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Dimension, Backed by Fidelity, Targets Hemophilia With Gene Therapy

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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.

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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.