New DNA technology expected to cure genetic diseases
Stargardt’s disease is a rare inherited ocular illness that affects the Canadian population. According to Fighting Blindness Canada, 1 in 8,000 Canadians suffer from Stargardt’s, a degenerative ocular illness that ultimately leads to vision loss. Typically, patients are diagnosed with the disease by the age of 13, and most experience progressive vision loss as they get older. It is not uncommon for patients to develop complete blindness by the age of 35. Still today, despite the many clinical trials taking place, there is no cure for Stargardt’s disease.
New DNA technology brings hope to Canadians
Mediphage Bioceuticals intends to cure genetic diseases with innovative DNA technology that is revolutionizing genetic medicine — as we know it. Their technology is called “DNA Ministrings” and will allow health care practitioners to target affected cells in a patient with a personalized DNA sequence to cure chronic diseases. This method was designed to be the safest solution available without sacrificing effectiveness, emphasizes Mediphage.
“Genetic medicine is poised to be the greatest advancement in medicine of our lifetime,” according to Lee Bowman, Chief Operations Officer at Mediphage. “The technology being developed right now, including in the Mediphage labs, is the catalyst that will accelerate genetic medicine forward and make all the difference in eliminating genetic diseases and providing a better quality of life for all Canadians.”
Traditional gene therapy is typically viral-based, whereby a virus serves as the conduit through which genes are delivered to cells in the human body, according to Dr. Shawn Wettig, Associate Professor, School of Pharmacy at the University of Waterloo. In contrast, DNA Ministrings are double-stranded DNA vectors that deliver personalized genetic sequences to targeted cells.
This new technology is safer and more effective than viral gene therapy because it does not result in cancers or cause a dangerous immune response in the body which can potentially hinder any form of therapy or cure, explains Dr. Wettig. Since DNA Ministrings technology has been designed to be scalable and personalized to fit the needs of individual patients, it is readily commercialized for the treatment of a range of genetic illnesses and at different levels of progression.
Another key element to consider when discussing DNA Ministrings is manufacturability. Mediphage is hard at work ensuring that this technology can be readily available for the greater Canadian public, thereby benefitting both the health of many Canadians and the national economy.
“We are taking genetic medicine from an idea that is possible to something that is therapeutically achievable,” says Dr. Wettig.
Key partnership and professional training
The DNA Ministring platform is the flagship product of Mediphage Bioceuticals, a private company operating at JLABS in Toronto. Dr. Roderick Slavcev, Founder and Chief Science Officer at Mediphage, started this research in his lab at the University of Waterloo in 2008.
Today, the organization continues the joint effort with the University in partnership with Mitacs’s Elevate program, which offers businesses the opportunity to solve challenges with top-ranked researchers. Leading the initiative is postdoctoral researcher Dr. Nafiseh Nafissi, University of Waterloo. As VP of Research & Development at Mediphage, she runs the Toronto lab and is currently applying and validating the technology in the laboratory.
According to Bowman, the Mitacs research fellowship provides, in addition to much necessary funding, a comprehensive suite of professional training to support Dr. Nafissi’s work, including project management and leadership courses. Skills that have proven invaluable when one is responsible for managing the development of groundbreaking technology such as DNA Ministrings.
Application of genetic medicine
Mediphage continues to design and validate various types of genetic medicines to treat inherited ocular illnesses, such as Stargardt’s disease, which can often go untreated due to limited therapy options.
DNA Ministrings are designed to be used in a variety of therapeutic applications, including gene therapy, cell therapy, DNA vaccines, gene editing, and iPSCs (induced pluripotent stem cells) to treat myriad chronic genetic diseases, according to Bowman.
Consequently, such ventures have a positive impact on and help diversify Canada’s job market and attract international talent. “I can see the creation of highly technical jobs,” says Dr. Wettig.
Mitacs continues to support this research by offering lead scientists a network of resources that transform in-lab ideas and tests into applicable, manufacturable technology with key benefits to the Canadian population.
Mitacs thanks the Government of Canada and the Government of Ontario for their support of the Elevate research fellowship in this story. Across Canada, the Elevate program also receives support from the Government of British Columbia, the Government of Manitoba, the Government of New Brunswick, the Government of Newfoundland and Labrador, the Government of Nova Scotia, the Government of Ontario, the Government of Prince Edward Island, the Government of Quebec, and the Government of Saskatchewan.
Do you have a business challenge that could benefit from a research solution? If so, contact Mitacs today to discuss partnership opportunities: BD@mitacs.ca