Real hope at last for people with rare genetic condition

27 March 2023

Britain’s AstraZeneca (LSE: AZN) has reported positive high-level results from the Phase III NEURO-TTRansform study of eplontersen.

Following a deal with USA-based RNA-targeted drug developer Ionis Pharmaceuticals (Nasdaq: IONS), the firm is developing the candidate for the rare disease transthyretin-mediated amyloid polyneuropathy (ATTR).

With new research coming to fruition, AstraZeneca is taking aim at a therapy area which analytics firm GlobalData estimates could be worth $14 billion by the end of the decade.

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