LifeArc and Action Medical Research Fund

The fund wants UK biomedical research projects that have moved beyond basic understanding of a rare disease mechanism into developing an intervention -- a therapeutic, diagnostic or device -- for infants, children and young people with a rare disease. Applications are expected to address a significant unmet need, rest on a strong scientific rationale, be target-driven with clear milestones, and have a defined route to patients. Any modality is eligible, including small molecules, antibodies, cell and gene therapy, vaccines, diagnostics and psychological interventions; past funded projects have ranged from small-molecule drug development for Duchenne muscular dystrophy to a biomarker-based diagnostic for a childhood heart condition and a stem-cell gene therapy for Hunter syndrome.