List of Gene Therapy Companies in Canada - 30

Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis (ALS). ALPHA-1062, a patented new chemical entity that has demonstrated safety and improved tolerability in human clinical trials. It is being developed as a new acetylcholine esterase inhibitor for the treatment of dementia of the Alzheimer’s type, with minimal gastrointestinal side-effects and novel routes of administration. ALPHA-0602 is a gene therapy program delivering progranulin, a neurotrophic protein. It is in preclinical development for the treatment of ALS. ALPHA-0602 is patented and has received Orphan Drug Designation from the FDA.

Amplitude Ventures is a capital catalyst for highly innovative companies at the point of value acceleration. We work with Canada's most promising healthcare companies that share our vision of bringing groundbreaking technologies to patients. Amplitude is focused on building world-class Canadian companies in precision medicine and next-generation medical devices. Since 2012, the Amplitude team has deployed a venture growth model used to build Canadian companies with world-class management teams and scale companies to breakout potential. This model has been used by the team to create, build, and grow companies, resulting in over $1 billion in company value. Our offices are in Montreal, Toronto, and Vancouver.

Aspect Biosystems is a biotechnology company pioneering the development of bioprinted tissue therapeutics to transform the treatment of currently incurable diseases. Aspect is creating these next generation cell therapies by applying its full-stack tissue therapeutic platform, which combines the company’s proprietary bioprinting technology, therapeutic cells, biomaterials, and computational design. Aspect’s bioprinted tissue therapeutics are designed to replace, repair, or supplement biological function inside the body with the aim of treating currently incurable diseases such as type 1 diabetes as well as genetic and acquired liver diseases.

C3i is a one-stop shop in the development chain of cell & gene therapy. With a proven track record of 99% manufacturing success rate across over 300 clinical products, we offer in-house, turnkey services to ensure a streamlined process, eliminating the need to transfer your technology between multiple sites. With over 15 years of expertise in diverse cell manufacturing, we deliver cost-effective solutions ensuring a seamless and successful journey from process development to commercialization.

Regenerative Medicine (RM), which aims to harness the power of stem cells, biomaterials and molecules to repair, regenerate or replace diseased cells, tissues and organs, has the promise to treat, manage and perhaps cure some of the most devastating and costly diseases in the world today. Many new and potentially life-changing RM-based treatments never reach patients because they are not successfully moved from the laboratory to a stage where they can be used in medicine. In order to fulfill RM’s promise to treat the many diseases affecting our population, a world-renowned group of stem cell scientists and engineers have come together to form CCRM, a leader in developing and commercializing regenerative medicine and cell therapy technologies. CCRM formally launched on June 14, 2011. CCRM supports the development and commercialization of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy.

CryoStasis is a biotech company specializing in bio-preservation of cells, tissues and organs. Located in Ottawa and based on 30 years of pioneering research in the Canadian Arctic, we have developed a method that overturns the long-held belief that biological material must be frozen for its function to be preserved. By using rationally designed cryoprotectants (Woolii™Cool media) along with temperature and pressure management systems (Woolii™Pod), our platform drops temperatures well below freezing, halting cellular metabolism WITHOUT TURNING WATER INTO ICE. Maintaining sub-zero, unfrozen conditions induces stasis (indefinite preservation) while avoiding the dramatic loss of cell function, therapeutic potency and manufacturing yield that occur as a result of freeze-thaw damage. CryoStasis works with BioPharma, CDMOs and research institutions to help ensure life-changing cell & gene therapies can be produced at-scale economically, and reach patients whenever / wherever they are needed.

DalCor Pharmaceuticals: DalCor Pharmaceuticals is pioneering precision medicine for patients with cardiovascular disease. By combining genetic and clinical insights, the company’s development program, dalcetrapib, is intended to reduce fatal and non-fatal myocardial infarction (MI) following a recent acute coronary syndrome and deliver superior cardiovascular outcome in a specific genetic subset of patients.

Dalhousie University is an academic institution that offers a wide range of undergraduate and graduate programs along with research opportunities.

enGene Inc. has developed a highly flexible nucleotide (DNA and RNAi) delivery technology that targetsmucosal tissues and can be used to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels. Our platform technology has the ability to significantly impact diseases of mucosal tissues such as the gastrointestinal tract, lung and bladder as well as provide systemic release of proteins from the gut to treat diabetes, anemia, hemophilia and others.

ExCellThera is an advanced clinical-stage company specializing in cell expansion and engineering, providing molecules and bioengineering solutions to expand stem cells.

Kingston Health Sciences Centre is a healthcare provider that offers a range of medical services and healthcare solutions.

MAGIC Clinic is a healthcare clinic that provides medical services and appreciates its staff's achievements.

