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.

2024 Panelists

Aileen Salares, MBA CFAPrincipal New Product Planning Apple Tree Partners
Kevin McBride PhDGeneral Partner & CSO AmorChem Ventures
Paul Cassar, MBA PhDAssociate, CTI Life Sciences Fund
Matthew Mistry, MScSenior Associate & Head of Therapeutics

2024 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:

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