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This Biotech Startup Aims To Speed Up Drug Testing On Animals

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When developing new therapies for diseases, biotech researchers are often limited by two time-consuming steps: first, screening thousands of drug candidates in test tubes and second, taking the best candidates and testing them on multiple animals to make sure it’s safe and effective. Combined, these steps can be slow and expensive.

Today, a startup called Gordian Biotechnology debuted a technology that could make this process better for both animals and people. The San Francisco-based company has developed a new animal screening platform which allows multiple gene therapies to be tested at the same time with just one animal. Instead of the gene treatment being given to the animal and affecting an entire area of its body, Gordian’s innovation enables it to test a drug inside of a single cell. That means one mouse could potentially support the evaluation of hundreds of possible new gene therapies in a way that’s faster and impacts fewer animals with less risk of harm to them.

“We’ve got a platform that allows us to essentially parallelize the hardest part of the drug discovery process, the animal testing process,” Gordian CEO Francisco LePort told Forbes.

LePort, 38, cofounded Gordian in 2018 with Martin Borch Jensen, 38, naming the company after the mythical, impossible-to-untangle knot Alexander the Great unraveled by cutting it in half. The two met each other at a Russian bakery where a conversation about age-related diseases evolved into the creation of a company to combat them. Their idea was to develop a method for drug discovery that focused on how someone’s age increases their risk for disease, something they said typically goes unaddressed. They realized the key to addressing it were new advancements in gene therapies — which led them to finding ways to test drugs in animals more efficiently. The company has raised $60 million to date from backers like Founders Fund, Gigafund and Horizons Ventures and has a $170 million valuation, according to Pitchbook.

Gordian’s platform could represent a leap forward in drug screening. First, it lets researchers skip the time-consuming process of screening drugs in the lab. Second, it enables scientists to more cost-effectively use animals that are better stand ins for humans because of similarities in the underlying disease biology. An example: Horses can develop osteoarthritis as they age, just like people. That makes them a good testing ground for arthritis drugs. But horses are expensive — especially if they are used to test only one drug. With Gordian’s technology, LePort estimates researchers could explore the effects of up to 60 different gene therapies on each of a horse’s joints. Better still, they can often be tested on an animal “without actually affecting the organism,” LePort said.

The company is able to do this by targeting just a single disease-affected cell in each animal with a cell therapy. This is a similar process to lab screening for drugs, where a treatment might be tested on a single cell in a test tube. But unlike a test tube, it has the advantage of being part of a whole living being, so the effects of the drug better mimic what would be seen in a real patient.

“We want to answer what happens therapeutically when everything is working together,” Jensen said.

After the testing is done, the cells are removed from the animal and analyzed by the company’s AI-assisted software, which uses past clinical data to predict whether a drug is a good candidate based on its activity in each cell. Ultimately, the process means that the animal’s overall health and wellbeing is not impacted.

Gordian said that it’s been able to validate its platform through several experiments. For example, in a mouse that had a common liver disease, it tested 50 different gene therapies, 16 of which have already existing clinical data. The company’s program was able to correctly identify 13 of those 16 therapies as potential treatments. The company saw similar accuracy for potential treatments of osteoarthritis in horses.

Moving forward, Gordian aims to use its drug screening platform to discover new potential treatments that it can move forward into clinical trials itself. Its initial focus is on fatty liver disease, osteoarthritis, and heart failure. It also plans to partner with other pharmaceutical companies to leverage its data into other potential drugs for diseases related to aging.

“The mission is to cure as many of these diseases as we can,” said LePort. “We see this as a universal mission because all of us have personal reasons for wanting to have something for these diseases, either for ourselves or for anyone that we love.”

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