TUESDAY, MAY 28, 2024 | 1:00 PM – 5:00 PM EST

The SickKids Innovation Showcase is an annual event that celebrates the most advanced health and life sciences ideas from the labs of SickKids researchers. Attendees get unparalleled access to emerging therapeutic and diagnostic technologies with high potential for market disruption and impact.

Innovative Health Technologies

A sneak peek of each of our highlighted technologies. Registered attendees will also receive our Showcase Pitch Book – a non-confidential brief for each of these technologies.

Therapeutics

Christine E. Bear, PhD 

Senior Scientist, Molecular Medicine | Co-Director, Cystic Fibrosis Centre | Professor of Physiology, University of Toronto 

Chronic obstructive pulmonary disease (COPD) is a lung disease affecting over 11 million Americans, for whom there is currently no cure. Current treatments for COPD aim to manage symptoms using bronchodilators (to improve airflow) and antibiotics (to eradicate pulmonary infections), however, more direct therapies are needed that tackle the underlying disease. The Bear lab at The Hospital for Sick Children (SickKids) has discovered a new CFTR potentiator SK-9919 and its water-soluble analog SK-2272. These potentiators have demonstrated efficacy in ameliorating cigarette smoke extract induced mucus aggregation in primary human airway cultures. This alternative therapy represents an innovative treatment approach that directly modulates lung function to target early stages of COPD pathogenesis.   

Christopher Edmund Pearson, PhD

Senior Scientist, Genetics & Genome Biology | Canada Research Chair in Disease-associated Genome Instability

Genetic expansions of repeat sequences, like CAG tracts, have been linked to at least 40 neurodegenerative, neurological, and neuromuscular diseases, such as Huntington’s disease (HD). The inherited repeat expansion continues to somatically expand in affected tissues as the individual ages, resulting in earlier age of disease onset, increased disease progression, and severityThe Pearson lab at The Hospital for Sick Children (SickKids) has identified Naphthyridine Azaquinolone (NA), a small molecule that targets somatic CAG/CTG expansions. Dr. Pearson and his collaborators have demonstrated NA can reverse disease onset, progression, and severity of Huntington’s disease in animal models. This approach unlocks a new therapeutic strategy for many genetic expansion disorders for which there are currently limited treatment options and no cure. 

James J. Dowling, MD, PhD, FANA

Senior Scientist, Program of Genetics and Genome Biology | Professor, Departments of Paediatrics and Molecular Genetics, University of Toronto

Skeletal muscle accounts for approximately 40% of our total body weight and plays a critical role in whole-body health and disease progression, making it a key focus for therapeutic innovation and advancement. Given the constraints of viral vector delivery, there is an urgent need for an efficient, safe, and minimally invasive approach to target adult skeletal muscle cells. Researchers at The Hospital for Sick Children (SickKids) have created a novel regimen for gene therapy delivery to skeletal muscles, using a clinically relevant administration method applicable to patients with skeletal muscle disorders. This innovative treatment relies on LNPs, offering a promising alternative to viral-based gene delivery techniques. Moreover, this formulation can encapsulate large gene constructs, overcoming historical challenges in the field. This new treatment approach has demonstrated no evidence of dose-limiting toxicity and is cleared from circulation within just one-week post-dosing, offering hope for effective and well-tolerated therapeutic interventions.  

Xi Huang, PhD

Senior Scientist, Developmental and Stem Cell Biology | Canada Research Chair in Cancer Biophysics

Glioblastoma (GBM) is the most common and aggressive brain cancer. The median survival of GBM patients is 15 months, despite a multi-modal standard-of-care that combines surgery, radiation, and chemotherapy. Chemoresistant GBM drives disease relapse and is universally lethal. Therefore, treating chemoresistant GBM is an urgent yet unmet clinical need. Dr. Xi Huang at The Hospital for Sick Children (SickKids) and his lab discovered a novel ion channel complex present only in GBM cells, and invented a designer peptide that can disrupt the ion channel complex. Furthermore, they defined a unique mechanism of action by which the designer peptide selectively kills GBM cells and activates the tumour immune microenvironment. Remarkably, the designer peptide is highly effective in treating GBM in a large panel of preclinical mouse models, including mice bearing tumours refractory to conventional chemotherapy, addressing a critical clinical need. 

