Published January 29, 2013
Therapeutics, a UB spinoff firm developing a promising new
therapy for muscular dystrophy, has opened its headquarters in UB’s New York
State Center of Excellence in Bioinformatics and Life
“Being located in a hub of research activity in Buffalo,
close to other entrepreneurs and biotech startups, is important to
us,” says Jeff Harvey, the firm's co-founder and chief
“The Buffalo Niagara Medical Campus provides ready contact with experienced entrepreneurs and potential clinical and commercial partners, including those within the Center of Excellence.”
Tonus is developing a drug derived from a peptide that UB
scientists discovered in the venom of the Chilean rose
The U.S. Food and Drug Administration has designated the
peptide, now made by chemical synthesis, an “orphan
drug” for muscular dystrophy, a designation recognizing
promising methods of treating rare diseases.
The Tonus researchers’ findings stem from their long-term
studies of venoms targeting mechanosensitive ion
channels—tiny conduits that help control the flow of
important substances, such as calcium, into cells.
Due to a defective gene, people with muscular dystrophy lack the protein dystrophin, which helps muscles keep their shape. Consequently, their cell membranes stretch more easily, prompting the ion channels to open, letting calcium flood in.
The peptide that the UB researchers discovered, GsMTx4, keeps the ion channels shut, offering potential for a drug that could suppress the debilitating symptoms of a host of diseases, including muscular dystrophy.
Tonus is also exploring the peptide’s effects on sickle cell anemia, xerocytosis and chronic kidney failure.
Researchers have gathered preliminary data showing that GsMTx4 is nontoxic in mice and does not disturb heart function in mice or ferrets or isolated human heart muscle.
The drug is capable of staying in the body for a long time
without breaking down, so it could be possible to deliver low doses
infrequently, reducing patients’ costs, Sachs notes.
Tonus was founded in 2009 when Harvey, a stockbroker whose
grandson has a severe form of muscular dystrophy, joined forces
with UB researchers.
In addition to Harvey and Sachs, the other co-founders, both in physiology and biophysics, are vice presidents Thomas Suchyna, research assistant professor, and Philip Gottlieb, research associate professor.
“The whole project on mechanosensitive ion channels and GsMTx4 and its use in treating muscular dystrophy is 100 percent Buffalo,” Sachs says.
“Everything was discovered here.”
Tonus has benefited from several UB resources and partnerships,