The Cambridge, MA-based biotech develops non-viral gene therapies that can be dosed more than once for rare and prevalent diseases. It claims that its cell-targeted lipid nanoparticle (ctLNP) delivery system allows for repeat dosing of a genetic payload, called closed-end DNA (ceDNA), without stimulating an immune response, adding that it has produced ceDNA constructs of 12 kilobases which have almost 3x the capacity of adeno-associated virus (AAV) gene therapies.
Its pipeline consists of programs in liver and retinal disorders. All are preclinical stage.
2019 Financials: Operating Expenses: $62.3M (+67%); Net Loss: ($61.3M) (-71%); Cash Burn: ($40.3M) (-43%).