Bepirovirsen made the hepatitis B virus undetectable in nearly 30% of patients in a Phase II study – but it remains to be seen whether this response be sustained once treatment with the novel antisense product ends.

Hopes rise as first four evaluable patients experienced no serious adverse events, and a sustained decline in level of key Huntington’s disease protein with the novel gene therapy product.

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.