Third Rock Ventures Launches Casma Therapeutics with $58.5 Million Investment to Harness the Cellular Process of Autophagy to Develop Breakthrough Therapies

Investors and Media, George Washington | May 3, 2018
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Company is focusing on lysosomal storage disorders, liver and muscle diseases, inflammation and neurodegeneration

CAMBRIDGE, Mass., May 3, 2018 — Casma Therapeutics, Inc., a biotechnology company harnessing the natural cellular process of autophagy to design powerful new medicines, launched today with $58.5 million in Series A funding from Third Rock Ventures.

Casma is pioneering novel strategies to boost autophagy to arrest or reverse the progression of rare genetic diseases and broader pathologies, including neurodegeneration. Autophagy is the natural process by which cells break down surplus or dysfunctional protein; organelles such as mitochondria; and viruses and other pathogens. Cells then recycle these components and reuse them as an energy source or as building blocks for new structures essential for cellular function. If autophagy doesn’t work properly or is insufficent, dysfunctional material builds up and fuels a cascade of problems — from protein aggregation to lipid accumulation — that drive disease. The 2016 Nobel Prize for Physiology or Medicine was awarded for the discovery of the key mechanisms of autophagy. Casma’s founders have advanced the understanding of these mechanisms to develop the foundation of potentially powerful disease-modifying therapies to improve the lives of patients.

Propelled by a deep understanding of the autophagy process and led by experienced biotech executives, 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 autophagy process to improve the process of clearing out unwanted proteins, organelles and pathogens. Preclinical data suggest that intervening in this way may lead to treatments for a wide range of disease, including lysosomal storage disorders, liver and muscle diseases, inflammatory disorders and neurodegeneration.

“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,” said Keith Dionne, Ph.D., Casma’s chief executive officer. “We are driven to expand our understanding of this fundamental cellular process — and to translate those insights into breakthroughs for patients.”

“Understanding autophagy has opened up a vast new set of targets for drug discovery and development,” said Beth Levine, M.D., a scientific co-founder of Casma who discovered the first known mammalian autophagy gene, BECN1. “We see a tremendous opportunity to make a difference for patients who have few other options.”

Casma’s scientific founders have made groundbreaking advances in understanding autophagy and translating those insights into drug discovery opportunities.

  • Andrea Ballabio, M.D., Ph.D., is the founder and director of the Telethon Institute of Genetics and Medicine (TIGEM) of Pozzuoli and a leader in the field of transcriptional regulation of lysosomal biogenesis and autophagy. Andrea’s research is being applied to the understanding and design of novel treatment strategies for rare genetic diseases, including lysosomal storage disorders as well as common neurodegenerative diseases.
  • James Hurley, Ph.D., is the Judy C. Webb Chair and professor of biochemistry, biophysics and structural biology at the University of California at Berkeley. Jim has performed groundbreaking studies to determine the structure and activity of autophagy core complexes using crystallography, mass spectrometry and cryo-electron microscopy.
  • Beth Levine, M.D., is 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. She is also an investigator at the Howard Hughes Medical Institute. Beth is recognized as a worldwide expert in autophagy for her discovery of the mammalian autophagy gene, BECN1, and other components of the autophagy pathway.
  • Herbert “Skip” Virgin, M.D., Ph.D., is currently executive vice president for research and chief scientific officer at Vir Biotechnology and previously served as the Edward Mallinckrodt Professor and chair of the department of pathology and immunology at the Washington University School of Medicine. Skip has forged new ground in the understanding of autophagy’s role in inflammation and immunity.

Casma’s leadership team has deep experience in both the science and the business of drug discovery.

Casma is led by CEO Keith Dionne – a serial entrepreneur with over 20 years of experience in biotechnology including leading three other biotech companies. Leon Murphy, Casma’s senior vice president, biology is an expert in the field of autophagy and drug discovery, most recently at Novartis Institutes for BioMedical Research. Frank Gentile, the interim COO, brings nearly 25 years of experience working with biopharma companies, including more than a decade with Tekla Capital Management. Bob Tepper, the interim CSO, is a co-founder of Third Rock Ventures and previous president of R&D at Millennium Pharmaceuticals. Cary Pfeffer, the interim CBO, is a partner at Third Rock Ventures and has assumed leadership roles in multiple portfolio companies, including Rheos Medicines, Neon Therapeutics and Tango Therapeutics.

Dr. Dionne, Dr. Gentile, Dr. Tepper and Dr. Pfeffer also sit on Casma’s board of directors.

About Casma Therapeutics

Casma Therapeutics is harnessing the natural cellular process of autophagy to open vast new target areas for drug discovery and development. Casma uses several approaches to intervene at strategic points throughout the autophagy system to improve the cellular process of clearing out unwanted proteins, organelles and invading pathogens. By boosting autophagy, Casma expects to be able to arrest or reverse the progression of lysosomal storage disorders, muscle disorders, inflammatory disorders and neurodegeneration, among other indications. Casma was launched in 2018 by Third Rock Ventures and is based in Cambridge, Mass. For more information, please visit

About Third Rock Ventures

Third Rock Ventures is a leading health care venture firm focused on disruptive areas of science and medicine to discover, launch and build companies that make a dramatic difference in people’s lives. By combining the team’s scientific vision, strategic leadership, operational expertise and innovative deal-making capabilities, Third Rock Ventures nurtures bold ideas that translate into successful business enterprises. Recognizing that the best way to create value for investors is to create value for patients, Third Rock companies are built on a solid foundation of science, medicine, people and business strategy. For more information, please visit

Media Contact:

Stephanie Simon
Ten Bridge Communications

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