Omega Therapeutics is taking it up a notch. The “genome-tuning” biotech raised $126 million to get its lead program, a treatment for liver cancer, into the clinic as well as to advance a clutch of other preclinical prospects including a treatment for acute respiratory distress syndrome (ARDS), a life-threatening lung injury that can result from COVID-19 infection.
The company is working on a new class of treatments called “epigenomic controllers,” which are designed to adjust the expression of target genes. Unlike gene therapies and gene editing approaches that switch genes on and off, cut out disease-causing genes or replace faulty genes with their healthy versions, Omega’s treatments tune gene expression up or down without making permanent changes to DNA.
Its work is based on “neighborhoods” of genes and their regulatory elements found in loops of DNA called Insulated Genomic Domains (IGDs). These loops occur because long strands of DNA need to fit into the cell’s nucleus. Omega's “epigenomic controllers” are designed to restore gene function to healthy levels by targeting the right place on specific IGDs.
The series C financing follows an $85 million B round in July 2020, which went toward identifying new targets and driving several programs across multiple disease areas through preclinical development.
“When we started, we needed to explore the depth of the platform—we didn’t want to pigeonhole ourselves,” said CEO Mahesh Karande. “That’s how we delineated eight programs at five different targets.”
Omega has started IND-enabling studies for its lead program, OTX-2002, an epigenetic controller programmed to control expression of c-myc, an elusive cancer-driving gene. It is developing the treatment for hepatocellular carcinoma, the most common form of liver cancer.
Unlike Gilead’s antiviral Veklury (remdesivir) and anti-SARS-CoV-2 antibodies from Eli Lilly and Regeneron, Omega’s COVID-19 treatment focuses on ARDS, which is caused by an inflammatory response in the lungs.
“We treat diseases created by functional or structural changes in IGDs and ARDS creates a functional change in a multigenic IGD where cytokines get supercharged and expressed,” Karande said. With its epigenomic controller, Omega aims to reduce the expression of those cytokines.
The company hopes the treatment will fill a gap in COVID-19 treatment.
“The standard of care [for ARDS] is quite insubstantial and largely palliative, involving mechanical ventilation and where possible, steroids,” said Omega Chief Scientific Officer Thomas McCauley, Ph.D.
Besides liver cancer and ARDS linked to COVID-19, Omega is focusing on regenerative medicine, inflammatory diseases, alopecia, non-small cell lung cancer and a group of skin conditions called neutrophilic dermatoses.
The financing will also bankroll a manufacturing scale-up as well as the expansion of Omega’s technology. As it develops drug candidates, it will continue to improve its platform, “taking the guesswork out of it” and making sure it can keep generating new drugs in a reliable and replicable way, Karande said.
As it continues its quest toward the clinic, Omega will build up its workforce, particularly its clinical organization and manufacturing unit.