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Biomolecular Condensate Research Is Gaining Momentum

11 months, 3 weeks ago

7017  0
Posted on Dec 11, 2020, 4 p.m.

Biomolecular condensates are membrane-less organelles made up of molecules that perform various functions within the cell. These membrane-less organelles form liquids in the cell separate from the cell’s cytoplasm, and they may be a key component to understanding how cells function and malfunction. 

Face Medicines is the third company in recent months to join in the pursuit of these targets. With $81 million in financing, the company will use the capital to finance preclinical work in neurodegenerative diseases, amyotrophic lateral sclerosis and a muscle-wasting disorder called myotonic dystrophy type 1, as well as support other research in other fields such as immunology, cancer, and virology. 

The company is starting out with a focus on these diseases as there are clear genetic links, and they hope to identify contributors to drug directly or target processes that can change the important contributor’s behaviors. 

Comparing biomolecular condensate to office building Cary Pferrfer, M.D., and interim CEO explains, “On any given day, there are different meetings going on—some are big meetings, some are small meetings, and they all need to have the right people in them or they can’t get the right stuff done. People come and go all day long in meetings, and that’s kind of the way to think about condensates.”

Rachel Meyers, Ph.D. and chief scientific officer further explains that much like a person disrupting a meeting mutated proteins in a condensate can lead to disease. “We need to figure out the most important contributors in a condensate. Those contributors will become our targets for drug discovery.” 

“There are lots of directions one could go—where we will go and we will see the field go—but we really chose to hang our hat on human genetics as a functional driver of what diseases to go after,” Meyers said.

The company is actively looking at virology, cancer, immunology, and metabolic diseases as well as other neurodegenerative diseases for potential new avenues to explore with its current staffing of 12, but there are plans to build their team to reach 25-30 by the end of 2021. 

Just months before Faze Medicines entered the market Nereid Therapeutics launched with $50 in financing and Dewpoint Therapeutics acquired a $77 million series B; both of these companies are also pursuing the field of biomolecular condensates. 

Nereid Therapeutics has plans to develop treatments for neurodegenerative diseases, certain cancers, and fibrosis. Dewpoint Therapeutics is working on a $305 million deal with Merck to pursue programs in HIV and they are also working with Bayer in a $100 million deal to develop treatments for cardiovascular disease and women’s health. 

Nereid believes that their platform will allow researchers to extract a fingerprint of the interactions that are happening within the cells to help their team gain better understandings that will help investigate how to modify these interactions with the hopes of moving cells away from disease-causing processes. According to Clifford Brangwynne, Ph.D., who is a pioneer in the condensates field and chair of the scientific advisory board “There have not been ways to quantitatively interrogate what's happening in cells, the underlying physical forces that are at play in organizing a cell and forming a healthy cell with healthy structures, and understanding how that process goes wrong….the reason why we have no treatments for these diseases is that there has not been a fundamental understanding of the thermodynamics, the molecular interaction landscape that drives assembly.”

Dewpoint believes that their technology will work for all of these diseases because “the proteins or the targets that cause the diseases in each case have been identified as being misregulated in a condensate. Something's gone wrong where this protein was supposed to be in a particular condensate, or suddenly it's ending up in a condensate, and we're able to intervene to change that behavior,” according to CEO Amir Nashat. 

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