Science
Welcome to another episode of Biotechnology Focus radio! I am your host – Michelle Currie – here to give you the rundown on what is happening in biotech across the country from coast to coast. There have been some interesting developments in the last couple weeks that are changing the scope of the life sciences industry. Some of which I get the pleasure to share with you today. As a first for Canada, Concordia University now houses a facility that will change how synthetic biology research will be conducted; Bioasis Technologies’ promising drug development may have found a way to cross the blood-brain barrier; the Centre for Commercialization of Antibodies and Biologics invests in ImmunoBiochem to advance their therapeutic candidate; and the Canadian government, as well as other investors, allocate $8.8 million to three projects in Ontario. Keep on listening to find out more details! +++++ A new facility at Concordia is about to change history. It will house robots that will bring a whole new concept of speed and scale to synthetic biology research. The Genome Foundry is the first Canadian laboratory of its kind, and amongst only a handful at leading institutions around the world. By automating notoriously labour-intensive lab work, it will eliminate bottlenecks in a rapidly evolving field where the design principles of engineering fuse with the tools of biology to create meaningful synthetic biological systems. Christophe Guy, vice-president of Research and Graduate Studies at Concordia says that the Genome Foundry solidifies Concordia’s position as the Canadian leader in synthetic biology research and will enable their scientists to work at the cutting-edge while facilitating partnerships with other institutions. Given that Concordia researchers are already engaged internationally in defining the future of this field, they are eager to witness how this new facility will support the transformative work being done at the university. At the moment, much of the lab work done by synthetic biologists involves moving and combining small amounts of liquids and cells. The Genome Foundry’s robotics will allow for speed and absolute precision, thus greatly increasing the variety and number of experiments that can be completed, and the accuracy with which they can be reproduced. The Genome Foundry was established with funds from the Canada Foundation for Innovation and the government of Quebec and is part of Concordia’s synthetic biology hub along with the Centre for Applied Synthetic Biology (CASB), the SynBioApps NSERC CREATE program and the soon-to-be-inaugurated District 3 Innovation Centre science hub. Vincent Martin, co-director of the Centre for Applied Synthetic Biology says that they are thrilled to open the doors of our Genome Foundry. That this is a monumental addition to Canada’s synthetic biology ecosystem. It empowers researchers to navigate uncharted waters alongside international colleagues, and to incubate the future leaders of the field. The Centre for Applied Synthetic Biology aims to develop high-value applications in human health, agriculture, chemicals and environmental technologies. It also provides a broad range of unique opportunities — such as the recently announced NSERC CREATE SynBioApps program — for training leading experts in the field. Launching this technology platform also marks Canada’s participation in the next generation of synthetic biology, with Concordia now engaged in directing how this infrastructure will be developed and used on a global scale. This facility will have real world, potential life-saving capabilities that deliver an innovative scientific approach to create genetic blueprints for individuals, bring more knowledge to researchers on a faster scale, and help physicians diagnose, treat and prevent their patients from contracting future diseases. +++++ With neurological diseases predicted to rise exponentially across the globe, whether resulting from the extension of life expectancy or aging populations, more novel solutions are necessary so that health care can stay ahead of the game. Neurological diseases, disorders and injuries – such as Alzheimer’s disease, amyotrophic lateral sclerosis, multiple sclerosis, brain tumours, and Parkinson’s disease – are some of the leading causes of disability amongst the Canadian population that take a toll not only on the patient and the Canadian health care system, but also have a significant economic impact. To date, these neurological diseases and disorders have been largely incurable and tend to worsen over time, typically involving invasive procedures by scientists and researchers as they attempt to penetrate the blood-brain barrier. Remarkable as the blood-brain barrier is to neuroscience, it is extremely fickle and highly selective, restricting the paracellular diffusion of water-soluble substances from the blood to the brain. Despite it being nature’s evolutionary way of protecting humans’ greatest asset, it does not come without its faults. Its defensive properties impede the way for medicinal compounds to penetrate the barrier and deliver the potential life-saving properties to their destination point. Statistics have shown that 1 in 6 will acquire a neurological disease, totalling about 1.25 billion people worldwide. It is for this reason Canadian company Bioasis Technologies Inc. is determined to deliver effective treatments to patients who suffer from one of these diseases. Vancouver-founded Bioasis has undertaken this challenge by focusing on a single goal: revolutionizing science by transporting therapeutic payloads across the blood-brain barrier and into the brain. They have developed and are in the process of commercializing their proprietary brain delivery technology, the xB3 platform, to make life-saving drugs brain-penetrant and deliver those therapies at a therapeutically relevant dose. Inception of the company began back in 2007 when researchers discovered an extremely large peptide that was capable