People. Pipeline. CMC Strategy.
We are built on 3 organizational pillars that are the foundation of everything we do. These pillars embody our mission—to make our vision of what is possible for precision genetic medicine a reality.
We’ve brought together pioneers in genetic research and experts in gene therapy development and commercialization who share the same bold vision and unwavering commitment to discovering and developing innovative genetic therapies and bringing those therapies to patients
We are rapidly building a diverse product pipeline of innovative precision genetic therapies combined with a novel immunomodulatory approach to target debilitating genetic diseases for which the unmet medical need is high and for which there are no approved therapies treating the underlying disease
Chemical, Manufacturing, and Controls (CMC) Strategy
Our CMC strategy embraces the challenges of commercializing gene therapy products. We are building a robust, scalable manufacturing process and network that is flexible and agile, allowing us to produce our precision genetic therapy product candidates efficiently and with breakthrough yields
We are headquartered in Cambridge, MA
We have discovery & manufacturing laboratory facilities in Gainesville, FL
We are leveraging a strategic partnership with the University of Florida
We have additional facilities planned for the Boston area