Medical Devices & Diagnostics

Adam Shlien, PhD, FCCMG 

Senior Scientist, Genetics & Genome Biology | Garron Family Chair in Childhood Cancer Research | Associate Professor, Laboratory Medicine and Pathobiology, University of Toronto

The traditional biopsy-based cancer workflow is slow and subjective. To provide patients with better treatment plans and prognosis, there is a pressing need for techniques that facilitate faster, accurate, and earlier diagnosis of cancer. NewCode Oncology, a new spinout company from the lab of Adam Shlien at The Hospital for Sick Children (SickKids), harnesses the diagnostic advantages of RNA to transform the traditional cancer workflow. Already in use for SickKids patients, NewCode Oncology leverages the high accuracy and scalability of its machine learning (ML) platform to benefit oncology patients worldwide. SickKids researchers envisage a “next generation digital oncology workflow” where RNA sequence-based assays are done early in the diagnostic workflow. Enhancing traditional methods, NewCode Oncology offers a cutting-edge solution for rapid, accurate, and advanced cancer diagnosis and treatment, leading to improved patient outcomes. 

Jayne S. Danska, PhD

Senior Scientist, Genetics and Genome Biology | Anne and Max Tanenbaum Chair in Molecular Medicine | Associate Chief, Faculty Development and Diversity

Administration of the T cell receptor-specific antibody teplizumab, has shown promise in delaying the onset of disease in individuals with pre-type 1 diabetes (T1D) by more than two years (Herold NEJM, 2019). However, this trial also revealed variations in how participants responded to the treatment, highlighting a gap in our understanding of markers that could better predict the timing of future T1D diagnoses and the effectiveness of teplizumab. To address this gap, the Danska group at The Hospital for Sick Children (SickKids) conducted research on antibody responses to normal intestinal bacteria (commensal bacteria) in the blood of trial participants, both before and after they were assigned to treatment groups. Their analysis identified antibody responses to three specific bacterial species present at the outset of the study, which were linked to the time it took for T1D to develop and how individuals responded to teplizumab treatment. This groundbreaking research establishes a connection between antibody responses to intestinal bacteria and the development of anti-islet autoimmunity and future T1D progression, suggesting that these responses could serve as valuable biomarkers for predicting T1D progression and response to immune therapies. 

Luc Mertens, MD, PhD

Section Head, Echocardiography | Professor, Department of Paediatrics, University of Toronto

Cardiac dysfunction is the leading cause of death worldwide. To better diagnose and manage conditions like heart failure or cardiomyopathy, a reliable biomarker for myocardial work is essential. Addressing this need, the Mertens and Villemain labs at The Hospital for Sick Children (SickKids) have developed a novel digital biomarker capable of evaluating how well the heart works in response to stress. This approach relies on innovative ultrasound technology capable of swiftly evaluating rapid and transient events within the heart. This digital biomarker is set to transform cardiac diagnostics and the evaluation of heart function in response to treatments. 

Steven Prescott, MD, PhD

Senior Scientist, Neurosciences & Mental Health | Professor, Department of Physiology, University of Toronto

Less than one in 10 drugs entering clinical trials succeed, with the majority failing in phases two and three due to lack of efficacy. This trend persists despite promising preclinical data, indicating shortcomings in preclinical testing. Criticism of animal testing reproducibility has been particularly pointed, especially in pain research. A two-decade-old study revealed that the experimenter conducting the pain test significantly influenced its outcome. Yet the status quo has remained unchanged, with stimuli applied manually and withdrawal responses assessed visually. Researchers in the Prescott lab at The Hospital for Sick Children (SickKids) have developed a robot capable of replacing human testers in pain research. Equipped with artificial intelligence (AI), the robot identifies mouse paws and administers precisely controlled stimuli while monitoring pain responses. This approach significantly enhances protocols in terms of standardization and throughput. Furthermore, AI analysis of video footage collected during testing allows for nuanced quantification of pain behaviours. This technology promises to transform preclinical pain testing, enabling better prediction of which drugs are likely to succeed in clinical trials. 

The SickKids Innovation Showcase is Generously Sponsored by:

Discover Highlights from our 2023 Event

On May 18, 2023, our Industry Partnerships & Commercialization office welcomed close to 200 guests to the first in-person annual event. Held in the Peter Gilgan Centre for Research and Learning, attendees saw six groups of SickKids researchers present their innovative therapeutic technologies to a panel of health and life sciences investors. Each presenter was tasked with answering probing questions from our panelists, demonstrating the utility and market potential of their innovations. Attendees also took part in a lively networking reception following the event to connect with the health and innovation community in attendance, and to further discuss advancing these promising therapeutics.

