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We follow the science that will lead us to the best possible outcomes for patients.
We are a mutation-agnostic in vivo gene editing company striving to cure liver disorders with high unmet needs by harnessing new advancements in genetic medicine. Our in vivo insertion methods knock in healthy copies of disease-causing genes, offering long-term, stable expression of those genes in patients.
Our science is at the leading edge, and so are we. Our team of experts is here to transform lives and bring potentially curative treatments to patients in need. Great work takes a deep level of collaboration at all levels, and we strive every day to foster an environment that inspires innovation, execution and an unrelenting commitment to patients.
We are built on the world-class translational engine at the University of Pennsylvania’s Gene Therapy Program (UPenn GTP). The work executed by this group of world‑renowned gene therapy experts on our pipeline provides a substantial developmental head start – meaning treatments in the hands of patients as quickly as possible.
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