Gotham Therapeutics launched in New York with a $54 million Series A financing. The round was co-led by founding investor Versant Ventures, Forbion and S.R. One. The investment syndicate included Celgene Corporation and Alexandria Venture Investments.
Gotham is built on the discoveries of co-founder Samie Jaffrey, a professor of pharmacology at Weill Cornell Medicine. The focus is on a subgroup of RNA metabolism called epitranscriptomics. This involves the biochemical modifications of RNA within a cell, which have an impact on gene expression.
The company used seed funding to build a platform that assesses how RNA-modifying proteins affect disease biology. They also developed small molecules against priority targets. The funds raised will be used to establish proof of concept and to invest in a pipeline of preclinical compounds.
“As we pursue several important targets, the information we glean will help us further validate and build our platform for increasingly broad applications,” said Lee Babiss, Gotham’s chief executive officer, in a statement. “Our goal is to become the leader in drugging key proteins that modulate mRNA functionality, thereby impacting disease onset and progression.”
The company’s board of directors includes Babbis, Carlo Rizzuto, Partner at Versant, and Jill Carroll, Principal at SR One. Jorge DiMartino, vice president, Translational Development Oncology at Celgene and Head of Celgene’s Epigenetics Thematic Center of Excellence, is on the board as an observer.
“While academic research and pharmaceutical industry focused initially on modifications of DNA, a growing body of evidence indicates that mRNA modifications help determine to which degree genes are translated into proteins,” Jaffrey stated. “RNA modifications and their associated protein complexes therefore represent an untapped frontier that could yield new therapeutic approaches.”
Gotham, located in New York City, also has a subsidiary near Munich, Germany. New York isn’t typically a big site of biotech startups, despite efforts to make it so. Xconomy notes, “Over the last several years, the city has made a concerted effort to bolster its startup biotech scene. City and state governments have committed more than $1 billion to growing New York’s biotech industry. Several startup incubators have sprouted up, and startup creators like Versant and Flagship Pioneering have arrived.”
Versant actually operates a biotech incubator in New York City, but Gotham is not part of that.
Another company, Accent Therapeutics, launched in May to target RNA-modifying enzymes in oncology. It took off with a $40 million financing. The investors were The Column Group, Atlas Venture and EcoR1 Capital. Accent, like so many other biotech startups, is located in Cambridge, Mass.
The U.S. Food and Drug Administration (FDA) approved the first-ever RNA interference (RNAi) drug, Onpattro (patisiran) in August from Alnylam Pharmaceuticals. Onpattro is approved to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare disease that affects about 50,000 people worldwide. In addition to polyneuropathy, a degeneration of peripheral nerves, it can lead to significant disabilities that include loss of ambulation that can lead to the inability to walk and a decline in cardiac function.