National Ultra Endurance

Ultra rare diseases list

Ensemble Therapeutics is putting its discovery engine to work on ultrarare diseases.

The Cambridge, MA-based biotech will explore its rapidly growing library of 10 million macrocycles to see what it can whip up for Alexion's pipeline. The biotech has developed the "Ensemblin" platform, and the booming Alexion ($ALXN)-which sells the world's highest-priced therapy-has agreed to a slate of unspecified milestones and an upfront followed by research support to see whether it can mine the technology for some additional development programs.

Just last fall Boehringer Ingelheim stepped up with a $186 million deal to join the list of Ensemble's partners, a marquee group that also includes Pfizer ($PFE), Bristol-Myers Squibb ($BMY) and Genentech.

These companies have come calling as Ensemble proselytized its work on mid-sized molecules that are billed to do orally what biologics-like antibodies-accomplish only after being injected into a patient. The big idea here is that Ensemble has figured out a new approach to synthetically create an easy-to-take regimen of therapies that have been beyond the reach of the industry. It's primarily focused on elusive protein-protein and protein-peptide interactions, with a big interest in oncology and immuno-inflammatory diseases. And it's still very much early-on in terms of clinical development work.

CEO Mike Taylor

Ensemble was founded on the work of Harvard professor and Howard Hughes Medical Institute Investigator David Liu, who's still actively engaged with the biotech. CEO Mike Taylor, a former senior vice president of R&D at Pfizer, says their success in recruiting a steady stream of high-profile collaborators willing to provide research support in their deals has helped grow the staff into the low 30s without requiring a lot of venture support. That staff number should rise modestly as the work expands on this "middle space" opportunity.

"These deals have been very useful in terms of providing non-dilutive funding, " says Taylor, who joined the company in 2007, the same year Ensemble raised its last round.

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Popular Q&A

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What is the top 10 list weirdest named diseases?

Moebius Syndrome (lack of developed facial nerves), Ondine's Curse (difficulty taking in O2), Elephantiasis, Werewolf Syndrome, Trichotillomania, Pica, Capgras Syndrome, Progeria, Vampire Disease, & Jumping Frenchman Disorder are some weird diseases.

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