Personalized antisense oligonucleotides ‘for free, for life’ – the n-Lorem Foundation.

The n-Lorem Foundation was established in 2020 with the mission to apply the efficiency, versatility and specificity of ASO technology to charitably provide experimental medicines to treat patients with ultra-rare diseases. n-Lorem is committed to providing these ASOs to the patients for whom they are designed ‘for free, for life’, focusing on severely debilitating or life-threatening conditions for which there are ≤30 patients who could benefit from a drug. This is both an ethical and practical approach considering the risk/benefit ratio, and is mandated by US Food and Drug Administration (FDA) guidelines5.