A gene therapy innovator goes from closet to clinic with Big Pharma support
CEO: Annalisa Jenkins
Based: Cambridge, MA
Clinical focus: Gene therapy
The scoop: Fidelity Biosciences doesn't mind being a late adopter. The biotech investor, known for its reticence to talk too loudly about its deals, watched the boom and bust of early approaches to gene therapy, content to sit on the sidelines as a generation of research projects toiled with mixed success. Three years ago, however, as the organization reassessed the field, gene therapy technology seemed to have turned a corner, Fidelity Executive Director Dr. Thomas Beck said. So, partnering with the pioneers at ReGenX, Fidelity pieced together the team and technology that would become Dimension Therapeutics, an emerging biotech with eyes on hemophilia and a pair of banner co-signs from the likes of Bayer and OrbiMed Advisors.
What makes Dimension Therapeutics Fierce: Hemophilia is a rare, inherited disease in which patients don't produce enough of the proteins needed to form blood clots, putting them at risk of life-threatening bleeds. As it stands, the multibillion-dollar market for hemophilia treatments is populated by so-called factor-replacement therapies, intravenous treatments that provide recombinant copies of those missing proteins to encourage coagulation. Such medicines are generally effective, but they have to be taken as often as three times a week, burdening patients with a lifetime of injections.
That's where Dimension comes in. The biotech is developing gene therapy approaches to the disease, treatments that promise to deliver corrective coding that will reverse the imbalances at the heart of hemophilia and spur the liver to resume churning out the proteins needed for healthy clotting. The company's delivery technology, licensed from University of Pennsylvania gene therapy trailblazer James Wilson and his compatriots at ReGenX, uses a harmless adeno-associated virus (AAV) to deliver functional genes that can replace or knock down the errant ones that lead to hemophilia A and B.
Dimension came out of stealth mode last fall with an undisclosed Series A courtesy of Fidelity, and, just 7 months later, it had picked up a Big Pharma partnership to jump-start one of its programs and a sizable venture round to get working on the rest.
First, VC giant OrbiMed joined up with Fidelity to invest $30 million in Dimension's ongoing R&D projects, with OrbiMed partner Rishi Gupta touting the biotech's AAV technology as the best in the industry. Then, Bayer stepped in to echo that sentiment, shelling out a $20 million upfront payment and promising another $232 million in milestones to get its hands on Dimension's hemophilia A program.
That's the largest single-product deal in gene therapy history, according to Beck, who served as the biotech's interim CEO upon its launch.
"One of the things that has become incredibly clear is that the players in the space understand that they need a position in gene therapy, and the Bayer deal makes it clear that we're really taken seriously," he said.
Combined, the two deals give Dimension plenty of breathing room to advance its in-house candidates, including a hemophilia B treatment and some preclinical products targeting undisclosed genetic diseases. Now, Dimension plans to get its top prospect into the clinic in the next 12 months, unveiling its third project by year's end and further expanding its pipeline in 2015.
To get there, Dimension has assembled a team its founders believe can make it a leader among next-gen companies in the space, including Genzyme's former head of gene therapy R&D and a few of the earliest practitioners of AAV delivery. Rounding out that list is Beck's replacement, former Merck KGaA R&D boss Annalisa Jenkins, whom the biotech appointed as its full-time chief executive last week.
As the interim CEO steps aside, he's confident he and the biotech's founders have put their seedling in a position to succeed.
"Personally and from Fidelity's point of view, we do believe that generation two (of gene therapy) is ultimately going to yield extremely important products for patients," Beck said, and Dimension's blazing developmental pace only serves as further encouragement.
"To go from a couple of us sitting in a closet to where the company is today, I'm very, very proud of it," he said.
Investors: Fidelity Biosciences, OrbiMed Advisors
Dimension leaps into gene therapy action with $252M Bayer deal, $30M round
Fidelity bankrolls gene therapy startup with sights on hemophilia
-- Damian Garde (email | Twitter)