Ionis finds new partner in a young biotech and its hopeful founder – Endpoints News

Omri Gottesman came to the US from the UK 10 years ago, wide-eyed like many others.

The human genome project was long-complete and, with great fanfare, researchers were beginning to try and leverage those insights into treatments. Mount Sinai School of Medicine offered him a fellowship focused on just that: genomic medicine.

At the time, there was a lot of hope and hype that we had solved health, and would be able to discover and prevent everything and treat everyone, Gottesman told Endpoints News. It was the reason I came to Mount Sinai.

Over the ensuing decade, human biology proved much more elusive than they imagined. Genetics was more complicated than A-T and C-G. There were no easy hacks. Still, the field advanced and after 4 years at Mount Sinai and a stint at Regeneron, Gottesman decided he wanted to advance it on his own and build a new platform around the original mission: Analyze genetic information to home in on ideal drug targets. He called the new company Empirico and soon raised $30 million.

Its not a unique concept, but its one Gottesman pulled off well enough to today score a three-year partnership with Ionis Pharmaceuticals, one of the oldest and more successful genetics-based drug developers.

Ionis will make a $10 million equity investment into Empirico and offer $30 million in near-term operational and preclinical milestones, with another $620 million promised for clinical, regulatory and sales goals. In exchange, Empirico will make up-to 10 platform-discovered drug targets available to Ionis.

We spent about a year building the discover-first platform, Gottesman said. Part of the motivation for the Ionis collaboration and exploring collaborations more broadly is that we now have more potential targets than our capacity to develop therapies for them.

On the Ionis side, this is the latest collaboration for a biotech that has sought many since its founding and is trying to keep early-stage strong while some late-stage programs come to fruition. The deal also includes an option for Empirico to license a drug back from Ionis as it strives to become a target-to-market company.

Youve heard of platforms like Empirico before; its the mixed machine learning and wet lab approach taken by a growing number of biotechs. Gottesman and his team use their computer platform to interrogate genetic data for the roots of diseases. Should the computer find a mispelling or other variant in genetic code that appears to cause the malfunction, they then produce cell lines with that mispelling in the lab. Should those cell lines show the same malfunction as the disease, they then use their platform to identify the best form for a therapy.

Theyve used that method to build 5 preclinical targets, leading to a small molecule program for an upper airway disease and an siRNA candidate for glaucoma.

The targets for the Ionis collaboration will be those Empirico finds can be best-treated with their partners signature antisense technology. The biotech has already identified a couple, although they didnt disclosed them.

A 40-year-old concept, antisense therapies have recently brought some of the genetics-based health gains Gottesman and others sought. Along with a handful of approved drugs, last year a neurologist in Boston created a personalized therapy for a young girl with a never-before-seen genetic error. And yesterday, Ionis founder Stanley and Rosanne Crooke launched a charity to make those kinds of genetics-based ultra-personalized medicines broadly accessible.

Those bespoke therapies are arguably the apex of the vision Gottesman and others had 10 years ago. Its a vision he thinks has made more progress than some think, even if it hasnt brought any utopian dream.

Over the last decade, weve realized its really harder than it seems, he said. But actually, if you work at it, theres real value.

Social image credit: Ionis via YouTube