of crossing the blood-brain barrier with a substantial amount of cargo. The team did a couple of experiments with Doxorubicin in mice models with cancer that positively showed higher survival rates and became the first proof of concept. The also acquired Trastuzumab data whereby they transferred Herceptin across the blood-brain barrier in sufficient quantities to reduce the number of tumours. Although researchers have been speculating about less invasive methods that will penetrate the barrier, Mark Day, the president and chief executive officer of Bioasis, comments that the key thing if you have brain cancer is that the only therapeutic benefit will come from a direct infusion into the brain – like drilling a hole in the head – and while some people are trying to inject into the spine and pump it into the central nervous system, none of it has worked. The brain methods do work, so there is at least some data if you inject it that you can get it approved for efficacy for small groups of patients. Recently, MedImmune, a wholly-owned subsidiary of AstraZeneca, did an independent validation of Bioasis’ xB3 platform technology that transpired incredible results. The study found that the xB3 fusion protein maintained the systemic pharmacokinetics of its payload and had significantly improved and sustained brain exposure of the payload molecule. It provided evidence that Bioasis’ platform technology was recombinant and chemically conjugated drugs across the blood-brain barrier to increase brain exposure. These data and validation from MedImmune provide promising results that it will work in a phase 1 study. Bioasis figured out that once they attached Trastuzumab to 12 active amino acids (peptide 12aa), 10 times the amount of the drug passed through the blood-brain barrier. Mark Day adds: “What is really important is that once the drug is in the brain it hits the tumour. Looking at these results you can see that there are significant therapeutic doses in the brain and in controlled regions. This shows us that the drug gets into the brain, it gets to the site of action, and binds to those specifically where there are HER2 positive cells.” Bioasis has four main programs: xB3-001: Brain Metastases, which is the most common form of brain cancer in adults and is often fatal due to anti-cancer drugs being unable to pass the blood-brain barrier, and is also the program that will progress first to human trials in 2019; xB3-002: Glioblastoma, one of the most aggressive cancers that originates within the brain, with 80 per cent of diagnosed primary malignant brain tumours as malignant gliomas. It is considered the deadliest form of brain cancer due to its high infiltration of surrounding brain tissue. This program is being done in collaboration with Minerva in Copenhagen; xB3-007: Neurodegenerative diseases, which entail a progressive loss of function by the neurons in the brain and in being diagnosed at an alarming rate partly due to an aging population; and xB3-008: Lysosomal storage diseases, which are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. If the xB3-001 and xB3-002 programs are successful, it would be the first time in human history that medicine for cancer has been properly received into the brain without having to drill into the patient’s head. This will be a breakthrough in science and open the doors to a floodgate of scientific possibilities. Bioasis’ technology platform has been so efficacious that there simply have not been any competitors that have been able to keep up. The receptor with which they work with is ubiquitous to the blood-brain barrier walls, providing more possible passageways for medicine to penetrate through. This receptor, even in its natural form, is critical in cleaning out harmful tissues in the brain like Alzheimer’s disease for example and is necessary to maintain brain integrity. Mark Day adds that the other thing that differentiates them is how they develop drugs. They know that if you engage the target and prove that the target engagement drives biologic effect – to schizophrenia that would be a lowering of dopamine – then you have a good sense of patient population. So, for some of these diseases, there is a very strong genetic basis to them and subsequent diseases that gives them the mechanism to recruit the patient who is most likely to benefit from the medicine in the first tranche. The proof of principal in the first point, get the proof of concept, on the back of a positive proof of concept then you would go earlier into the diseases. That’s what they can do with our last two neurodegeneration products. Basically, they go into a smaller niche indication, get the proof of concept and then expand it into other disease areas. That’s been the strategy. With recent editions to their scientific advisory board and looking ahead to put them in the best financial position for Nasdaq, the future looks promising for this biotechnology company. Penetrating the blood-brain barrier has been an arduous task, but if Bioasis is successful, their technology will revolutionize the treatment for neurodegenerative diseases and brain tumours, potentially slowing the progression of disease, and maybe someday offer a cure. +++++ The Centre for the Commercialization of Antibodies and Biologics (or CCAB) provides a new investment to help advance ImmunoBiochem’s novel breast cancer therapeutic candidate one step closer to the clinic. The agreement marks a first for CCAB as part of its new business strategy, which aims to attract investment to create successful life sciences companies in Canada. CCAB will develop ImmunoBiochem’s lead candidate towards regulatory filings, and merge their business acumen with its research and technical expertise to support the co-development of new biological therapeutics. CCAB CEO Robert Verhagen says that today’s announcement marks the beginning of a very exciting period of growth. The agreement with ImmunoBiochem is a natural extension of an already fruitful