The future waves of gene therapy could look quite different from the gene replacement offerings currently on the market, Forbion reckons.
It’s the key idea behind VectorY, a startup seeded by the European VC firm that’s out to develop vectorized antibodies for muscular and neurodegenerative disorders.
Voyager Therapeutics, which briefly pushed the technology to the limelight with its short-lived AbbVie collaboration, is among the “several other companies working on it too, but definitely it’s a pretty relatively empty space,” said Marco Boorsma — a general partner at Forbion who’s stepping in as interim CEO.
At the intersection of viral gene therapies and antibodies, vectorized antibodies are AAV vectors that carry genes encoding for antibodies. With some capsid engineering and “interesting efficacy enhancing tools,” they promise to treat a wider range of diseases and do a more thorough job at, say, cleaning up misfolded proteins inside and outside of cells than either of the traditional modalities can.
“With naked antibodies you hardly get into the brain,” Boorsma said. “Also difficult to get into muscle.”
Building the company required expertise in both gene therapy and antibodies, he added, and VectorY co-founders bring a diverse skill set: Chairman Carlo Incerti is an operating partner and veteran of Sanofi Genzyme; Pavlina Konstantinova spent some years at uniQure (another Forbion-backed gene therapy venture) before taking up the CSO post; COO Anthony Newcombe learned the ins and outs of manufacturing through stints at Pfizer, GlaxoSmithKline, Pfizer and Samsung Biologics; and Barbara Sanders, the VP of vector development, cut her teeth at J&J and held a similar role at uniQure.
“I have been working in the gene therapy field for over 30 years, and, from experience, know that accurately targeting the right cells with the transgene has always been of paramount importance,” Incerti said in a statement. “Using viral vectors developed at VectorY to deliver the genetic sequence for therapeutic antibody expression in target cells has the potential to address some of the key shortcomings of intravenously injected molecules.”
With access to the deep pockets of Forbion — which recently added a $557 million new fund — one thing the Amsterdam-based biotech has decided to do early is to set up its own manufacturing facilities.
“Definitely CDMOs are expanding capacity, they are developing well also in gene therapies,” Boorsma said. “Still I think they are not there where we think they should be to serve a company like VectorY.”
The issues can range from varied standards to waiting times up to one and a half years that are “simply too long for a young biotech company.”
Having started operations last October, the team is now aiming to double the headcount to about 40 by the end of the year.
“VectorY is working very hard, but also the field is working on that very hard,” he added. “The first wave of gene therapies was more in kind of repairing genes, now you also see a lot of capsid engineering to get your payload to the right tissues, and then of course also manufacturing that’s being scaled up […] being more robust.”