From novel therapies to fight bacterial diseases to ground-breaking treatment options that target aggressive human cancers, presentations at this year’s Innovation Showcase serve as a strong reminder of SickKids’ trailblazing efforts in paediatric health care. The Industry Partnerships & Commercialization office is proud to play a role in the advancement of these innovations, and grateful for the opportunity to host an event that puts them on display.

Thank you to all presenters, panelists, sponsors and attendees for making the 2023 SickKids Innovation Showcase a success!

2023 Panelists

Jacki Jenuth, PhDPartner
Juliana Muñoz Escobar, PhDAssociate
Katie Spielberg, PhDAssociate
Kuldeep Singh Neote, PhDEntrepreneur-in-Residence

2023 Innovative Health Technologies

A sneak peek of each of our highlighted technologies. Registered attendees will also receive our Showcase Pitch Book – a non-confidential brief for each of these technologies.

Martin Post, Ph.D.

Senior Scientist, Translational Medicine
Professor, Departments of Paediatrics, Physiology, and Laboratory Medicine & Pathology, University of Toronto

Michael Litvack, M.Sc., Ph.D.

Research Associate, Translational Medicine

Sheena Bouch, Ph.D.

Research Associate, Translational Medicine

Patients with lung cancer have some of the worst prognosis of all cancers and are in need of more effective therapeutic treatments. The Post lab at The Hospital for Sick Children has harnessed the power of the lung innate immune system by developing a method for the in vitro differentiation of human stem cells into human Alveolar-Like Macrophages (ALMs). These ALMs have been engineered to limit the propagation of the “don’t eat me” signal commonly expressed by tumour cells, allowing for phagocytosis of the tumour cells. Furthermore, they can be targeted to tumour cells while remaining sequestered from the rest of the body. This could prevent many of the off-target effects observed with systemic immunomodulatory antibody and T-cell directed therapies. The Post lab has also successfully delivered their ALM-based therapy through various pulmonary administration methods in pre-clinical models. These uniquely engineered ALMs represent a novel cell therapy platform that could revolutionize the way lung cancer and other lung diseases are treated.

Xi Huang, PhD

Senior Scientist, Developmental and Stem Cell Biology
Canada Research Chair in Cancer Biophysics

Glioblastoma (GBM), which accounts for 60% of adult brain tumours, is the most common and aggressive brain cancer with a median survival of 15 months. The standard of care combines surgery, radiation, and chemotherapy using the DNA alkylating agent temozolomide (TMZ). More than half of GBM patients develop TMZ resistance. Dr. Xi Huang (The Hospital for Sick Children) and his lab recently discovered a novel ion channel complex expressed only in GBM cells that promotes tumour growth. They used multiple mouse orthotopic xenograft models derived from i) patient-derived TMZ-sensitive cells, and ii) patient-derived TMZ-resistant cells, and targeted the ion channel complex with a novel designer peptide administered through cannula mediated delivery. Remarkably, the designer peptide demonstrated robust killing of both types of GBM cells and extended the survival of GBM-bearing mice, thereby addressing a tremendous unmet clinical need. This novel peptide, which acts through a unique and unprecedented mechanism of action, provides an effective method which may be translated into a new drug to treat GBM at a global scale.

Nicola Jones, M.D., FRCPC, Ph.D.

Senior Scientist, Cell Biology
Staff Physician, Division of Gastroenterology, Hepatology and Nutrition
Professor, Departments of Paediatrics and Physiology, University of Toronto

Helicobacter pylori (H. pylori) infects the stomach of half of the world’s population. It is recognized by the World Health Organization as a carcinogen that causes gastric cancer – the third leading cause of cancer-related deaths worldwide. Current treatment for H. pylori fails to reach the recommended eradication rates due to increasing antibiotic resistance. Furthermore, no current therapy targets the pool of bacteria that produce the virulence factor vacuolating cytotoxin (VacA), which live inside gastric cells and serve as a reservoir for persistent infections and gastric cancer. Dr. Nicola Jones (The Hospital for Sick Children) and her research team discovered that VacA inhibits a protein called TRPML1 to protect the bacteria from antibiotic treatment, leading to infection recrudescence after therapy. The Jones lab has identified an orally available novel gut-restricted small molecule that can reverse this phenomenon by activating TRPML1. They have successfully demonstrated that TRPML1 activation can eradicate intracellular H. pylori in infected cells and have also shown their novel compound can control the progression of infection in vivo. With appropriate support from a development partner, this promising gut-restricted compound is only 12 months from an IND submission.

