Casma Therapeutics, a Cambridge, Massachusetts, based biotechnology company, announced its launch today with a $58.5 million Series A investment from Third Rock Ventures. Casma is developing strategies to boost autophagy in the body — the process by which cells break down components and recycle them for energy or as functional building blocks — to target lysosomal storage disorders, liver and muscle diseases, inflammation and neurodegeneration.

Third Rock’s portfolio currently encompasses investments in 50 companies, including household names such as Bluebird Bio, Sage Therapeutics, Blueprint Medicines, Agios and a dozen other publicly traded biotechnology and healthcare companies.

Autophagy facilitates the orderly trafficking of millions of proteins and is also essential for recycling old proteins and mitochondria. Although the body boosts autophagy in response to exercise or reduced nutrient intake, only recently have scientists begun to understand how in certain diseases this quality control process goes awry and is detrimental. According to Jonathan Saltzman in The Boston Globe, the 2016 Nobel Prize in medicine was awarded for discoveries about how yeast cells break down and recycle their own components during autophagy.

Casma views its emerging understanding of autophagy and lysosomal function as the key to opening up novel ways of treating a host of diseases that are neglected or inadequately treated today. Based on disease-specific insights into key autophagy and lysosomal drivers, Casma is working to intervene throughout the entire autophagy / lysosomal process, from the identification and capture of dysfunctional material to the engagement and completion of the autophagy process in the lysosome.

Autophagy system

Source: Casma Therapeutics

According to its press release, Casma has built a robust product engine to identify and drug specific drivers of autophagy. The company is developing the capability to intervene at strategic points throughout the process to improve the clearance of unwanted proteins, organelles and pathogens. Preclinical data suggest that intervening in this manner may lead to treatments for a wide range of diseases.

Source: Casma Therapeutics

This is an exciting time to launch Casma, as new discoveries have shed light on just how vital the process of autophagy is to maintain cellular health and prevent disease. We are driven to expand our understanding of this fundamental cellular process — and to translate those insights into breakthroughs for patients.

– Keith Dionne, PhD, CEO, Casma Therapeutics.

Dr. Dionne is a serial entrepreneur with over 20 years of experience in biotechnology including leading three other biotech companies. Among the company’s scientific co-founders is Beth Levine, MD, discoverer of the first known mammalian autophagy gene, BECN1, and director of the Center for Autophagy Research and the Charles Cameron Sprague Distinguished Chair in Biomedical Science at the University of Texas Southwestern Medical Center.

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