BUFFALO, N.Y. — Tonus Therapeutics, a University at
Buffalo spin-off company, has opened its first-ever headquarters in
UB’s New York State Center of Excellence in Bioinformatics
and Life Sciences.
Tonus develops drugs that target mechanosensitive ion channels,
which are tiny conduits that help control the flow of important
substances, such as calcium, into cells. The firm’s first
project is developing a therapy for muscular dystrophy using GsMTx4
— a peptide that UB scientists first discovered in the venom
of the Chilean rose tarantula.
This research is of particular significance because the peptide,
now made by chemical synthesis, is considered an “orphan
drug” for muscular dystrophy, a designation that the U.S.
Food and Drug Administration gives to recognize promising methods
of treating rare diseases.
The move to the Center of Excellence, located on Ellicott St. on
the Buffalo Niagara Medical Campus, took place in early December.
The company was founded in 2009 by three UB researchers and a local
stockbroker whose grandson has a severe form of muscular dystrophy,
a disease that causes patients’ muscles to atrophy.
“Being located in a hub of research activity in Buffalo,
close to other entrepreneurs and biotech startups, is important to
us,” said the grandfather, Jeff Harvey, who serves as
Tonus’ chief financial officer. “The Buffalo Niagara
Medical Campus is extremely important to us because it provides
ready contact with experienced entrepreneurs and potential clinical
and commercial partners, including those within the Center of
(To learn how Harvey’s grandson, JB, inspired the creation
of Tonus Therapeutics, visit http://www.buffalo.edu/home/feature_story/good-venom.html.)
The opening of offices in the Center of Excellence follows other
recent milestones for Tonus. In November, the company licensed UB
patents relating to GsMTx4 through UB’s Office of Science,
Technology Transfer and Economic Outreach (UB STOR).
“When companies like Tonus partner with the Center of
Excellence, it’s a win for everyone involved,” said
Marnie LaVigne, UB associate vice president for economic
development. “Tonus benefits from the technological and
business resources available here, and their continued growth will
be an asset to Western New York’s economy. We’re
excited to work with such a passionate and driven team as they
navigate this important stage of development.”
Besides Harvey, the founding principals of Tonus Therapeutics
are three UB faculty members: SUNY Distinguished Professor
Frederick Sachs, Research Assistant Professor Thomas Suchyna and
Research Associate Professor Philip Gottlieb, all members of the
Department of Physiology and Biophysics in UB’s School of
Medicine and Biomedical Sciences.
Sachs’ team first came across GsMTx4 more than a decade
ago while studying the effect of venoms on mechanosensitive ion
These channels, which Sachs co-discovered in 1983, connect the
inside of a cell with its outside. Normally, in healthy cells the
channels are closed, but when a cell is stretched or contorted, the
channels open and let calcium and other substances into the
This is what happens in muscular dystrophy: Due to a defective
gene, patients are missing the fiber-like reinforcing protein
called dystrophin that helps muscle cells keep their shape, Sachs
said. This causes the cell membrane to get stretched more easily,
prompting the ion channels to open, letting calcium flood in. The
end result of this chain reaction is that the body “starts
digesting muscle from the inside out,” Sachs said.
Tonus has gathered preliminary data showing that GsMTx4 is
nontoxic in mice and did not disturb heart function in mice or
ferrets or isolated human heart muscle, Sachs said. He added that
the drug is quite “sticky” and is capable of staying in
the body for a long time without breaking down. This means it could
be possible to deliver low doses infrequently, reducing costs for
Prior to their move into the Center of Excellence, the Tonus
team benefited from other UB resources and partnerships. Harvey is
a graduate of the UB School of Management’s High-Tech Center
for Entrepreneurial Leadership program, and Tonus received funding
through the UB Center for Advanced Biomedical and Bioengineering
Technology (UB CAT). The Children’s Guild Foundation and the
John R. Oishei Foundation have provided additional funding.
“The whole project on mechanosensitive ion channels and
GsMTx4 and its use in treating muscular dystrophy is 100 percent
Buffalo; everything was discovered here,” Sachs said.
Besides investigating the effects of GsMTx4 on muscular
dystrophy, Tonus is also exploring its application to other
diseases, including sickle cell anemia, xerocytosis and chronic
Real-Life Spider Men Using Protein Found in Venom to Develop
Muscular Dystrophy Treatment: http://www.buffalo.edu/news/releases/2012/07/13542.html
Good Venom: https://www.buffalo.edu/home/feature_story/good-venom.html