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Biotech millionaire funds free drugs for ultra-rare diseases

The n-Lorem Foundation seeks to provide therapies for ultra-rare diseases free of charge
  1. Mark Zipkin

    1. Haddon Township, NJ, USA.

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The launch of the n-Lorem Foundation by Ionis Pharmaceuticals’ Executive Chairman Stan Crooke last week began to crystallize the drug development pathway for ultra-rare diseases, but it also brought to the forefront a question raised in recent years with the maturation of genomic screening and antisense oligonucleotide (ASO) therapies: how can patients with ultra-rare diseases afford n-of-one therapies? The n-Lorem Foundation intends to form the connective tissue between patients, researchers, facilities and regulators needed to supply individual patients with RNA therapies designed to treat their own ultra-rare disease — at no cost. The nonprofit launched with $1.5 million from Crooke and his wife Rosanne, a senior strategic advisor at Ionis; $1.5 million from Ionis; and $1 million from Biogen.

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doi: https://doi.org/10.1038/d41587-020-00001-3

Updates & Corrections

  • Correction 03 June 2020: In the original version of this article, Timothy Yu’s comment that "academics are not well positioned to lead the charge for n-of-one therapies" is inaccurate. Yu thinks that academic medical centers will play a key role in developing n-of-one therapies, although they aren’t positioned to scale up yet. Also, the cost Yu mentions for oligonucleotide synthesis refers to research grade oligonucleotides, not clinical grade ones, which are more expensive. The text has been corrected to address these errors.

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