Robert Kotin is one of the world’s top experts on the viral transport of gene therapies. Ex-NIH, ex-Voyager exec, he’s played a sizable role in shaping the field.
So when Kotin says there’s a better way to do gene transfer without a viral vehicle, he managed to get the attention of quite a few people in the Boston/Cambridge hub.
Jason Rhodes, a partner at Atlas, was one of them. Atlas formed a company around this called Generation Bio with a $25 million A round and Rhodes is chairing the board. Geoff McDonough, who stepped aside as CEO of Stockholm-based Sobi last summer after the company decided to go with a new chief who preferred to live closer to home, is taking the helm.
Like Ed Kaye, who’s having his launch party today at Stoke, McDonough is a high profile figure in the Massachusetts hub. And he’s pumped about pursuing a breakthrough approach to gene transfer.
Kotin’s big idea centers on what he calls close-ended DNA (ceDNA), which can move from the cytoplasm of the cell to the nucleus without that AAV transport vehicle. Once inside the nucleus, the theory goes, it can create durable high levels of gene expression capable of addressing a broad range of genetic diseases. And the biotech plans to get started by using a lipid nanoparticle to deliver ceDNA to the liver, where it will make systemic proteins to fight disease.
“The first organ is the liver because that’s where lipid nanoparticles go,” McDonough told me in a preview to today’s announcement. “So we go to rare diseases of the liver, where it can be a factory.”
And nanoparticles have some inherent qualities that make it preferable to an AAV approach, with a bigger payload that can pack a bigger punch. It’s also a new way to do this without triggering an immune response that is limiting the first generation of gene therapies to a one-time use.
Notes McDonough: “This is a completely novel modality which is tremendously disruptive to AAV.”
While much of Atlas’ investing can be highly asset-centric, crafting companies around one or two lead programs in a carefully managed drive to the clinic, Generation Bio is every bit a platform company with a lot of potential directions to move. But Rhodes says you can’t relegate a venture group like Atlas — which has enjoyed the ability to raise considerable funds — to a narrow focus. The VC, he says, is “science-first, seed-led, pragmatic about the business model and intensely focused on creating innovative new products. And not too worried about ideology beyond that.”
In response to a query from me, Rhodes added:
We always match the nature of the opportunity to the business model and build both major platform companies and more focused product plays. This strategic matching is a hallmark of Atlas’ approach vs a cookie cutter, whether always a monster platform or always an asset play, etc. Recent platforms include NTLA, Surface, Magenta, Translate, Nimbus (with an LLC structure in the mix), etc. Early M&A tends to occur with the product-oriented companies, like Delinia, Padlock, and IFM and so generates early press and attention. Padlock is really a platform around an area of biology as is IFM, which sold the onc portfolio and kept the immunology portfolio. We do consistently use a seed approach, where we pressure test the core science, begin building a team etc. then ramp us the respective business model. I’ve been involved with all of these kinds of companies whether at Alnylam (new modality platform), Epizyme (new biology multi-product platform), Replimine (product play, though more than one), Disarm (new biology product), and Generation, which is obviously a major new modality, among a few examples.
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 51,100+ biopharma pros who read Endpoints News by email every day.Free Subscription