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Medicine by Design invested $1-million in the Convergent Working Groups, that convened experts through a series of workshops to explore a range of ideas and possibilities related to regenerative medicine, encompassing community members and researchers from disciplines and institutions that may not typically be involved in regenerative medicine research.

Funded Convergent Working Groups

Novel approaches for transplantation in patient-derived induced-pluripotent stem cells for cell regeneration (Co-funded by MITO2i)

Lead Investigator: Ana Andreazza
Faculty of Medicine, University of Toronto

Mitochondria are parts of the cell responsible for generating energy. This Convergent Working Group will come together to discuss feasibly advancing mitochondria transplantation for successful applications in regenerative medicine. Mitochondrial dysfunction is involved in a variety of diseases, ranging from genetic mitochondrial disorders to chronic diseases such as diabetes, heart disease, and Alzheimer’s. Mitochondrial transplantation involves the addition of live and healthy mitochondria into the patient. The team will include researchers, clinicians, foundations, patients and patient families and industry partners all of which have a goal to advance mitochondrial related therapies to treat and cure disease.

Unleashing the Potential of RNA Nanomedicine to Promote Regeneration in End-Organ Diseases

Lead Investigator: Mamatha Bhat
Ajmera Transplant Centre, University Health Network

This Convergent Working group will discuss the challenges in translating RNA nanomedicine into the clinic to treat end-organ disease. By inviting local as well as international pioneers in RNA nanomedicine, clinical regenerative medicine and AI, they will develop a position paper that addresses how to control delivery of RNA-containing nanoparticles into cells of interest, and how these therapies can be made safe for ultimate clinical translation.They aim to exploit the growing expertise in RNA nanomedicine and bring it to the bedside to stimulate regeneration inpatients with chronic diseases.

A policy framework to enable the commercialization of living therapies

Lead Investigator: Shiri Breznitz
Munk School of Global Affairs & Public Policy, University of Toronto

Canada, especially the Greater Toronto Area, is one of the leading regions in living therapies research. However, policy to support the full-scale commercialization of these efforts is limited, which makes the translation pathway of collaborative teams, who are largely funded by provincial and federal agencies, unclear. This collaboration will bring together regenerative medicine researchers, clinicians, commercialization organizations, university and hospital administrators, institutional commercialization offices, and government representatives to discuss both the barriers for commercialization in Toronto, as well as to develop a policy framework to facilitate the commercialization of living therapies across Canada.

TOGETHER: Toronto Gene Therapy Network

Lead Investigator: James Dowling
Genetics & Genome Biology, The Hospital for Sick Children

As a key therapeutic strategy within regenerative medicine, gene therapies have potential to treat rare genetic diseases, which affect up to one in 12 Canadian families. This groups aims to make Toronto a world leading centre for rare disease gene therapy by identifying gaps and solutions to individualized gene therapy implementation, discussing equitable and ethical considerations, and determining optimal process and workflow from preclinical development through to clinical trials and ultimately, clinical implementation.

Building trusting relationship and efficient communication between African, Caribbean and Black (ACB) communities and research and clinical communities about regenerative medicine and living therapies (including cell therapies and organ transplant)

Lead Investigators: Carl E. James and Istvan Mucsi
Faculty of Education, York University
Ajmera Transplant Centre, University Health Network

This project focuses on building trusting relationships, mutual understanding,and efficient communication between African, Caribbean and Black (ACB) communities and professionals in the field of regenerative medicine. This collaboration will include community members and leaders, religious and spiritual leaders, and regenerative medicine researchers and professionals and engineering, psychology, social sciences, arts, public health and information technology experts. The Convergent Working Group will generate a call-for-action paper to represent ACB perspectives, goals, and priorities; and inform researchers, clinicians, and policy makers to facilitate better understanding and positive change.

Electrical stimulation (ES) for neural repair

Lead Investigator: Cindi Morshead
Professor and chair of the Division of Anatomy, Department of Surgery, University of Toronto

Neurological disorders are a major cause of disability and affect millions of people worldwide, with increasing prevalence with aging. ES has received renewed attention as a potential strategy to treat neurological disease and injury. This collaboration considers how we can achieve the goal of leveraging ES to accelerate discovery and early-stage translation of engineered, customized living therapeutics to enhance neuroplasticity and regeneration. A team of experts from regenerative medicine, electrical engineering, material science, design and manufacturing alongside patient facing practitioners will consider minimally invasive, economically feasible, accessible and scalable technologies to advance the potential of ES.

Stem cell-based therapy: a novel regenerative medicine approach for neonates with necrotizing enterocolitis

Lead Investigator: Agostino Pierro
Senior associate scientist and thoracic surgeon at The Hospital for Sick Children

This Convergent Working Group will focus on creating a platform for the future design, development and coordination of regenerative medicine clinical trials in critically ill newborns. Necrotizing enterocolitis (NEC), a serious disease of preterm babies in which the gut tissue becomes damaged and begins to die, will be considered as the use case. NEC often causes death or lifelong health issues. By bringing together stakeholders including neonatologists, pediatric surgeons, scientists, clinical trialists, ICU nurses, parents, clinical trainees, government representatives and industry partners, this collaboration aims to prepare the health system for the introduction of trials in newborns for diseases like NEC.

Convergent Working Groups Research Themes

  • Leveraging new technology platforms (e.g., AI-driven design of experiments, robotic self-driving labs, immune engineering, gene editing, synthetic biology, precision medicine) to accelerate the discovery and early-stage translation of engineered, bespoke living therapeutics.
  • Enabling the development of regenerative medicine technologies and living therapies for faster, better recovery from organ failure, severe infections and/or cancer.
  • Strategies that support maternal and children’s health. For example: (i) exploring the role of biological inheritance (e.g., epigenetics, intergenerational factors) in health and disease and the implications for new approaches to regenerative medicine (e.g., therapeutic modulation of the epigenome); (ii) novel regenerative medicine-based approaches to infertility and/or addressing perinatal complications.
  • Innovative biomanufacturing strategies (e.g., on-chip/microfluidics, biosensors, vector-free manufacturing strategies, automation, machine learning) to drive technological breakthroughs that will enable affordability and accessibility of living therapies.
  • Policy frameworks to support the delivery and wide-spread uptake of regenerative medicine innovations and to ensure a constructive impact on the local bioinnovation ecosystem.
  • Novel business models and/or frameworks for the payment, distribution and delivery of regenerative medicine therapies that take into consideration the nature of these complex products compared to conventional pharmaceuticals.
  • Frameworks for engaging diverse communities (inclusive of Indigenous and other equity-seeking populations) and integrating culturally sensitive, patient perspectives to explore disparities, gaps and needs. Ensure that regenerative medicine research is conducted considering equity and inclusion (e.g., conditions that predominantly affect equity-seeking populations are considered). Enhance ethical access and accountability (e.g., health disparities research seed funds).
  • Needs assessment (e.g., regulatory support, correlative study suites, dedicated beds, nursing and data management) for clinical trials of new living therapies and for their adoption into the health system.
  • Methodologies for evaluating the cost-effectiveness of regenerative medicine therapies (e.g., cost-effectiveness of regenerative medicine therapies; patient preferences). For example: Lifecycle Health Technology Management (HTM) frameworks that can be deployed to guide the process of evidence generation early in the R&D process. Proposed frameworks could support the development of high value technologies by capturing and evaluating data on clinical evidence, patient preferences, social impact and economic value propositions prior to regulatory approval.