Dimension, Backed by Fidelity, Targets Hemophilia With Gene Therapy

10/31/13Follow @benthefidler

It wasn’t too long ago that the words gene therapy would send VC investors running for cover. Clearly, times are changing, because today, a new startup named Dimension Therapeutics has become the second company in the space to come out of the woodwork with significant venture backing since July.

Cambridge, MA-based Dimension was formed today through a collaboration between Fidelity BioSciences and Washington, D.C.-based Regenx Biosciences, and an undisclosed Series A round from Fidelity Biosciences. The company’s plan is a familiar one for those who’ve heard of Cambridge-based Bluebird Bio (NASDAQ: BLUE) and San Francisco, CA-based startup Audentes Therapeutics: Much like those two companies, Dimension wants to use gene therapy to tackle rare diseases. And just like Audentes, it’s using viral vectors created by Regenx to do it. But unlike those two, Dimension’s first program will be a treatment for hemophilia.

Regenx, the creator of a gene delivery platform, is a “very significant shareholder” in the company. It’s also giving Dimension “broad access” to its viral vectors as part of the deal, according to chairman and Fidelity Biosciences partner Ben Auspitz. Dimension also has rights to several unnamed rare disease programs, but Auspitz wouldn’t disclose which ones they are.

Dimension has turned to veterans of gene therapy and rare disease research to help with the effort. Sam Wadsworth, who ran all of Genzyme’s gene therapy programs, is its chief scientific officer. James Wilson, Regenx’s scientific founder and the director of the gene therapy program at the University of Pennsylvania, will lead Dimension’s scientific and technical advisory board. Ultragenyx CEO and former Biomarin chief medical officer Emil Kakkis is also an advisor.

Gene therapy, of course, has more than a checkered past. The idea is to use a modified virus to deliver healthy genes into a cell to replace faulty or missing ones. This, in theory, is supposed to attack the root cause of a disease and cure it for the rest of a patient’s life—potentially in a single treatment. But despite the billions of dollars poured into gene therapy research and startups in the ‘90s, no gene therapy has won FDA approval for commercial use. Safety problems and difficulties with the viral vectors delivering the new genes dogged the space.

Recently, however, sentiment has begun to shift. Netherlands-based UniQure won approval of the first gene therapy in Europe in November. Others have intertwined the idea of gene therapy with rare diseases, or orphan disease types that have no approved treatment. This is part of the reason Bluebird was able to carry out a successful IPO. And Audentes used a similar model to raise a $30 million Series A. It’s going after rare diseases like X-linked myotubular myopathy and Pompe disease.

“We have an approved drug now in Europe for gene therapy, we have a number of programs that have shown benefit, and I think that’s what’s driving people—small companies that are starting up, as well as Big Pharmas and Big Biotechs—to really look at this area again as a place that could provide a whole plethora of important medicines,” Auspitz says.

Now, Dimension is part of the mix. Fidelity Biosciences has wanted to get into gene therapy over the past few years, feeling the technology is now ready to match its scientific promise. But the investment firm preferred a platform approach, rather than just making a single drug, and it wanted to find the technology that best fit the subset of diseases it plans to target with gene therapy—one of them being hemophilia, according to interim Dimension CEO and Fidelity Biosciences executive partner Tom Beck.

That led it to Regenx. Beck notes, for instance, that part of the challenge of gene therapy is finding the right vector that gets the gene where you want it to go. He thinks Regenx’s platform has the best ability to do so among the existing approaches out there.

“If you want to introduce genes into the liver to make clotting factors, the vectors included in the Regenx portfolio are the best to do that based on their natural inclination to go to liver cells,” Beck says.

So Fidelity Biosciences constructed a deal to use Regenx’s gene delivery technology—a platform for delivering genes via ultra-small modified adeno-associated viruses (AAV)—to form the basis of Dimension (Audentes, too, is using an AAV vector from Regenx). It even invested in Regenx and took a board seat in conjunction with the formation of Dimension.

Dimension is starting with hemophilia for three reasons. First, there’s a substantial need for better treatment (patients have to get IV infusions of the clotting factor they’re genetically missing a few times a week). Second, a study published in the New England Journal of Medicine in 2011 showed one of Regenx’s AAV vectors was effectively used to perform gene therapy in patients with hemophilia. Lastly, there’s a product candidate in Wilson’s lab that Dimension thinks it can quickly move into development, Beck says.

“The idea here would be to use the AAV vector to introduce the gene into the natural source of the clotting factors, which for the most part is the liver, to spontaneously replace the deficient factor,” he says.

Of course, Dimension is only in its infancy. It’s a tiny company that still has to staff up and begin industrially developing the hemophilia product candidate before it can think about testing it (Beck wouldn’t give any timelines for when it would be ready for a trial). And Dimension isn’t the only one doing this. Baxter International (NYSE: BAX), for instance, is also chasing gene therapy products for hemophilia with an AAV vector approach. But Dimension is betting there will be “many winners” in gene therapy, even for hemophilia alone, and that the vectors it has access to will ultimately stand up to the competition.

“We know that this is a competitive field, we know that there’s published data, we know that there are existing programs, but we also feel comfortable that the product candidate we have in our hands is extremely competitive, and we’re optimistic it represents best in class,” Beck says.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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  • Lah99

    I think they are the third or fourth company going after hemophilia with a gene therapy and at least 2 of them are well downstream in development.