Solving CNS delivery will open big doors for epigenome editing: a Perspective

Epigenome editing companies could bring big benefits to patients and, in some cases, have entire markets to themselves, if they set their sights on neurological diseases. Most, however, are starting in the less risky territory of validated targets for liver diseases. If these companies aim to transition to areas of higher unmet need, they should not wait to begin investing in new delivery vehicles.

Platform companies with new modalities have always faced the question of whether to start with a de-risked target and indication, or to go for the technology's bigger promise of solving problems other modalities cannot...