Gotham Therapeutics has raised $54 million from a syndicate of big-name VCs and strategic backers. The series A round sets Gotham up to advance small-molecule inhibitors of mRNA-modifying proteins as far as clinical proof of concept.
New York City-based Gotham is built upon research Cornell University’s Samie Jaffrey has performed into the link between post-transcriptional mRNA modifications and disease. The work has elucidated the effect the proteins and enzymes that add, remove and recognize post-translational modifications have on human health, opening the door to a new class of medicines that act on these processes and thereby treat cancers, autoimmune conditions and neurodegenerative diseases.
“What we're focused on are the enzymes and proteins involved in differential modulation of mRNAs, with regard to the status of methylation at unique sites within that messenger RNA, and how that plays a role … in a wide variety of different aspects of RNA biology,” Lee Babiss, Ph.D., the CEO of Gotham and former director of global pharma research at Roche, said.
Working with a small internal team and upward of 20 scientists at CRO partners, Gotham has spent the past 18 months validating data published by other groups. Gotham was able to reproduce around 85% of the studies, Babiss said, giving it the confidence to step up development of small molecules that act on the proteins and enzymes.
Versant Ventures seeded the early work and joined with other investors to put together the series A. Forbion, fresh from raising a $412 million fund, and GlaxoSmithKline’s SR One co-led the round with Versant. Celgene and Alexandria Venture Investments rounded out the syndicate.
The syndicate has set Gotham up to fund the next stage of its evolution.
“We hope to be able to transition from doing target validation using shRNAs, siRNAs, CRISPR/Cas and so forth, to actually trying to pursue the phenotypes that are emerging upon inhibition of these enzymes and proteins,” Babiss said.
Gotham hopes to get a drug as far as clinical proof of concept using the series A cash but will need to navigate hurdles in a new field to do so. Babiss acknowledges that the methylases and methyltransferases Gotham is interested in “are deemed to be very difficult to drug targets” but thinks the field has advanced to the point where this challenge can be overcome. Gotham is applying three different commercially available small-molecule screening platforms to targets of interest.
The R&D activity is taking place across multiple sites. Leveraging his contacts at Alexandria, Babiss secured wet-lab space in New York City. Gotham has also set up a subsidiary in Munich, where CSO Gerhard Müller and COO Birgit Zech work out of. And Babiss, the former CSO of leading CRO PPD, is continuing to rely on service providers for a chunk of Gotham’s research requirements.
Babiss sees advantages to the multilocation model. In New York, Gotham is well placed to work with the city’s academic researchers as they start to engage more with industry. Across the Atlantic, the German offshoot positions Gotham to keep tabs on the science in Europe.
Handled badly, these benefits could be more than offset by the challenges of keeping remote teams informed and pulling in the same direction. But Babiss thinks Gotham is on top of the communication challenge and poised to establish itself as a leader in a new field with broad therapeutic horizons.