We have highlighted companies previously in this space that are involved in drug development based on epigenetics—the study of how changes in gene expression are caused by factors other than the underlying DNA. Today we focus on a company whose advances in the related field of epitranscriptomics—the science dealing with modifications to RNA and how those changes affect protein expression—have attracted serious interest from scientists and life sciences investors. Think of epitranscriptomics as the epigenetics of RNA.
As many diseases are caused by abnormal protein levels, understanding the regulation of protein expression in normal cells is a critical step in identifying novel therapeutic options. New York-based Gotham Therapeutics is developing a novel class of drugs that target RNA-modifying proteins—the machinery of epitranscriptomics—to develop new treatment options for patients suffering from cancers, auto-immune diseases and neurodegenerative diseases.
Gotham Therapeutics announced its launch today debuted today with a $54 million Series A financing co-led by founding investor Versant Ventures, Forbion and S.R. One. The round was filled out by Celgene
The company was founded in 2017 and seeded by Versant Ventures based upon the ground-breaking work of co-founder Samie Jaffrey, MD, PhD, the Greenberg-Starr Professor in the Department of Pharmacology at the Weill Cornell Medical College. Dr. Jaffrey and his team developed a technology to globally profile one chemically modified nucleotide demonstrating that chemically modified bases were widespread throughout the transcriptome, were dynamically regulated during development and disease, and have regulatory potential. This work is often described as the study that launched the resurgence of interest in epitranscriptomics.
While academic research and the 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. RNA modifications and their associated protein complexes therefore represent an untapped frontier that could yield new therapeutic approaches.
-Samie Jaffrey, MD, PhD, co-founder and scientific advisory board member, Gotham Therapeutics.
Gotham has built a platform to assess the impact of RNA-modifying proteins on disease biology and has developed small molecules designed against priority targets. The Series A proceeds will enable the company to establish clinical proof of concept and to invest broadly in a pipeline of preclinical candidates that have potential to treat diseases in the areas of cancer, auto-immune diseases and neurodegeneration that have proven to be resistant to classical approaches.
CEO Lee Babiss, PhD, was most recently President and Board Chairman of X-Chem and CEO of X-Rx. He was formerly President of Pharma Research at Roche
As we pursue several important targets, the information we glean will help us further validate and build our platform for increasingly broad applications. Our goal is to become the leader in drugging key proteins that modulate mRNA functionality, thereby impacting disease onset and progression.
-Lee Babiss, PhD, CEO, Gotham Therapeutics.
Versant Ventures is among a small number of venture firms, including Flagship Pioneering, that have opened offices in New York, which has only recently begun to attract biotech startups that may someday compete with counterparts in California and Massachusetts. New York City and the state have committed $1.15 billion in funding to the biotech industry, but it’s still a nascent effort, as noted by Corie Lok in Xconomy. “Gotham’s $54 million round is the largest Series A for a New York biotech startup this year.”
Gotham Therapeutics’ management will be at the 24th annual Bio-Europe, Europe’s largest life science partnering conference, in Copenhagen in November 2018, and at JP Morgan Week, the 37th annual healthcare gathering in San Francisco in January 2019.
Given the promise of Dr. Jaffrey’s research and the involvement of Dr. Babiss and Versant Ventures, Gotham Therapeutics is clearly a company worth watching. We’ll eagerly await news of developmental progress and clinical advancement.
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DISCLOSURE: The author has no positions or any other beneficial interest in the companies mentioned in this article
