Welcome/Bienvenue
IGUANa (Interventional Genetics Unit At the Neuro) is dedicated to finding, investigating, and treating rare neurodevelopmental disorders. We use genetic strategies to target the underlying cause of disease, and we are based at the Montreal Neurological Institute (the Neuro) and McGill University. Our expertise is in developing and delivering care for children with neurodevelopmental disorders that provide meaningful improvement in their lives.
Rare disorders collectively affect about 2 percent of the population but are individually rare. Many of these rare diseases are caused by changes in DNA that affect a single gene and so may be amenable to state-of-the-art interventions to correct the underlying cause of disease. The vision of the IGUANa is to design, develop and deliver new therapies that can be given to patients with severe neurodevelopmental diseases where the underlying genetic cause is known. We aim to provide personalized care for debilitating and life-threatening diseases which have no current treatment. We are part of the Montreal Neurological Institute-Hospital (The Neuro) in Montreal, Canada which has significant expertise in brain imaging, nervous system biology, and clinical trial delivery. From the first steps of treatment design to single patient clinical trials, IGUANa is focused on delivering high quality and rapid care for severe diseases that are often overlooked.
IGUANa functions to integrate patient genetic data with rapid development of treatments, even in the first months of life. We work with referring hospitals to assess DNA changes that are known to cause disease to determine the feasibility of using specific classes of interventional genetic tools; specifically, mRNA therapy, oligonucleotide therapy, or CRISPR/Cas based therapy. We derive brain cells from patient’s blood and use these brain cells to assess therapies we design. These therapies target the mutation causing the disease in each specific patient. Once tested in a patient’s cells we iterate and modify the therapy to improve stability, specificity and efficacy, and test these therapies in other models to ensure safety. We coordinate the manufacturing and testing of this new therapy to the point that it is suitable for use in humans. Working with partners within The Neuro including the C-BIG, the EDDU, TOSI, the Clinical Research Unit, the Brain Imaging Centre, and those outside of our hospital including the McGill School of Social Work, Montreal Children’s Hospital, and St Justine Hospital, we develop the clinical trial protocols to allow us to take this therapy back to the patient and deliver it in a timely manner. We work with patient groups, governments, foundations, private enterprises, and philanthropic organizations to raise funds to support this work.
We occupy two floors of space at the Neuro where we can receive families for discussion, process cells, and convert somatic cells to brain cells. We are set-up to design and test any interventional genetics tool and assess downstream effects in brain cells. We are driven by the need to scale our approach where we can provide care for many patients with different rare disorders. We are committed to training the next generation of leaders in interventional genetics and hosting seminars and conferences related to this unique clinical trial framework for severe diseases. We contribute to international efforts in this area including the N1 collaborative and the Oligonucleotide Therapeutics Society.