Roman Melnyk, Ph.D.

Senior Scientist, Molecular Medicine
Co-Director, SPARC Drug Discovery Facility
Associate Professor, Department of Biochemistry, University of Toronto

Clostridium difficile (C. difficile) is the leading cause of hospital acquired diarrhea and antibiotic associated colitis, affecting 500,000 people annually and incurring $5B USD of inpatient hospitalization costs. Once colonized in the gut, C. difficile releases a highly potent toxin called TcdB that destroys the colonic epithelial barrier, leading to watery diarrhea, fever, nausea, and abdominal pain. Studies show neutralizing TcdB alone is sufficient to prevent primary C. difficile infection and recurrence, however, current treatments targeting TcdB are expensive, only moderately effective, and are difficult to administer. Capitalizing the therapeutic potential of the TcdB target, the Melnyk lab (The Hospital for Sick Children) and Dosa lab (University of Minnesota), synthesized novel gut-restricted bile acid variants to inhibit TcdB and treat C. difficile infection. Initial testing of a promising candidate molecule revealed it is a highly potent inhibiter of TcdB in vitro, is non-toxic to human cells and prevents disease recurrence in in vivo mouse models. Gut-restricted bile acids are promising next-generation therapies for treating C. difficile infection.

Jason T. Maynes, M.D., Ph.D.

Chief, Department of Anesthesia and Pain Medicine
Curtis Joseph and Harold Groves Chair in Anesthesia and Pain Medicine
Associate Chief of Perioperative Services
Research Scientist, Molecular Medicine

John Coles, M.D., FRCSC

Cardiovascular Surgeon, Labatt Family Heart Centre
Senior Clinician-Scientist, Translational Medicine
Professor, Department of Surgery, University of Toronto

Heart failure is the single largest contributor to morbidity and mortality in the developed world, affecting 1-2% of the adult population. However, current treatments are limited to targeting symptomatic pathways and do not improve organ function. Evident in most forms of heart failure, cardiac fibrosis accelerates patient mortality but is currently untreatable. Integrin-linked kinase (ILK) is a key cardiac scaffolding protein involved in heart development, transduction of mechanical stress between the extracellular matrix and the intracellular cytoskeleton, and induction of cardiomyocyte survival pathways. Research in the Coles and Maynes labs (The Hospital for Sick Children) have identified inhibitors of ILK binding to α-parvin, demonstrating that inhibition of the interaction rescues cardiac function. Their first-in-class drug disrupts a promising new therapeutic target for heart failure and fibrosis patients.

Jennifer Crosbie, Ph.D., C.Psych

Psychologist, Department of Psychiatry
Susan J. Bradley Health Clinician Scientist
Associate Scientist, Neurosciences & Mental Health
Associate Professor, Department of Psychiatry, University of Toronto

Attention Deficit Hyperactivity Disorder (ADHD) is a childhood onset clinical disorder, with characteristic symptoms of inattention, hyperactivity and impulsivity. Although hyperactivity tends to decrease with age, inattention and impulsivity continue to be frequent problems throughout adulthood. Cognitive-based video games have been shown to improve the symptoms of not only ADHD, but other neurodevelopment disorders (i.e. autism) in which patients struggle with executive function and emotion regulation. Drs. Jennifer Crosbie and Russell Schachar (The Hospital for Sick Children) have created a video game-based assessment and rehabilitation software. The application, which recently underwent a randomized clinical trial with over 200 patients, aged 6 to 12, involves the embedding of cognitive assessment and rehabilitation tasks within an engaging video game environment. It trains cognitive control and working memory for ADHD patients and other neurodevelopmental disorders with executive function deficits. The digital therapeutics market is valued at approximately 4.2B USD, as interest in harnessing technology to supplement or potentially replace traditional clinical therapy grows. As a form of rehabilitation therapy intelligently disguised as an interactive video game, this technology has strong potential to improve the quality of life of millions of patients worldwide who struggle with neurodevelopmental disorders.

2023 Sponsors

Thank you to our 2023 sponsors for their generous support.

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