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Few biotech executives describe their experimental therapies by comparing them to computer game. Nan Ji, president and CEO of PAQ Therapeutics, does so enthusiastically, and by utilizing a 40-year-old video game at that. In the 1980s, Pac-Man was an arcade fixture and kids with pockets complete of quarters lined up for a chance at browsing the name character through a maze, gobbling up pellets for points.
In a similar vein, PAQ Therapeutics is establishing drugs that get a part of a cell to chomp on targets associated with disease. Ji notes that proteins arent the only parts of a cell that can cause disease.
” This is a very novel technique based on new biology,” Ji said. “Were still early however we think there is fantastic potential.”.
The Cambridge, Massachusetts-based startup is making steady progress with its research study, enough to level up with $30 million in financing. The Series A funding round announced Thursday was led by Sherpa Healthcare Partners.
Utilizing a cells built-in parts to deal with disease is catching on. A growing number of biotechs are delving into targeted protein deterioration, in which disease-causing proteins are directed to the part of a cell that gets rid of old or damaged proteins. Ji understands the area well, having worked for three years as head of chemistry at Kymera Therapeutics, one of the business that has reached clinical testing with this brand-new approach to treating disease.
In 2019, while Ji was still at Kymera, he read a paper released in Nature that described utilizing autophagy, a process of disposing of other cellular parts, as a way of dealing with illness. That research did focus on a protein, specifically, the mutant huntingtin protein that cause the neurodegenerative disorder Huntingtons disease. However Ji said he was particularly interested in autophagys potential to attend to non-protein targets.
” Right away I recognized this could offer an opportunity that is not there for targeted protein destruction,” he stated.
Ji stated he reached out to Boxun Lu, a professor at Fudan University in Shanghai and the last author on the Nature paper. Together, they started PAQ to establish new drugs that utilize autophagy. In the body, autophagy is brought out by the lysosome, a different cellular disposal system than the one that carries out protein deterioration. Waste gets to the lysosome by means of the autophagosome, a sphere-shaped element of a cell. Ji said PAQ is establishing small particles that speed up the interaction between the autophagosome and the disease-associated parts of a cell. The particles PAQ is establishing are called autophagosome-tethering compounds, or ATTECs.
PAQs lead illness target is a neurodegenerative condition that the business is not divulging, other than to say that its a genetic illness that does not presently have any restorative alternatives. Ji stated that the businesss little particle can cross the blood brain barrier to reach its target. Up until now, the business has actually done pharmacodynamic and pharmacokinetic studies in animals, tests that reveal how a drug is taken up by the body and the bodys biological action to the therapy. Ji stated he is particularly interested in the latter, since in this concealed illness target, pharmacodynamics have a direct correlation with efficacy, and might be a harbinger of what to expect in human beings.
PAQ isnt the first biotech startup wanting to autophagy as a way to attend to neurodegeneration. Libra Therapeutics is establishing small particles that increase autophagy. The San Diego-based biotech released last year with $29 million in Series A money and amyotrophic lateral sclerosis as its lead disease target. QurAlis, a Cambridge start-up, raised $42 million in Series A financing last year for its research on a number of different methods to ALS, consisting of one in autophagy.
The other financiers in PAQs brand-new funding are Huagai Capital, MSA Capital, and MRL Ventures Fund. They are joined by seed financiers, Nest.Bio Ventures and Matrix Partners China. With its fresh cash haul, Ji stated that PAQ can continue to advance its lead program further in preclinical advancement. However he included that the innovation is a platform with the potential to support drugs addressing targets beyond the main nervous system, and the new cash will likewise support the advancement of a couple of compounds for other diseases. PAQ presently utilizes six and Ji stated that the company will make additional hires as it broadens its work.
Pac-Man is more than a practical method to explain how PAQs drugs work. Scientific illustrations of the autophagosome resemble the letter C shape of the video game character, Ji explained.
The “Q” in the name referrals polyQ conditions, which are defined by a portion of a protein that has duplicated sequences. Huntingtons illness is a polyQ disease, and the letter Q in PAQs name is a nod to the research study from clinical creator Lu that initially caught Jis eye. When it came time to officially call the startup, Ji stated the Pac-Man recommendation made sense.
” It was appealing and brief,” he stated. “It represents the history of company.”.
Photo by Flickr user Gamescore Blog via a Creative Commons license.

Ji keeps in mind that proteins arent the only parts of a cell that can cause illness. Ji knows the area well, having worked for 3 years as head of chemistry at Kymera Therapeutics, one of the business that has reached medical screening with this brand-new approach to treating illness.
Ji stated PAQ is establishing little molecules that speed up the interaction in between the autophagosome and the disease-associated parts of a cell. Ji stated he is particularly interested in the latter, due to the fact that in this undisclosed disease target, pharmacodynamics have a direct connection with effectiveness, and could be a harbinger of what to expect in people.
Huntingtons illness is a polyQ disease, and the letter Q in PAQs name is a nod to the research from clinical creator Lu that first captured Jis eye.