Casma Therapeutics Announces Partnership with Fondazione Telethon

Casma Therapeutics 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.

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