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Scipher Medicine, Ionis Pharmaceuticals Collaborate on Oligonucleotide Precision Drugs

NEW YORK – Scipher Medicine and Ionis Pharmaceuticals are together developing oligonucleotide precision drugs for various diseases.

In a collaboration announced Monday, Waltham, Massachusetts-based Scipher said that it will use its Spectra platform and proprietary repository of patients' molecular data to identify targets that Ionis Pharmaceuticals can advance into oligonucleotide therapies, focusing specifically on cardiovascular and central nervous system diseases. Scipher's platform relies on artificial intelligence and a network map of human biology to provide insights on how expressed proteins interact to cause disease phenotypes, which in turn can help identify molecular signatures underlying disease pathophysiology, predict drug response, and identify drug targets.

Under the terms of the agreement, the companies can leverage data from the Spectra platform to identify subgroups of patients most likely to benefit from treatment and employ patient stratification strategies in clinical trials that can shorten drug development timelines. Although the companies didn't disclose the financial terms of their agreement, Scipher said it is eligible to receive payments if it achieves certain milestones and royalties on net sales of any commercialized products that result from the collaboration.

"The collaboration with Scipher is part of Ionis' strategy to invest in precision genomic initiatives that enable new avenues for target discovery, which we believe can help accelerate the development of potentially transformative therapies for patients with unmet needs," Eric Swayze, executive VP of research at Ionis, said in a statement.

Carlsbad, California-based Ionis develops RNA-targeted drugs and has several treatments for cardiovascular and CNS diseases in its pipeline. Recently, the US Food and Drug Administration approved Qalsody (tofersen), which Biogen licensed from Ionis, for treatment of amyotrophic lateral sclerosis patients who have mutations in the SOD1 gene.