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This blog post is just one of many in Signal’s seventh annual blog carnival discussing what hurdles need to be overcome for cell and gene therapies in clinical trials to reach the marketplace and whether the system is prepared. Please click here to read what other bloggers think about this topic.

Those of us who have followed developments in the cell and gene therapy industry in Canada and beyond have no doubt noticed the pace of innovation picking up over the past few years, spurred in part by the pandemic and a focus on biomanufacturing. Gene therapy in particular seems to be having a moment.

The first gene therapy, Luxturna, was approved in 2017 for the treatment of congenital retinal dystrophy. Now there are at least five approved gene therapies, mainly focused on rare diseases, globally. The most recent, CSL Behring’s Hemgenix, for hemophilia B, is now the world’s most expensive drug at $3.5M USD per treatment and was approved by the FDA in Nov. 2022, which I’m sure will catch industry’s attention.

There are more than 160 industry sponsored gene therapy clinical trials currently recruiting in North America. The regulatory road map has been paved and now the flood gates have been opened. It’s clear the approvals will be accelerating. How do we deliver these therapies efficiently to patients beyond academic hospitals? How do we design and develop the next generation of gene therapies in an equitable way? How do we keep Toronto at the forefront of gene therapy innovation to continue to be an engine of innovation?

To begin to answer questions like these, Medicine by Design has funded a Convergent Working Group project, called TOGETHER (The Ontario GEne ThERapy Network), which is focused on addressing the critical unmet needs to identify and translate therapies for rare and ultra-rare diseases. The overarching goal is to make Toronto a world leading centre for rare disease gene therapy and to increase the speed at which we deliver gene therapies to patients through diagnosis, development, and delivery.

Led by Jim Dowling, a clinician-scientist at The Hospital for Sick Children (SickKids), the effort began with a workshop that featured a highly interdisciplinary group of contributors including patients and families, clinical pediatricians, molecular biologists, geneticists, chemists, health policy experts, and ethicists across Toronto.

The focus of the workshop was to identify the gaps in expertise, representation, and knowledge that must be addressed to reach their goal. Questions regarding patient identification, stakeholder engagement, preclinical, pre-trial, and clinical experiments, clinical implementation, accessibility, sustainability and funding were addressed, resulting in several Calls to Action.

What hurdles need to be overcome for gene therapies to reach the marketplace?

The group acknowledged a challenge with the development of novel gene therapies for rare disease patients in Ontario, including crucial needs for preclinical studies to create and translate gene therapies and develop robust clinical guidelines for the safe and effective implementation of them.

To answer these needs, a gene therapy innovation hub will be created to improve and speed the manufacturing and development of therapeutics. This hub will produce viral vectors and perform the insertion of the gene of interest for preclinical studies. A gene therapy clinical working group has also been formed and will establish and disseminate standard operating practices (SOPs) to ensure best practices and services of the hub that can be accessed by the community.

Will regulatory approval be a bottleneck?

The group also expressed the need to outline and highlight current gaps in gene therapy development and to identify specific hurdles related to regulatory challenges.

Through the experience of the TOGETHER team, many issues with the Health Canada approval, reimbursement processes, and implementation have been identified. The TOGETHER network will perform a landscaping review and evaluation of the current translational pathway of gene therapy in Canada and, using input from network stakeholders, will produce a review article summarizing observations to be submitted for publication.

Can the system handle all the new therapies getting approved?

There is a need to democratize the gene therapy commercialization ecosystem, according to the group’s observations, to foster knowledge sharing and professional development among healthcare providers and strengthen the TOGETHER network across Ontario.

Expanding TOGETHER to additional stakeholders across the province will ensure equitable access and scalability and build a common rare disease community out of the current fragmented state. The large number of rare disease and gene therapy focused seminar series will all be combined into a common platform. Also, TOGETHER will re-convene one year from the initial meeting to discuss the progress made thus far as well as establish the long-term plan.

Addressing clinical challenges will also be part of preparing the system for new therapy implementation including identifying the needs of gene therapy in the clinic and developing strategies for gene therapy-related adverse events.

Convergence as a tool to address potential challenges

The seven Convergent Working Groups projects funded by Medicine by Design can be looked at as an effective model for addressing the challenges of developing and commercializing regenerative medicine therapies, including the hurdles and bottlenecks in gene therapy development. By bringing a wide range of experts together at the beginning of the process, the challenges can be identified and addressed before any work even begins.

Andrew Haller, PhD, is the Entrepreneur-in-Residence for Medicine by Design. He is passionate about the development, commercialization and delivery of the next generation of living therapies. Andrew got his PhD in Molecular Pharmacology, did a post-doctoral fellowship at UHN, and gained experience through several roles in Toronto’s innovation ecosystem. He’s developed a wide range of experience in the commercialization of medicine in the pre-clinical and clinical trials space, business development, and venture capital. He wears a lot of hats in the health innovation ecosystem and uses the synergistic learnings across each to implement and execute strategic business plans for early-stage technologies.