Fidelity bankrolls gene therapy startup with sights on hemophilia

Fidelity Biosciences is placing a big bet on gene therapy, funding the launch of startup Dimension Therapeutics with some licensed patents and a plan to develop novel treatments for hemophilia.

With an undisclosed Series A in its wallet, Dimension has struck up a deal with ReGenX Biosciences for access to that company's adeno-associated virus (AAV) vectors, part of a platform it calls NAV. The license and collaboration agreement gives Dimension preferred access to gene-delivery technology for its lead hemophilia program in addition to multiple rare-disease indications, the company said, fueling its plans to build out a full spate of gene therapy targets.

Thomas Beck, a Fidelity executive partner, will serve as interim CEO of the new company, joined by former Genzyme gene therapy R&D chief Sam Wadsworth as CSO and ReGenX founder James Wilson as chairman of its scientific advisory board.

Despite the field's promise, countless gene therapy projects have been derailed by struggles delivering unmutated genes to target cells, as in vitro successes have a habit of turning toxic in the real world. That's where ReGenX and its AAV technology comes in, Beck said.

"A core challenge for gene therapy has been the development of safe, efficient 'vectors' to enable delivery of the replacement gene to the correct cells and tissues of the patient to yield benefit," Beck said in a statement. "We believe ReGenX NAV vectors are the most promising approach for in vivo gene therapy and represent the potential for transformative therapy for patients."

That promise was enough to attract Fidelity, a heavyweight biotech investor known for its close-to-the-chest deals, and the firm is augmenting its stake in Dimension with an undisclosed investment in ReGenX, giving the company a broader platform to promote its gene therapy vectors, CEO Ken Mills said.

"We view the formation of Dimension as important in the evolution of ReGenX's mission to enable access to NAV vector technology through partnership and licensing to create successful new AAV therapeutics," Mills said in a statement.

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