An Oxford-based start-up has raised $30 million in funding to develop an RNA drug for liver disease and tested it on hundreds of donated human livers that survived in the lab.
Ocher Bio, in collaboration with the University of Oxford, has already analyzed data from over 1,000 diseased livers in an approach called deep phenotyping that combines imaging, genetic and biochemical analysis to discover the underlying causes of disease. doing.
The new funding will help us take the next step: transforming biological insights from early research into RNA-based medicines. Drug candidates will be tested on hundreds of live-maintained human livers in what the company calls its “liver ICU” in New York.
“Like many chronic diseases, we realized that the challenge in tackling liver disease was the lack of good models,” said Jack O’Meara, CEO of Ocher. . “We can solve liver disease in mice, but not in humans. Animal models do not replicate the disease very well.”
Instead, Ocher uses human organs for preclinical research. “Our liver ICU accepts donor livers that cannot be used for transplantation because they are mildly ill, old, or have adipose tissue,” he said. “We will keep him alive for more than five days on the OrganOx perfusion device while we study the effects of RNA therapy on liver physiology.”
Investors in Series A funding round following $10 million seed funding in June 2021 include Khosla Ventures, Hermes-Epitek, Backed VC, LifeForce Capital, Selvedge, AixThera, LifeLink, and biotech industry individuals It is included.
Ocher is one of many biotech companies that have come to see RNA technology as a fast and sure way to new drugs. It focuses on a category called siRNA (short interfering RNA) molecules, which have about 20 biochemical units representing letters of the genetic code. These block the transfer of certain genes in which the product is detrimental to health.
O’Meara said: “We can go from confirming a target in genomic data to synthesizing an siRNA drug candidate and testing it in human liver in days instead of months or years.”
The company initially aims to manufacture a compound that extends the life of transplanted livers, with preclinical trials in New York next year and clinical development in patients in 2024.
“We’re focusing on liver metabolism to make the donated organ more dependent on cellular stress,” said O’Meara.
Longer term, Ocher plans to develop drugs for chronic liver disease. Chronic liver disease is often associated with excess fat accumulation in the organ. It is her third leading cause of premature death in the UK and other developed countries.
Peter Friend, a professor of transplantation at the University of Oxford who has worked with the Ocher researchers but has no role at the company, said he was “very enthusiastic” about the “very exciting” work the company is doing. ‘ said he has
“The concept of first using an isolated organ to identify genes associated with disease and then silencing those genes via siRNA before entering clinical trials is very elegant to me. It seems to me.”
