Multi-platform, multi-disease to multiply the benefits of gene therapy.
Our technologies, advanced targets, and leading drug candidates are designed to treat more diseases for many more patients by overcoming important limitations of current gene therapy and gene editing approaches. We are pioneering gene correction across unprecedented areas, including proprietary approaches that persistently correct large genetic defects independent of gene size or mutation pattern without risk of permanent genomic alteration. Further, we are developing novel delivery technologies and proprietary cargo for non-viral gene therapy.
We have eight novel and mechanistically distinct gene therapy programs in development. Our proprietary platforms: