Delivering a successful launch:
a world's first gene therapy
for an ultra-rare condition

The challenges

The annual incidence of ADA-SCID  is estimated to be between 1/200,000 and 1/1,000,000 live births, with  both males and females affected 1. Mapping out and predicting potential diagnosis, referral and treatment pathways was a fundamental part of the landscaping process, and engagement with all stakeholders (key treatment centres, KOLs and patient advocacy groups) was vital to build up a rational and meaningful model for what would be a brand new approach to treatment. Disparate stakeholder needs and attitudes were uncovered that needed to be addressed in the communications strategy.

    Work article

    Our solution covered research, strategic, creative and operational work 

    • Understanding and building the diagnostic and treatment pathways informed the structure of internal sales and medical teams
    • A bespoke training programme ensured in-depth scientific understanding with a strong patient focus
    • The launch plan was designed to maintain engagement across multiple touchpoints over two years
    • A creative and messaging platform was developed that has shown enduring value and impact
    • The communications plan focused on engaging and supporting  the multi-disciplinary team whilst addressing their varying needs 


    • Cross-functional collaboration ensured delivery of an integrated global launch across digital, live and face to face channels
    • The campaign was consistently implemented and addressed the disparate stakeholder needs and attitudes
    • All internal core KPMs for launch were achieved


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