Few biotech executives explain their experimental therapies by comparing them to video games. Nan Ji, President and CEO of PAQ Therapeutics, did so enthusiastically and used a 40-year-old game. In the 1980s, Pac-Man was an arcade game. The children’s pockets were filled with dormitories. They lined up in a row to navigate the maze with the characters of the same name and swallow the pellets to earn points.
Similarly, PAQ Therapeutics is developing drugs that allow a component of the cell to chew on disease-related targets. More and more biotech companies are developing drugs that use one of the cell processing components to consume the protein in question. But Ji pointed out that protein is not the only part of a cell that can cause disease. Just as Pac-Man scores higher by eating fruits, bells, and ghosts, PAQ drugs can treat more diseases by consuming a range of targets, including lipids, mitochondria, and organelles.
“This is a very novel method based on new biology,” Ji said. “We are still too early, but we believe that the potential is huge.”
This Cambridge, Massachusetts-based startup is steadily advancing its research, enough to compete with Raising $30 millionThe Series A financing announced on Thursday was led by Sherpa Healthcare Partners.
The use of built-in components of cells to treat diseases is popular. More and more biotechnology companies are beginning to target protein degradation, directing disease-causing proteins to the part of the cell that processes old or damaged proteins. Ji knows this field very well. He was the head of chemistry at Kymera Therapeutics for three years, one of the companies that used this new method of treating diseases for clinical testing.
In 2019, when Ji was in Kymera, he read a paper Published in “Nature” Describes the use of autophagy, a process of processing other cellular components, as a way to treat disease. The research really focused on one protein, specifically the mutant huntingtin protein that causes the neurodegenerative disease Huntington’s disease. But Ji said he is particularly interested in the potential of autophagy to address non-protein targets.
“I immediately realized that this could provide an opportunity, which may not be available for targeted protein degradation,” he said.
Ji said he contacted Lu Boxun, a professor at Shanghai Fudan University and the last author of the “Nature” paper. Together, they launched PAQ to develop new drugs that utilize autophagy. In the body, autophagy is performed by lysosomes, which is a cell processing system different from protein degradation. Waste enters the lysosome through autophagosomes, which are the spherical components of cells. Ji said that PAQ is developing small molecules to accelerate the interaction between autophagosomes and cellular disease-related parts. The molecules being developed by PAQ are called autophagosome-binding compounds, or ATTECs.
The main disease target of PAQ is a neurodegenerative disease. The company did not disclose it, except that it is a genetic disease for which there are currently no treatment options. Ji said that the company’s small molecules can cross the blood-brain barrier to reach their targets. So far, the company has conducted pharmacokinetic and pharmacodynamic studies on animals. These tests show how the drug is absorbed by the body and the body’s biological response to treatment. Ji said he is particularly interested in the latter, because in this undisclosed disease target, pharmacodynamics is directly related to curative effect and may indicate expectations for humans.
PAQ is not the first biotech startup to use autophagy as a solution to neurodegeneration. Libra Therapeutics is developing small molecules that increase autophagy.San Diego-based biotech company Launched last year with US$29 million in A-series cash and amyotrophic lateral sclerosis as its main disease targets. QurAlis is a Cambridge start-up company, Raised $42 million In its Series A financing last year, it studied several different ALS methods, one of which is autophagy.
Other investors in PAQ’s new financing are Huagai Capital, MSA Capital and MRL Ventures Fund. Seed investors Nest.Bio Ventures and Jingwei China also joined them. Ji stated that with the new cash income, PAQ can continue to advance its leading projects in preclinical development. But he added that the technology is a platform that has the potential to support drugs targeting targets outside the central nervous system, and the new funding will also support the development of one or two compounds for other diseases. PAQ currently has 6 employees, and Ji said that the company will hire more employees as it expands its scope of work.
Pac-Man is more than just a convenient way to explain how PAQ drugs work. The game was in the mind of the founder from the beginning. Ji explained that the scientific illustration of autophagosomes resembles the letter C shape of video game characters. In fact, in the early days of the company’s establishment, scientists referred to their research as the “Pac-Man Project.”
The “Q” in the name refers to polyQ disease, which is characterized by a repetitive sequence in part of the protein. Huntington’s disease is a polyQ disease. The letter Q in the name of PAQ is an endorsement of scientific founder Lu’s research, which first attracted Ji’s attention. When it came time to officially name the startup, Ji said that Pac-Man’s reference made sense.
“It’s short and attractive,” he said. “It represents the history of the company.”
Photos of Flickr users Game Core Blog Through knowledge sharing license
