA is that scientists are not limited by the size of the gene construct they’re trying to deliver. McVeigh says they have data so far showing successful delivery of gene constructs up to 10 kb in length.
“What that means is that we could actually go after diseases that you can’t currently go after with AAV approaches, and that we can also go after diseases (for which) there are some AAV approaches but they’re not necessarily giving the optimized gene construct because it can’t fit,” he said.
Code is currently in the discovery phase for therapies to treat type 1 diabetes and Duchenne muscular dystrophy (DMD), a debilitating disorder caused by mutations in the dystrophin gene. McVeigh guesses those programs are still about two years away from entering the clinic.
The biotech will face stiff competition for the longtime holy grail. Generation Bio closed a $230 million IPO last June to fund its preclinical push for a non-viral gene therapy to treat diseases of the liver and retina. And Michael Ehlers is leading a new effort over at Apple Tree Ventures.
4BIO Capital and UPMC Enterprises co-led the seed financing, with a little help from CureDuchenne Ventures, JDRF T1D Fund, New Enterprise Associates, and Takeda Ventures. The company recently tapped Spark Therapeutics alum Lauren Kaskiel as CBO. Kathy Rouan, former SVP of R&D at GSK, is on the board.
“This is the thing that has so caught my attention and passion more than anything else,” McVeigh said. “It’s got the potential here to be such a game changer and to move the field forward in a dramatic way.”