The McGill University Health Centre (MUHC) is a bilingual academic health network, and one of the largest and most modern in North America. We provide tertiary and quaternary care to the population of Montreal and Quebec. In other words, care that requires hospitalization, complex surgery, emergency care or specialized treatment.

McMaster University is a research university that provides post-secondary education and academic services.

Cell and gene therapy promises to one day cure any pathology; Modulari-T’s MARC Platform produces the tools to fulfill that promise.

Morphocell Technologies Inc. is a regenerative medicine company focused on the development and commercialization of stem cell therapies and engineered tissues targeting liver failure. The Company is a spin-off of the Hepatology and Cell Therapy lab of Sainte-Justine University Hospital Center, in Montreal. Founded in April 2018 on the technology developed by Dr.Paganelli, a pediatric transplant hepatologist and stem cell scientist, Morphocell Technologies aims at bringing to market the first effective treatment for acute, chronic and acute-on-chronic liver failure.

NanoVation Therapeutics is a pioneer in the design and creation of lipid nanoparticles (LNPs) for nucleic acid delivery. The company’s LNP technologies aim to overcome the limitations of existing nucleic acid delivery approaches, including the ability to target tissues outside the liver (extrahepatic delivery). NanoVation’s lead technology is its proprietary long-circulating LNP (lcLNP™) platform, which enables functional nucleic acid delivery to extrahepatic cell types and has demonstrated an improved therapeutic index in preclinical studies. NanoVation partners with leading pharmaceutical and biotech companies develop genetic medicines for previously untreatable diseases. NTx is delivering tomorrow's genetic medicines, today. For more information, please visit nanovationtx.com.

Notch is an early-stage biotech company, working to maximize the benefit of cell therapies through a proprietary T cell-production platform which combines sophisticated product design with commercial-compatible processes to enhance patient outcomes. Notch’s iPSC-based technology platform allows for precision control of notch signaling, which removes several critical limiting factors in the development of cell therapies, delivering the ability to design and manufacture a uniform and unlimited supply of therapeutic T cells.

Oncolytics Biotech Inc. is developing pelareorep, a safe and well-tolerated intravenously delivered immuno-oncolytic virus (IOV) that targets cancer through a unique mechanism of action with two components, selective tumor lysis and activation of the innate and adaptive immune systems, creating an inflamed phenotype to treat a variety of solid tumors and hematological malignancies.

Precision NanoSystems, now part of Cytiva, is a global leader in ushering in the next wave of genomic medicines in infectious diseases, cancer, and rare diseases. We work with the world's leading drug developers to understand disease and create the therapeutics and vaccines that will define the future of medicine. PNI offers proprietary technology platforms and comprehensive expertise to enable researchers to translate disease biology insights into non-viral genomic medicines.

Ranomics offers high throughput solutions for protein engineering, drug discovery, genomics research and synthetic biology. Using our VariantFind and VERG genomics platforms, Ranomics offers partners the a scalable solution to engineer and screen genetic variants in a wide range of model organisms. Our industry leading DNA Variant libraries and high-throughput cell assays help researchers to hit their research objectives faster and with better results than traditional methods. Over the years, we have worked with top biotech and pharma companies in the US and worldwide. Our partners have used our technologies to advance their R&D and drug discovery projects, and to publish papers in Nature Biotechnology, mAbs, and other journals.

Replicor Inc. is a Canadian biotechnology company focused on the development of its nucleic acid polymer (NAP) technology for the treatment of hepatitis B (HBV) and delta (HDV) infections. NAPs are the only antiviral agent to directly target the immunosuppressive protein (HBsAg) which drives chronic HBV infection and liver disease. Our lead clinical NAP drug product is REP 2139-Mg, a proprietary formulation of REP 2139 which makes once weekly subcutaneous injections easily tolerated. Replicor's latest clinical data demonstrates that NAP-based combination therapy can restore immune control of HBV infection in 78% of patients and eliminate HDV infection in 74% of patients. These effects persist in the absence of any therapy, allowing the liver to heal. REP 2139-Mg based combination promises to revolutionize the therapy of chronic HBV and HDV infection and play a pivotal role in the WHO's goal of eliminating viral hepatitis by 2030.

Sendum offers Advanced Therapy Visibility Solutions for Life Science companies. We help our customers maintain product integrity and visibility across their value chain, reducing their losses by up to 70%. Sendum’s advantage is our real-time visibility enabling a proactive response. We are the industry leader with over 60% of all active CGT clinical trials and commercially available CGT products.

As Canada’s engaged university, SFU works with communities, organizations and partners to create, share and embrace knowledge that improves life and generates real change.

Virogin Biotech is a biotechnology company that specializes in oncolytic virus drug development to create impactful drugs for patients with the highest medical need.

Developing partial reprogramming gene therapies