Casma Therapeutics Announces Partnership with Fondazione Telethon, Founder of the Telethon Institute of Genetics and Medicine (TIGEM)

September 11, 2018

CAMBRIDGE, Mass., September 11, 2018 — Casma Therapeutics, Inc., a biotechnology company harnessing the process of autophagy to design powerful new medicines, today announced a research collaboration with Fondazione Telethon, founder of the Telethon Institute of Genetics and Medicine (TIGEM), to develop new therapies for lysosomal storage disorders and more common disorders such as neurodegeneration.

TIGEM, an international research institution based in Naples, Italy, employs over 200 scientists working to understand the mechanisms of rare genetic diseases and to develop new therapies. The partnership will bring together Casma’s strong foundation in autophagy biology and drug discovery with TIGEM’s clinical experience and insights into rare genetic diseases in a push to advance novel therapies into the clinic in coming years.

“Our team at Casma is leveraging insights in autophagy to discover therapies to treat patients in need,” said Leon Murphy, Ph.D., senior vice president of biology at Casma. “I am very excited to be working alongside an outstanding team of researchers at TIGEM as we pursue our goal of halting and even reversing the progression of severe diseases.”

TIGEM is led by Scientific Director Andrea Ballabio, M.D., one of Casma’s scientific co-founders and Chief Scientific Officer Graciana Diez-Roux, Ph.D., who oversees many of TIGEM’s strategic collaborations. Ballabio is the author of over 300 scientific papers concerning human genetics; his current research focuses on the role of autophagy in lysosomal storage disorders and neurodegenerative diseases.

“We see great potential for synergy in this partnership and we’re excited to begin working together to translate discoveries into new therapies for patients,” Ballabio said.

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-lysosome 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 Fondazione Telethon and TIGEM

Fondazione Telethon (the Telethon Foundation) is a major biomedical charity in Italy whose mission is to advance biomedical research to cure rare genetic diseases. Throughout its 28 years of activity, the Telethon Foundation has invested almost €500 million in funding over 2,600 projects to study 570 diseases, involving more than 1,600 researchers. The Telethon Institute of Genetics and Medicine (TIGEM) was founded in 1994 by the Telethon Foundation and represents one of the most important centers for fundamental life science research in Italy. Located in Naples, TIGEM is a multidisciplinary research institute devoted to the study of the mechanisms underlying rare genetic diseases and to the development of new therapies. The institute employs over 200 researchers from around the world in 20 independent research groups, which are engaged in both applied research and numerous clinical trials. For more information, please visit and

Ten Bridge Communications
Stephanie Simon, 617-581-9333

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