We will be integrating the Rare Therapies Launch Pad (RTLP) into our organisation, marking an important next step in efforts to accelerate the development, delivery and access of individualised medicines for people living with rare diseases.

The RTLP is a UK-led, multi-stakeholder initiative established by partners including Genomics England, The Association of the British Pharmaceutical Industry, University of Oxford, Oxford-Harrington Rare Disease Centre, Mila’s Miracle Foundation, Great Ormond Street Hospital, University College London and the Yu Lab. It has played a central role in bringing together clinical, regulatory, academic, industry and patient expertise to address one of the biggest challenges in rare disease: how to turn advances in genetics and molecular science into treatments that can be developed and delivered, at scale, for individual patients.

As part of this next phase, the programme will become the LifeArc Rare Therapies Launch Pad, which will provide a long-term institutional home to build on the progress achieved to date and support the continued development of a more coordinated UK pathway for individualised medicines.

Our established expertise in rare disease translation, underpinned by our scientific, clinical, and convening capabilities, drives promising research towards patient impact by shaping and implementing innovative pathways for rare disease therapeutics development and access.

“Individualised medicines hold enormous promise for people living with rare diseases, but delivering them in practice requires coordinated effort across the whole system.

“The Rare Therapies Launch Pad has shown what can be achieved when partners come together around a shared goal. Bringing this work into LifeArc provides the platform, capability and long-term support needed to build on that progress and accelerate patient impact.”

Sam Barrell, CEO of LifeArc

The RTLP has established strong momentum across the UK rare disease ecosystem, including supporting the development of a world-first regulatory master protocol for individualised medicines, approved by the Medicines and Healthcare products Regulatory Agency. This approach shows how flexible regulation can enable the safe and efficient development and clinical translation of individualised medicines.

“The RTLP has built a strong collaborative foundation over recent years, and this next step ensures that momentum continues. “Within LifeArc, the programme is well placed to further develop the pathways needed for individualised medicines and to deliver meaningful impact for patients and the wider healthcare community.”

Dr Dan O’Connor, Chair of the RTLP Board

We will provide dedicated programme leadership and coordination, while connecting the initiative more closely with our existing work in rare diseases therapeutics development, clinical trials, health data and regulatory engagement. We will be recruiting a dedicated director to lead the programme, convene partners across the system and support progress on key challenges including regulation, clinical development, patient identification and wider access.

The LifeArc Rare Therapies Launch Pad will continue to work closely with the established RTLP community and its working groups, building on the collaboration and momentum already in place while focusing on areas where it can have the greatest impact, with the aim of translating scientific progress and system-level change into patient benefit more effectively.

“The Rare Therapies Launch Pad was created to move beyond discussions and actually demonstrate the system that needs to be in place for patients to access individualised medicines at scale. “This next phase of the RTLP is critical as the UK regulatory framework opens up, and no one is better suited than LifeArc to ensure that key organizations work together with urgency to show the world how this can be done.”

Julia Vitarello, Founder and CEO of Mila’s Miracle Foundation, a founding partner of the RTLP