It began with one individual.

In 2018, a young girl in the USA called Mila became the first person in the world to be treated with a medicine designed for one person - named milasen. Inspired by her example, a group of aligned stakeholders including government, academics, industry and patients, came together in the UK to build on Mila’s experience and to form the Rare Therapies Launch Pad (RTLP).

As we face a global crisis of genetic disease, affecting millions of children alone with rare conditions, we have a moral imperative to ensure equitable access to 21st century technologies, irrespective of the number of patients that could benefit from them.

Founded on the principle that the UK is well suited to model this new system of individualised medicines, the RTLP was established in 2023 as a ground-breaking pilot programme to help put in place the processes and infrastructure to bridge the gap between the patients and the science.