Biotech looks to upset the preclinical model for liver drug development, and now it’s gearing up for the clinic

Liver disease is one of the hardest therapeutic areas to hit, and some researchers have pointed to a dearth of meaningful preclinical models during drug development. Now, a UK biotech has figured out a workaround for its own siRNA liver drugs, and it all starts with discarded human organs.

Ochre Bio announced the closing of a $9.6 million seed financing round, led by Khosla Ventures. Backed VC, Apollo Health Ventures, Selvedge, Hoton and Hermes Epitek also participated in the fundraising.

Ochre, which is led by co-founders Quin Wills and Jack O’Meara, uses a strategy called “deep phenomics” to scan donated livers and digitize that info into a crunchable data set. So far, the group says it has identified 200 new targets for liver disease.

Instead of working with animal models, the company goes straight to human livers that have been donated and deemed unfit for use in transplants. The group tests its siRNA therapies directly on those “explanted” livers maintained outside the body. But the company has more planned: By 2022, the biotech says, it will have developed a completely in silico liver that can be used to digitally perturb a range of genes to identify new targets at an even higher scale.

While Wills was working as the head of cellular and systems genomics for Novo Nordisk, he was pitching a pared-down version of the work Ochre is doing, and he increasingly realized that discovery in the space was going the same way as a lot of cardiometabolic diseases.

“You can find a new target if you want, but where do you go with this? Because there are very poor in vitro models, terrible animal models of unclear relevance, the clinical trials have unclear endpoints, lack of biomarkers,” he said in an interview with Endpoints News. “It’s just a messy messy space, and I don’t want to do target discovery for the next 10 years and not see it go anywhere.”

The company is aiming to head to clinical trials by 2023. This funding will help onboard five transplant center partners on discarded donor livers. The company will go through another round of funding before it heads to clinical trials, O’Meara said in an interview.

In a statement, Alex Morgan, partner of Khosla Ventures, said:

I have been impressed with the progress this very talented team has made to accelerate development of their deep phenotyping platform. The liver is a major convergence point in human metabolism and many aspects of health throughout the body, and the company’s approach of developing treatments that can improve the transplant success of livers and then using those insights to create therapies for the larger market of general diseases associated with the liver is a creative and unique approach.

The company, which relies on RNA, has been able to ride the success that has come with the technology’s journey into becoming a household name, Wills said, thanks in part to Moderna and Pfizer’s Covid-19 vaccines.

“It’s definitely helped in terms of perception, that’s very important for us in terms of the funding we’ve had, and it’s important in terms of people who’ve wanted to work with us,” Wills said. “If I had said even a year ago, people would have been far less receptive.”

The company also recently opened up a Taiwanese lab to study liver disease in Asia, in addition to its global network of transplant partners that it has established in Europe and North America.

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