Startups, Venture

A fast-moving entry in the gene editing race is speeding to the clinic, fueled by an $83.5M round

The last time I talked to Arthur Tzianabos a little more than a year ago, the CEO at Homology Medicines had a team of about 10 and a new approach to in vivo gene transfer and gene editing that was still in preclinical liftoff mode. Today, Tzianabos is whipping the cover off a whopping $83.5 million venture round with some A-lister support so his team of 40-plus can take their work into humans for the first time.

“We really have made a tremendous amount of progress,” Tzianabos tells me by way of catching up.

There’s a lead indication now — an inborn error of metabolism involving the liver is as specific as he wants to get right now — with a long list of preclinical projects in CD34 diseases, cystic fibrosis, CNS diseases, Duchenne muscular dystrophy and so on. When you’re a preclinical biotech working on the foundation for a platform tech that seeks to edit and correct genes using your own adeno-associated virus vectors, you can think about what you want to keep and what you want to partner.

Homology is one of the upstarts traveling one step behind the pioneering crews that booted up gene editing in a big way. That group includes Editas, CRISPR Therapeutics and Intellia — which all made virtually overnight debuts onto the public markets with a set of IPOs that attracted a tremendous amount of investor attention — and which sagged as the long clinical road to an actual therapy came into view.

These days, the pioneers also are spending a significant amount of time wrangling over patents as they work to get into the clinic. It’s no easy task birthing a new technology, as Editas found out when it had to delay its timeline recently in order to work through a delay on the manufacturing side.

Tzianabos and his team are learning from the leaders, and may also be moving faster toward the clinic than the out-front rivals know.

“We want to be there in a short period of time,” says the CEO about human trials. (No, he isn’t being more specific than that.) He has plenty of cash now to get through a proof-of-concept trial, and a chance to sign up a partner or two to help expand the pipeline work more quickly.

Tzianabos gives his Bedford, MA-based biotech high scores for their preclinical effort on fine tuning gene transfer and editing, starting with a page out of the book of nature on the way DNA is repaired to point to an in vivo approach that can be a simpler and more direct means of potentially curing genetic diseases.

But he’s also starting to look at what needs to happen on manufacturing to prevent future foul-ups.

“I think people got a little over their skis on that point,” he says about the field. And he has built up internal and external manufacturing ops.

“CMC, biologics manufacturing is complicated anyway,” says Tzianabos, pointing to the crisis that Genzyme went through before Sanofi acquired the company. The new gene editing companies are coming straight out of academia, he adds, and that kind of tech always takes time to industrialize.

At Homology, says the CEO, “that was one of our main focuses right out of the gate.”

Add it all up, he says, and “I would put us not too far behind where these guys (the pioneers) are. That’s what attracted this team to the technology.”

It is also what attracted Deerfield, which led the round. New investors include Fidelity Management and Research Company, HBM Healthcare Investments, Maverick Ventures, Novartis, Rock Springs Capital, Vida Ventures, Vivo Capital and Alexandria Venture Investments. Existing investors 5AM Ventures, ARCH Venture Partners and Temasek also jumped back in.

As of now, Homology has raised a total of $127 million in a little more than a year, offering another example of the kind of funds that are being spent to pursue a radical new technology. It’s still early stages, with years of clinical work ahead, but Homology is moving fast.


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RAPS Regulatory Convergence 2017