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

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

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


Ten Bridge Communications
Stephanie Simon, 617-581-9333
[email protected]

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