(Gerd Altmann, Pixabay)
3 May 2018. A start-up enterprise is discovering therapies to address a variety of disorders that result from faulty cell degradation processes known as autophagy, which when functioning normally contribute to cell health. Casma Therapeutics Inc. in Cambridge, Massachusetts is a creation of venture investment company Third Rock Ventures, which staked the Casma Therapeutics to $58.5 million its first round of venture financing.
Casma plans to design and develop treatments for diseases that restore autophagy functions disrupted by genetics or other factors that the company says represent a number of serious unmet medical needs. Under normal functioning conditions, autophagy plays a key role in the processing of degraded proteins and even cell death in some cases. Processed cell components provide raw materials for the cell’s energy center or for building new cell structures. When autophagy is blocked or disrupted, however, cells come under stress, leading to a build up of disease-causing substances.
Among the conditions traced to these malfunctioning functions are lysosomal storage disorders, metabolic diseases resulting from a build-up of toxins resulting from various enzyme deficiencies linked to autophagy. People with lysosomal storage disorders cannot metabolize or break down large molecule proteins, carbohydrates, and fats into their components. Casma expects to discover and develop treatments that intervene when autophagy cycles break down in lysosomal storage disorders, as well as liver and muscle conditions, inflammatory disorders, and neurodegenerative diseases.
Casma is founded by four researchers, three from the academic world and an industry scientist:
– Andrea Ballabio, director of the Telethon Institute of Genetics and Medicine in Naples, Italy, and affiliated with Baylor College of Medicine
– James Hurley, professor of biochemistry, biophysics, and structural biology at the University of California in Berkeley
– Beth Levine, director of the Center for Autophagy Research at University of Texas Southwestern Medical Center
– Herbert “Skip” Virgin, executive vice president for research and chief scientific officer at Vir Biotechnology
“Understanding autophagy has opened up a vast new set of targets for drug discovery and development,” says Levine in a company statement. “We see a tremendous opportunity to make a difference for patients who have few other options.” Levine is credited with discovery of the first known autophagy gene.
The company’s CEO is Keith Dionne, a serial entrepreneur who led three other biotechnology enterprises. Most of the other company executives are on loan from Third Rock Ventures, and will serve on an interim basis. Third Rock, a venture capital company in Boston and San Francisco specializing in early-stage life science enterprises, is providing all of the $58.5 million in first round funding for Casma.
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