Vertex is expanding its investments across advanced therapies again, R&D chief David Altshuler adding a second in vivo gene-editing deal to its pipeline.

Novartis is once again looking to be a pioneer in cell and gene therapy, though this time is selecting a potentially less complex administration method.

Companies such as Novartis, Roche or Takeda are heavily invested in the advanced therapeutics field, usually spreading their bets across multiple modalities. The remaining challenges offer opportunities for the next wave of innovation to transform the field once again and make a dent in the larger market of common diseases and solid tumors.

AstraZeneca is preparing a US filing for hereditary transthyretin-mediated amyloid polyneuropathy for eplontersen, which it hopes will compete with Pfizer and Alnylam's therapies in that rare disease space.