Published May 7, 2021
Two UB drug development and discovery projects have received investments from the Empire Discovery Institute.
One project focuses on an immunotherapy platform — developed by UB pharmacy researcher Sathy Balu-Iyer and his team — that could benefit patients who are receiving gene therapy for a variety of diseases. The other will seek to design a therapy for multiple sclerosis (MS) that leverages a novel drug target discovered by a team led by UB medical researcher M. Laura Feltri.
The institute will advance both efforts through its Medicines Discovery Award Program, a competitive incubator and accelerator program designed to identify and advance promising early-stage drug discovery into early “proofs of concept.” Successful projects will exit the program as either a licensing transaction to a strategic pharmaceutical partner or as an Empire Discovery Institute-created startup company.
Empire Discovery Institute is a nonprofit New York drug discovery and development accelerator created to translate important academic discoveries into new medicines for commercialization by providing strategic support and pharmaceutical development expertise. Established with $35.4 million in initial funding from Empire State Development, it works in partnership with research labs at UB, the University of Rochester and Roswell Park Comprehensive Cancer Center.
TIPS immunotherapy platform
UB principal investigator: Sathy Balu-Iyer, professor of pharmaceutical sciences, School of Pharmacy and Pharmaceutical Sciences
This project seeks to develop an immunotherapy platform that could improve treatment outcomes for patients receiving gene therapy for rare disease conditions such as hemophilia and Pompe disease. Gene therapy holds great promise for treating a variety of diseases. However, these therapies are delivered using “vectors” that can include inactivated viruses, and patients sometimes have immune responses to the vectors and against transgene products, which can decrease treatment efficacy.
The UB immunotherapy platform — called Tolerance Inducing Phosphatidylserine (TIPS) — is designed to prevent, reduce or reverse immunogenic reactions to gene therapies that leverage adeno-associated viruses (AAV) as the vector. Because people are commonly exposed to various forms of wild AAVs, some patients have developed enough immunity to AAV that their immune systems attack AAV-delivered gene therapy. UB has a portfolio of patent applications tied to the TIPS technology.
The investment will support preclinical research in Balu-Iyer’s lab, and by contract research organizations. In addition to potential applications tied to gene therapy, the TIPS platform also holds potential for improving treatment for autoimmune conditions such as Type 1 diabetes and rheumatoid arthritis and allergies, among others.
A novel multiple sclerosis drug target
UB principal investigator: M. Laura Feltri, SUNY Distinguished Professor of Biochemistry and Neurology, and acting director of the Hunter James Kelly Research Institute in the Jacobs School of Medicine and Biomedical Sciences at UB
This project seeks to design and develop a therapy for MS, leveraging a novel drug target discovered by Feltri’s team.
Within the central nervous system, a coating called the myelin sheath protects nerve cells, akin to the coating around electric wires. When this protective layer is damaged, the body will make repairs. In patients with MS, this process, called remyelination, becomes inefficient over time.
The focus of the project is on developing a treatment to correct the remyelination repair function in MS patients. This will involve synthesizing, testing and optimizing compounds that could inhibit the novel drug target that Feltri and colleagues have identified.
The investment will support this work in Feltri’s lab, and by contract research organizations.
In MS, damage to the central nervous system causes problems that can include fatigue, numbness, weakness, dizziness, blurred vision and difficulty walking. Symptoms vary between patients, and can range from mild to severe. The Multiple Sclerosis International Federation estimated in 2020 that 2.8 million people globally have MS. Western New York has a high prevalence of MS, with a 2013 statement from the upstate New York chapter of the National Multiple Sclerosis Society noting that diagnosis rates in the region were double the U.S. average.
In other news, the institute and health care investment firm Deerfield Management Company announced on April 26 a research partnership to accelerate development of novel therapeutics.
While separate from the funding mentioned above, Deerfield expects to invest up to $65 million over the next five years through a company the institute and Deerfield launched that’s called Empire Deerfield Discovery & Development LLC (ED3). The company will fund and support projects in high-need therapeutic areas, as well as hard-to-treat and rare diseases, to accelerate them toward clinical validation in patients and commercialization. ED3 will solicit drug development projects from UB, University of Rochester and Roswell Park Comprehensive Cancer Center.
UB participation in the institute and ED3 drug discovery and development programs is coordinated through Business and Entrepreneur Partnerships, a UB team connecting researchers and growing startups with funding, space, facilities, expertise and commercialization mentorship to reach key business milestones.
“The Deerfield partnership significantly enhances Empire Discovery Institute’s ability to advance novel therapeutics discovered at UB and our founding partners’ institutions,” says President Satish K. Tripathi, who serves as chair of the institute’s board of directors. “Building upon the $35 million grant from New York State to the institute, the Deerfield partnership gives our faculty researchers access to additional funding and expertise to help them move their innovations from an idea to the market, bringing new lifesaving drugs to improve the health and well-being of individuals, communities and society.”
Adds Martin Graham, chief executive officer of the Empire Discovery Institute: “The institute was founded to help overcome the key challenges often faced by life science researchers in academia, namely a lack of external funding to advance pre-clinical development and access to pharmaceutical industry expertise to advance programs in an efficient manner to the clinic. Our partnership with Deerfield will enable us to accelerate our mission to identify medically important targets for human disease to facilitate the creation and development of novel, highly differentiated new therapeutics.”
Graham, who was appointed CEO of the Empire Discovery Institute in April 2019 after a national search, has more than 30 years of experience in the drug development field with both startups and large pharmaceutical companies. He has assembled a highly experienced Scientific Advisory Board with extensive pharmaceutical drug discovery and development expertise from leading life science companies such as Merck, Janssen, Centocor, Johnson & Johnson, AstraZeneca, Pfizer, Sanofi, Schering-Plough and GSK, among others. Adam Tulgan serves as chief operating officer. In March, the institute hired seasoned pharmaceutical executive Ronald Newbold to serve as chief business officer.