Accelerating the Translation of Genomic Insight to Therapeutics

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There is no cure for many common, complex diseases. Moreover, the best treatments available for these diseases often fail to help patients manage their illness. Rising to this challenge, drug companies seek better insights into drug target biology so that they can identify better treatments. The increasing availability of high-quality whole genome sequencing data is fueling this deeper understanding. Importantly, drug targets with genomic validation result in a higher likelihood of a successful therapeutic development program.

Jeffrey Gulcher, MD, PhD
Jeffrey Gulcher, MD, PhD
Chief Scientific Officer and Co-founder
Genuity Science

Genuity Science is on a mission to provide deeper insight into the genomic drivers of disease. In multiple disease areas, they combine detailed clinical data from consented participants with their whole genome sequence data. Clinical data on disease subtypes and rates of progression enable the Genuity Science team to identify causal links to any differences in whole genome sequence found when comparing to non-affected individuals. These genomic variants then point drug companies to actionable targets.

Last month, Genuity Science announced a multiyear collaboration with Ionis Pharmaceuticals aimed at accelerating this transition from target to therapeutic in multiple disease areas. Ionis is a biotech company located in Carlsbad, CA, that works to bring novel and transformative therapies to underserved patient communities.

GEN caught up with Leo Sahelijo, MD, vp of translational medicine at Ionis, and Jeffrey Gulcher, MD, PhD, chief scientific officer at Genuity to discuss the collaboration.


GEN: Leo, let’s start with you. How did this collaboration come to be?

LS: When Jeff approached me slightly more than a year ago, with a proposal to collaborate, I immediately thought that this would be a unique opportunity to generate novel data in a multitude of diseases. The uniqueness of this collaboration really is based on combining Ionis’ technology platform, which gives us an opportunity to target the human genome in its totality in principal, with Genuity’s deep experience in whole genome sequencing and the associated bioinformatics.


GEN: It sounds like you have very complementary expertise. Can you explain further?

JG: At Genuity, we look at the entire genome, which includes 60,000 different genes, only 20,000 of which encode proteins. And only about 2,000 of those genes and coding proteins are amenable to traditional pharmaceutical therapeutics of small molecules and antibodies. So that leaves 58,000 other genes that are not amenable today with traditional therapeutic technology.

Ionis, on the other hand, can cover all 60,000 genes with its antisense technology. So, if we find some very important genes for a certain subset of patients in a disease, unfortunately, those patients may not be well treated by traditional pharmaceutical technology, but they have a chance because of Ionis’ much more broad technology.


GEN: And what do each of you see as the key outcome of this collaboration?

JG: The Ionis technology wedded to Genuity Science’s technology really opens up the landscape of therapeutics to perhaps treat even more patients effectively. We see this collaboration as a way to accelerate movement of gene discoveries that advance to clinical trials and eventual drug development.

LS: The abilities of Genuity in combination with our technology platform at Ionis provides us with an opportunity to not only pursue novel diseases and specific subcategories of these diseases, but also to offer true alternative therapies to existing modifiers of diseases and have therapies available for diseases that today have no treatment. So, I think it’s the combination of having improved alternatives and often new therapies for communities that might have been left out of therapeutic approaches today.


To learn more about the research, and clinical partnerships Genuity Science is developing around the globe, visit them at