Biotech, Analytics Companies Partner on Drug Discovery

(National Human Genome Research Institute, NIH)

1 Oct. 2020. A biotechnology company and genomics data science enterprise agreed in a multi-year agreement to discover new therapies for up to 20 diseases. While the deal calls for a series of payments from Ionis Pharmaceuticals Inc. to Genuity Science over the course of the agreement, dollar amounts of those payments were not disclosed.

Ionis Pharmaceuticals, in Carlsbad, California develops therapies that target ribonucleic acid or RNA, nucleic acids that carry instructions from genetic codes in DNA to cells for production of proteins. The company’s antisense technology platform uses short DNA or RNA sequences, called oligonucleotides, designed to complement a specific gene sequence. Ionis says it designs antisense drugs to interact precisely with a specific sequence of RNA, with many of the therapies in its pipeline binding to messenger RNAs, or mRNAs, and blocking the production of disease-causing proteins.

Ionis has approved therapies for spinal muscular atrophy and polyneuropathy in adults with hereditary transthyretin amyloidosis, a rare genetic disorder. The company says it has some 40 treatments in its pipeline designed to address neurological, cardiovascular, infectious, and pulmonary diseases.

Genuity Science in Boston designs medical analytics based on population genomics. The company says its Genomically Ordered Relational Database, or GORdb, can take in and process next-generation or high-throughput sequencing data at population scale. In addition, says Genuity, its platform contains both genomics and phenotype, or traits, data at this large population-wide scale to enable clinical and genomic analysis over extended periods of time. The company’s GORdb database was originally designed at deCode Genetics, in Reykjavik, Iceland, a division of Amgen with genomics data from about half of the country’s population and genealogical data from Iceland going back about 1,000 years.

The agreement calls for the companies to collaborate on discovery of therapies for up to 20 new diseases. The work is expected to combine Genuity Science’s whole genome and deep trait analysis with Ionis’s antisense technology to better define and understand disease mechanisms for new drug discovery. The combined analysis is also expected to help speed drug development by providing more direct connections between targets and therapeutics.

Under the deal, Ionis is providing Genuity Science with an initial payment and Genuity is eligible for development milestone payments and royalties on sales of products from the collaboration. Dollar amounts of those payments were not disclosed.

Rob Brainin, CEO of Genuity Science, says in a company statement, “Combining the scale and depth of our datasets and our analytical expertise with Ionis’ pioneering antisense technology can more rapidly translate information into insights with potential to deliver breakthrough therapies to even more patients who need them.”

Brett Monia, CEO of Ionis adds, “The collaboration with Genuity Science gives us the potential to significantly increase our discovery opportunities and better understand the complexities of human diseases based on genetics.”

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