Copernicus Therapeutics notes, opioid use disorder is a serious public health issue in the United States and throughout the world. The Centers for Disease Control and Prevention estimates that the total “economic burden” of opioid misuse in the United States was $1.02 trillion in 2017.
Through this grant, NIDA is providing funding to address this serious public health issue with a novel non-viral gene therapy approach. DNA nanoparticles developed and produced by Copernicus will be administered intranasally. They have been shown to cross the blood brain barrier after intranasal administration and are thus able to deliver their patented therapeutic gene therapy for disorders of the brain, such as opioid use disorder. The work will be directed by Dr. Waszczak in association with the academic expertise and laboratories of co-Principal Investigators from Tufts University, Drs. Elizabeth Byrnes, Professor of Comparative Pathobiology at Cummings School of Veterinary Medicine, and Emmanuel Pothos, Associate Professor of Immunology at Tufts University School of Medicine.
The Phase 1 segment of the awarded grant for $6.7 million covers the first two years of work. Upon successful completion of Phase 1 (projected for year end 2023), the investigators may be eligible to receive an additional ~$8 million in funding from NIDA to further advance this therapeutic approach towards human clinical trials.
The intranasal approach does not require a surgical procedure because the DNA nanoparticles can travel directly from the nose into the brain. This novel approach is expected to restore normal functioning of the brain’s reward system, reduce drug craving, and thus enable patients in recovery avoid relapse. An additional benefit is that the therapeutic effect of a single dose is expected to last for many months or even years, thus reducing relapse long term.
The Waszczak laboratory has worked many years with Copernicus to establish that DNA nanoparticles can be used to non-invasively deliver a therapeutic gene for the treatment of brain disorders, such as Parkinson’s disease. And now with the expertise of Drs. Byrnes and Pothos, the team is excited to expand into targeting opioid use disorder. This grant may not only provide the path to a new gene therapy approach to treat opioid use disorder, but also a template for exploring gene therapy approaches for other substance use disorders and the cellular and molecular mechanisms that are involved in this novel and promising approach. This NIDA grant provides the opportunity for an industry/academic team to work together to address a serious public health issue.
“We are pleased to continue to work with these talented academic scientists in utilizing the DNA nanoparticles developed by Copernicus to treat serious brain disorders, such as opioid use disorder,” stated Mark J. Cooper, MD, Senior VP of Science and Medical Affairs of Copernicus, “and we believe this grant award enables the team to complete important steps necessary before this promising approach can be tested by Copernicus in human trials.”
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Copernicus Therapeutics, Inc. is a U.S.-based privately held clinical stage gene therapy company with a non-viral, nucleic acid platform technology. Copernicus’ non-viral gene therapeutics programs are designed to address diseases of the brain, eyes, and lungs.