Whoever comes out on top of the current race to gain pioneering approvals for new AAV-delivered gene therapies will have to look over their shoulders to watch the next tech wave forming on the horizon for gene therapy 2.0.

One of those next-gen players, Generation Bio, just brought in $110 million of venture cash to cover the cost of the rest of their preclinical journey toward something completely new in the field. The latest round brings the biotech which now has about 80 staffers up to $235 million in total since its inception about 3 years ago. That will fuel the rest of its preclinical stage of development as it looks to break into human studies in the back half of 2021.

That kind of 4-plus year timeline before the first human dosing could test the endurance level of a venture player. But Generation CEO Geoff McDonough looks over the past 2 years advancing a new lipid nanoparticle delivery system for their closed-end DNA therapies working to the day when gene therapies can be produced and sold for far less than the $2 million-or-so price tag today and sees lots of fast-paced advances.

I think the reality is we didnt have an expectation at the outset (on timelines), McDonough tells me. Recognizing the novel work needed to build the platform, the investors knew it would take time and money to bring them up to a GMP level.

I would say for a 40-year problem, adds the CEO, 2 years seems pretty good.

The founding tech at Generation was designed to do what AAV treatments do in the nucleus, offering enduring expression, while allowing manufacturing at a biologic scale with a more economical, capsid-free production method. Taking a page from the tech handbooks at companies like Alnylam and Moderna, theyre building a gene therapy that they believe can do much better than the fragile, one-time-only pioneers. And without the $1 million production cost that keeps wholesale prices in the low 7-figure range.

Theyre looking for much greater economy, eventually taking these therapies to much broader ailments and out of the realm of rare diseases with a new approach that they believe can be infinitely redosable on an as-needed basis.

Thats the big picture.

Generations team is working on 2 lead programs for hemophilia A and phenylketonuria (PKU) to go into IND-enabling studies. Theyve now identified Wilson disease and Gaucher disease as likely starting points for the next steps as they move past the liver to skeletal muscle and the retina and then other tissues. And McDonough the former CEO at Sobi is looking down the road 12 to 18 months when hed like to turn to the public markets with an IPO to fund the first clinical-stage work.

In the meantime, hed like to concentrate on opening another new chapter of the company on the dealmaking side.

It felt very important not to partner initially, says McDonough. The investors wanted to retain ownership of platform. We just had tremendous good fortune we didnt need to do that for finance reasons. But now that they have a better grasp of the technology and what needs to be done, its time to partner probably later in the year.

T. Rowe Price funds and accounts led the round, with Farallon and Wellington Management Company jumping in alongside. Existing investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital and an entity associated with SVB Leerink came back to stay in the syndicate. Cowen served as exclusive placement agent for the offering.

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With $110M to add to the bankroll, Generation Bio sets its sights on engineering a revolution in the gene therapy field - Endpoints News