Casma Therapeutics Receives Grant from Michael J. Fox Foundation to Support Parkinson’s Drug Development

May 29, 2019

CAMBRIDGE, Mass., May 29, 2019 —  Casma Therapeutics, Inc., a biotechnology company harnessing the process of autophagy to design powerful new medicines, today announced receipt of a Therapeutic Pipeline Program research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support development of a novel class of drugs to treat Parkinson’s disease.

The $370,000 grant will advance Casma’s research into drugs that activate the calcium channel TRPML1 to promote the cellular process of autophagy to clear a toxic protein from the brain. Parkinson’s disease is believed to be caused by a build-up of these toxic proteins in specific neurons. By accelerating autophagy – the cell’s natural mechanism for disposing of pathogens – the Casma team hopes to slow disease progression.

In collaboration with co-MJFF grantee Ellen Sidransky, M.D., a Parkinson’s disease expert and a senior investigator at the National Human Genome Research Institute (NHGRI), National Institutes of Health (NIH) (Bethesda, MD), the Casma team will test TRPML1-activating drugs in human induced pluripotent stem cell-derived dopaminergic neurons that model Parkinson’s disease that will be generated by the NHGRI team from patient samples collected at NIH.

“Inducing the natural process of autophagy to clear toxic proteins is a promising new approach to treating Parkinson’s, and we’re thrilled to have support for our research from The Michael J. Fox Foundation,” said Daniel Ory, M.D., senior vice president, translational medicine at Casma Therapeutics. “We share the Foundation’s sense of urgency, and we’re eager to move our program forward.”

“Funding therapies against emerging targets such as TRPML1 is a crucial plank in our effort to find a cure for Parkinson’s,” said Liliana Menalled, Ph.D., MJFF senior associate director of research programs. “We are committed to leaving no stone unturned as we pursue therapies that will help the more than 6 million people worldwide living with this disease.”


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 The Michael J. Fox Foundation for Parkinson’s Research

As the world’s largest nonprofit funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $800 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit


About the National Human Genome Research Institute

NHGRI is the driving force for advancing genomics research at the National Institutes of Health. By conducting and funding world-class genomics research, training the next generation of genomics experts, and collaborating with diverse communities, NHGRI accelerates scientific and medical breakthroughs that improve human health. Learn more at

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Ten Bridge Communications
Stephanie Simon, 617-581-9333

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