One of Vertex’s key promises to investors and patients has been that it will deploy its success in cystic fibrosis — which finally turned the 30-year-old biotech into a profitable business three years ago — to fund more R&D projects with innovative technologies. It has propelled them to dive into gene editing, team up with CRISPR Therapeutics to test what could be a pioneering treatment for sickle cell disease.
With a new collaboration with Atlas-incubated Kymera Therapeutics, Vertex $VRTX is putting its foot down in the burgeoning protein degradation field.
Vertex is infusing $70 million — part cash, part equity — to bankroll a 4-year research odyssey at Kymera to generate clinical candidates against up to six targets for “serious diseases.” A decision to license would kick off additional milestone payments racking up to more than $1 billion.
While both partners declined to discuss the targets, Vertex spokesperson Heather Nichols said they will be consistent with its focus on “validated targets that address causal human biology.” Nello Mainolfi, CSO and co-founder of Kymera, told me they bonded over a shared eagerness to identify the right responder patient populations and targets.
Hot on a trail blazed by the likes of C4 Therapeutics and Arvinas, Kymera boasts of a platform built on a complex approach using informatics-driven target identification coupled to predictive modeling, new degradation tools as well as novel E3 ligases and ligands. It promises to deliver a more sophisticated way to tag disease-causing proteins for disposal — one that has garnered the interest of GlaxoSmithKline.
“The beauty about targeted protein degradation is you [combine] the flexibility of small molecule, power of genetic knockdown, and the precision of genetic manipulation in one single technology,” Mainolfi said.
Its in-house pipeline so far has focused on oncology and inflammation/immunology. This new partnership will help broaden the platform’s application beyond cancer, CEO Laurent Audoly said, a testament to the disease agnostic potential of Kymera’s tech as well as their ambition to grow into a fully integrated shop.
Meanwhile, Vertex’s undertakings outside of CF have spanned pain, blood diseases and a genetic disorder dubbed alpha-1 antitrypsin deficiency (which causes lung and liver damage).
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