UB scientists awarded $1.5 million for hemophilia treatment

BUFFALO, N.Y. – University at Buffalo researcher Sathy Balu-Iyer has been awarded a three-year, $1.5 million grant to continue his work on enabling a patient’s immune system to accept treatment for hemophilia.

The award comes from the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health. The research will focus on developing a way to deliver Factor VIII, a blood-clotting protein, by desensitizing the immune system so that it does not reject this lifesaving therapy.

“My interest in hemophilia started when I read a mother’s story about her son who couldn’t tolerate the infusions to treat his hemophilia,” said Balu-Iyer, professor of pharmaceutical sciences. He explained that hemophilia is considered a rare disease affecting one in 5,000 male births. Most forms of hemophilia are genetic, either inherited from a mother who is a carrier or from a spontaneous genetic mutation. Rarely, hemophilia can be acquired.

Through earlier research funded in part by NHLBI, Balu-Iyer and his collaborators developed an immunotherapy platform called Tolerance Inducing Phosphatidylserine (TIPS), which contains a tolerogenic nanoparticle. “TIPS essentially teaches the immune system to tolerate something it would normally see as a threat,” explained Balu-Iyer.

Nonprofit supports novel drug development, application

Empire Discovery Institute (EDI) is a nonprofit drug discovery and development venture studio formed under an affiliation with UB, Roswell Park Comprehensive Cancer Center and the University of Rochester.

In 2021, EDI awarded $250,000 to Balu-Iyer’s team at the School of Pharmacy and Pharmaceutical Sciences to support research into using TIPS for gene therapy applications through EDI’s Medicines Discovery Award Program (MDAP). That initial investment, described earlier in UBNow, led to encouraging results, and EDI awarded the project a second award of $750,000 earlier this year.

EDI’s ultimate objective is to invest in drug development programs originating from the three founding institutions and then license a successful discovery to a new or existing pharmaceutical company and benefit Western New York’s biotech infrastructure. EDI is funded by an initial $35.4 million grant from Empire State Development.

Balu-Iyer has devoted much of his career to furthering knowledge of immunotherapy.

“We are interested in any kind of immune system response that is harmful, including allergies to simple things like eggs or gluten,” he said.

Autoimmune diseases are another target; one example is Type 1 diabetes, which is usually caused when the body’s own immune system destroys insulin-producing cells. Another problem occurs when the immune system attacks a medical intervention intended to improve a patient’s health. That is the case with about one-third of patients receiving Factor VIII through existing protocols.

TIPS has the potential to be used in treating other diseases where gene therapy is used. Balu-Iyer said that the next phase of research at UB will include additional research into using TIPS to treat diseases such as Type 1 diabetes.

UB’s Center of Excellence in Bioinformatics and Life Sciences (CBLS) has provided an additional $100,000 to supplement the recent MDAP project. CBLS provides an infrastructure to bring lab findings and promising research to the public by partnering with the private sector and other research institutions. EDI and UB are developing a startup company this year to take commercialization of the TIPS platform to the next phase.