RARE Daily

n-Lorem Foundation Partners with Alnylam to Expand Technology Access to RNAi Therapeutics for Patients with N-of-1 diseases

January 18, 2022

The nonprofit n-Lorem Foundation, which provides experimental antisense oligonucleotide medicines free for life to people with rare diseases affecting 30 or less people, said it entered a new partnership with Alnylam Pharmaceuticals.

“At n-Lorem, using ASO technology, we take advantage of three decades of experience, unparalleled quality control and integrated safety databases that are used to select frequency and dosage for each individual ASO therapy,” said Stanley Crooke, founder, CEO and Chairman of n-Lorem Foundation. “Alnylam’s RNAi therapeutic technology is the perfect complement to Ionis’ ASO technology, both are well understood, proven drug discovery technologies. Adding Alnylam as a partner is another step in the creation of a broad network of partner stakeholders committed to treating nano-rare patients.”

n-Lorem is addressing the challenges of developing ASO therapies for individual or small groups of rare disease patients. The effort is supported by a consortium of well-renowned and diverse organizations worldwide. Alnylam now joins n-Lorem’s other biotechnology and pharmaceutical partners, including Ionis Pharmaceuticals, Biogen, and Ultragenyx.

The organization’s clinical research partners include Charles River Labs, Korea Institute of Toxicology and Parexel. Its contract manufacturing partners include ChemGenes, Cytiva, and Nitto Avecia.

“Although we have the tools and expertise to understand nano-rare patients at a genetic level,” said Sarah Glass, chief development officer, n-Lorem Foundation, “the extreme rarity of these patients present insurmountable challenges to the current healthcare systems.”

Author: Rare Daily Staff

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