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NIH Giving In-Kind Technical Services for Rare Diseases

Oakland Bay Bridge and fireboat (A. Kotok)

(A. Kotok)

National Institutes of Health (NIH) is supporting three new projects with technical research services for two companies and one university lab developing therapies for rare diseases. The projects aim to develop treatments for acute radiation syndrome, brain injury following cardiac arrest, and beta thalassemia, a rare blood disorder.

The awards are a product of the Bridging Interventional Development Gaps (BrIDGs) program, where researchers receive expert lab services from NIH’s contractors at no cost. BrIDGs services aim to help recipients with early evidence showing a potential therapy for treating rare diseases over technical obstacles, leading to submission of an Investigational New Drug application with the FDA followed by clinical trials. The work is funded by NIH’s Common Fund, but the dollar value of the in-kind services for the three new recipients was not announced.

One recipient, Terapio Corp. in Austin, Texas, is designing a treatment for acute radiation syndrome, also known as radiation sickness, resulting from a large dose of radiation in a short period of time, as in a nuclear reactor emergency or “dirty-bomb” terrorist attack. The company’s technology is based on a recombinant protein, RLIP76, that was shown to protect lab mice from radiation sickness, even when administered as late as 24 hours after exposure to radiation. Terapio’s work is based on research by Sanjay Awasthi, then of University of Texas at Arlington, who co-founded the company that licensed the technology for commercialization.

A second company receiving BrIDGs help is Merganser Biotech LLC in Newtown Square, Pennsylvania. The company is developing a treatment for beta thalassemia, a blood disorder that reduces the production of hemoglobin, the iron-containing protein in red blood cells carrying oxygen to cells in the body.

Patients with beta thalassemia produce too little of a hormone called hepcidin, that is made in the liver and regulates the production of iron. With too little hepcidin in their bodies, beta thalassemia patients produce too much iron, which can damage the heart. Merganser Biotech aims to develop a treatment that increases levels of hepcidin and lowers the damaging effects of too much iron.

Laurence Katz, an emergency medicine professor at University of North Carolina in Chapel Hill, is the third recipient of BrIDGs services. Katz is developing a therapy for acute brain injury brought on by cardiac arrest, based on hypothermia, the process of lowering the body’s temperature. Research by Katz and others shows hypothermia can improve neurological outcomes in more than half of patients who remain in a coma after cardiac arrest.

Katz is designing an intravenous treatment for paramedics called HBN-1 that can induce hypothermia in patients with cardiac arrest. While the BrIDGs award supports Katz’s work at UNC-Chapel Hill, he also founded a company, Hibernaid Inc., that licensed the technology from the university and is commercializing HBN-1. Katz serves as a scientific advisor to Hibernaid.

NIH says BrIDGs services, such as preclinical testing for toxicity, help generate 12 Investigational New Drug applications, with three compounds so far reaching intermediate stage clinical trials. In addition, seven compounds supported by BrIDGs services are now licensed by companies for commercialization.

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