partnership and that they are looking forward to helping the company get to the next crucial stage in the development of this promising anti-cancer therapy. As CCAB continues to expand its mission in this space, they plan on establishing similar partnerships with other emerging companies in the near future. ImmunoBiochem is developing novel potentiated biologics to treat triple-negative breast cancer (TNBC), an aggressive form of breast cancer for which there are currently no targeted biological treatment options. Earlier this quarter, ImmunoBiochem secured an additional private investment to support its pipeline and entered into a license agreement with the University of Toronto for novel therapeutic antibodies. ImmunoBiochem’s CEO Dr. Anton Neschadim says that ImmunoBiochem’s highest priority is to make new treatment options available for patients with this difficult-to-treat breast cancer. They have made significant progress and have validated their approach in vivo. CCAB has been tremendously supportive of their work and they are excited that this new agreement will help them advance their lead candidate even further. Breast cancer is the most common cancer among Canadian women and is the second leading cause of death from cancer. In 2017, 26,300 women were diagnosed with breast cancer and 5,000 women died from the disease. Triple-negative breast cancer accounts for up to 20 per cent of breast cancers and is one of the most heterogeneous diseases, comprising multiple breast cancer sub-types. Consequently, even highly promising treatments that are in late stages of the clinical pipeline are likely to only address the needs of a partial number of triple-negative breast cancer patients. ImmunoBiochem has developed therapeutic candidates that aim to close on this gap by overcoming treatment challenges associated with tumor heterogeneity. Much of biological therapeutics distinguish cancer cells from normal cells based on proteins differentially expressed on their surface. In solid tumours, most such targets are heterogeneously expressed, impeding complete responses and driving resistance and relapses. ImmunoBiochem is focusing instead on selective targets in the tumour microenvironment that are broadly present and interact with all cells in a tumour, including tumour-supporting stroma. ImmunoBiochem has shown that this approach could be more effective and safer than conventional surface-targeted therapeutics. This agreement between the two companies has the potential to lead to viable therapeutics that are sorely needed, especially for cancers that have a high rate of morbidity and mortality. +++++ Genome Canada announces federal funding for seven new projects under the Genome Canada’s Genomic Applications Partnership Program (GAPP), three of which hail from Ontario. This will be driving $2.9 million of federal funding into the province and an additional $5.9 million from investments in the industry, government, and funding partners. For a total of $8.8 million, this could heed rewarding results. The announcement was made by the Minister of Science and Minister of Sport and Persons with Disabilities. The Honourable Kirsty Duncan, at the Vineland Research and Innovation Centre. Vineland is partnering with a team of University of Toronto researchers to develop genomics-based technologies that will induce broad-spectrum disease resistance in greenhouse vegetables, allowing new varieties of vegetables to thrive and reducing waste. This will give growers across Ontario and Canada a competitive advantage in a national industry that already generates more than $1 billion annually from retail sales and exports. In another Genomic Applications Partnership Program project, researchers at McMaster University are partnered with Hamilton-based start-up Adapsyn Bioscience Incorporated to use its proprietary technology platform that combines genomic and metabolomic data with artificial intelligence and machine learning to redefine and accelerate drug discovery for novel treatments of a wide spectrum of diseases. This partnership secured significant foreign and domestic investment and is creating new high-tech jobs in Ontario. The third Ontario-based Genomic Applications Partnership Program project announced brings together researchers at the Sunnybrook Research Institute and the University of Toronto with Canadian start-up Fusion Genomics to further develop novel infectious disease surveillance tools. Their technology is unique in its ability to detect and genetically characterize infectious viral pathogens through bioaerosols to serve as an early warning for disease outbreaks in both humans and agricultural animals. The development of this pre-emergence environmental detection technology will drive a paradigm shift in public health and animal welfare by offering complete genomic data to anticipate outbreaks, inform disease transmission dynamics and enable vaccine design and production. Genomic Applications Partnership Program is a program that partners researchers with companies and other end-users who will apply their innovations with the goal of increasing and accelerating the positive social and economic impact of Ontario’s and Canada’s genomics R&D capacity. Since 2013, approximately $86.1 million, including co-funding has been invested in 23 Ontario-based Genomic Applications Partnership Program projects, fuelling innovations, spurring job creation and attracting foreign investment in Ontario’s health, agriculture & agri-food, fisheries, environment and natural resource sectors. In such an emerging industry, there is nothing better than seeing companies and research succeed. With these recent investments, there is high hope that we will see encouraging results in the future. +++++ Well that wraps up another episode of Biotechnology Focus radio! Thanks for listening! If you have a story or a story idea, feel free to contact me at press@promotivemedia.ca. Until the next time, from my desk to yours – this is Michelle Currie.