Northeastern University, Copernicus Therapeutics, Inc. awarded $14.7 million NIDA/NIH grant to develop a gene therapy approach for treating opioid use disorder

This article was originally published on newswise.com.

  • Dr. Barbara Waszczak, Professor of Pharmacology at Northeastern University’s Bouvé College of Health Sciences, is the Principal Investigator and Project Director for the grant.
  • Copernicus Therapeutics, Inc. will be supplying the DNA nanoparticles that constitute the gene therapy approach to be used to treat Opioid Use Disorder.
  • Grant provides an initial Phase 1 funding of $6.7 million from the National Institute on Drug Abuse (NIDA), part of the National Institutes of Health, with a potential for additional Phase 2 funding up to a total of $14.7 million to further move the gene therapy towards clinical trials. Barbara Waszczak - Nusring innovation - Bouvé college of Health Sciences - Northeastern University

Newswise — CLEVELAND, March 31, 2022 /PRNewswire/ — 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 toward 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.”

About Northeastern University 
Northeastern is a global research university and the recognized leader in experience-powered lifelong learning. Our world-renowned experiential approach empowers our students, faculty, alumni, and partners to create impact far beyond the confines of discipline, degree, and campus. Through our growing global university network, we expand opportunities for flexible, student-centered learning and collaborative, solutions-focused research.

About Copernicus Therapeutics, Inc.
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.

Pilot data research was done with the support of awards from the Brain Research Foundation and Tufts Clinical Translational Science Institute.

This research is supported by the National Institute on Drug Abuse of the National Institutes of Health under award number 1UG3DA050942-01A1 . The content presented in this release is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.