LifeArc Rare Disease Clinical Trials Programme

Funding for researchers at non-profit research institutions and SMEs in the UK and overseas to undertake early-stage clinical trials to develop therapeutics, advanced therapies, medical technologies, devices and diagnostics for rare diseases.

LifeArc maintains several Translational Challenges to address unmet needs in specific medical fields (eg antimicrobial resistance, chronic respiratory infection, emerging viral threats) and disease areas (motor neuron disease, childhood cancer, neglected tropical diseases) where the translational infrastructure needed to advance promising scientific discoveries for patient benefit is currently lacking. LifeArc makes available funding, expertise and support to aid the development and translation of promising therapeutics, diagnostics and medical technologies.

Within the framework if its Translational Challenge for Rare Diseases, LifeArc has established the Rare Disease Clinical Trials Programme to support the clinical progress of life-changing innovations with potential to address unmet needs for people suffering from rare diseases. The programme offers significant funding to support early-stage clinical trials that will deliver high-quality efficacy and safety data to advance rare disease innovations that demonstrate strong scientific rationale and potential to impact on patients within three to five years.

Potential interventions under investigation can include:

• Therapeutics (including repurposed drugs) and advanced therapies (including gene and cell therapies).
• Medical technologies or devices.
• Diagnostics that demonstrate potential to improve decision-making and outcomes.

Support is aimed at well-developed clinical-stage projects, particularly those that are ready to progress to the next stage of clinical development within 12 months. Proposals will focus on proof of efficacy and delivering high quality clinical data. Projects that align with any of the LifeArc Translational Challenges (rare neurodegenerative, rare respiratory, childhood cancer), are especially encouraged.