Approval gives patients access to new targeted option

New indication adds oral option for young patients

Acquisition adds new frataxin‑targeted modalities

Phase 2 data show strong safety and immunogenicity

Half of patients remained progression-free without maintenance

Partnership brings key medicines to eight markets

Company prepares seamless transition into phase 2a development

Nations commit US$240 million to CEPI’s 2027–2031 strategy

Grants support studies into restorative sleep and disease mechanisms

New option for patients with systemic lupus erythematosus