Collaboration, expertise, and infrastructure: key ingredients for gene therapy to thrive in Europe
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Collaboration, expertise, and infrastructure: key ingredients for gene therapy to thrive in Europe
by Dirk Vander Mijnsbrugge(Vice President Medical Affairs International Developed Markets for Rare Disease, Pfizer)
Gene therapy has emerged as one of the most significant innovations in the biopharma sector. This technology holds promise for the 30 million people in Europe living with a rare disease – for which there are currently few, or no existing treatment options.
With four new gene therapies expected to receive regulatory decisions in Europe this year, and another two expected in 2023 – now is the time to ensure these potentially life-changing treatments can reach those who need them.
To unlock the promise of this emerging science for rare disease patients, Europe must foster an environment where innovation can flourish at scale. This is an environment which focuses on encouraging public-private collaboration, boosting capacity and expertise, and enhancing infrastructure.
Encouraging collaboration and innovation
Innovation cannot happen in a vacuum. Investment and collaboration across Europe are critical to transform the region into an environment where research and development can thrive, and where policy regulation facilitates timely access to innovative treatments, especially in areas of unmet medical need.
In rare disease, scientific innovation and access go hand in hand. If we want to facilitate timely access to innovative treatments such as gene therapy medicines, there will need to be some EU policy changes, for example in cross border healthcare. Novel and collaborative approaches to assessment and reimbursement are also critical to advance sustainable innovation and futureproof healthcare systems.
Boosting capacity and expertise
Gene therapy manufacturing is complex, requiring specialized technology and high levels of expertise. Common manufacturing challenges include specialized handling requirements, securing scarce raw materials, scaling up production and navigating stringent storage conditions.
As an example of this complexity, the average manufacturing process for our rAAV gene therapy technology – a method of gene therapy which uses a vector modeled after adeno-associated virus (AAV) – is around nine to 10 months from start to finish. What’s more, currently gene therapies must undergo additional tests to ensure they meet quality control requirements – which means batch size can be severely reduced during production.
This means that only a few manufacturers have the capacity to meet demand for gene therapies. Companies that do not have such capabilities must rely on contract manufacturing, and contract development and manufacturing organizations (CMOs, CDMOs). However, there is currently a significant industry backlog, with an average wait time of 16 months for CMOs to start new projects involving viral vectors.
For Europe to realize the full potential of gene therapy, we must focus on building manufacturing infrastructure, capacity and growing the expertise needed to produce these innovations at the quality, speed and scale required.
Enhancing infrastructure
At Pfizer, our rAAV gene therapies are manufactured in specialist facilities in North Carolina, USA. We then work with our expert distribution and packaging network to reach our patients – currently, those involved in our clinical trials around the world.
This is another complex story. Gene therapy medicine is fragile, so it’s stored in a specialized vial at minus 60 to minus 90 degrees Celsius – a process known as ultra-cold storage. This may sound familiar. Indeed, our real-world experience with optimizing cold-chain logistics for our COVID-19 vaccine will inform our strategies for distributing potential gene therapy medicines in Europe and beyond.
Gene therapies aren’t your average prescription, they are a potentially one-time treatment. For this reason, we’re investing heavily in Europe to ensure flawless distribution through our facility in San Sebastian de los Reyes, Madrid – one of three global packaging and distribution sites for our potential gene therapy medicines. Upon approval, the site in Madrid will be responsible for delivery direct to clinics throughout Europe, Latin America, and Canada. This is part of an $800 million global investment into our infrastructure for gene therapy.
Preparing for the future
With a renewed focus on health innovation, investment and excellence, Europe is poised to make the changes needed for gene therapy to reach its full potential. At Pfizer, we will continue to explore multiple areas of gene therapy science and apply our mRNA COVID learning in the field of rare disease. This includes progressing our rAAV technology, and following our recent collaboration with BEAM Therapeutics, we also to hope to advance several gene editing programs. We look forward to the day that all these options may become available to patients across Europe.
To learn more about gene therapy, including Pfizer’s rAAV manufacturing and what it takes to bring a gene therapy from concept to clinic, watch this video or tune in to our podcast series Science Will Win.
[1] European Commission. Rare diseases. Available at: https://health.ec.europa.eu/non-communicable-diseases/steering-group/rare-diseases_en Accessed August 2022. [2] Alliance for Regenerative Medicine. (2022). Regenerative Medicine: Disrupting the status quo. Available at: http://alliancerm.org/wp-content/uploads/2022/03/ARM_AR2021_FINAL-singles.pdf Accessed August 2022.
by Dirk Vander Mijnsbrugge(Vice President Medical Affairs International Developed Markets for Rare Disease, Pfizer)
Gene therapy has emerged as one of the most significant innovations in the biopharma sector. This technology holds promise for the 30 million people in Europe living with a rare disease – for which there are currently few, or no existing treatment options.
With four new gene therapies expected to receive regulatory decisions in Europe this year, and another two expected in 2023 – now is the time to ensure these potentially life-changing treatments can reach those who need them.
To unlock the promise of this emerging science for rare disease patients, Europe must foster an environment where innovation can flourish at scale. This is an environment which focuses on encouraging public-private collaboration, boosting capacity and expertise, and enhancing infrastructure.
Encouraging collaboration and innovation
Innovation cannot happen in a vacuum. Investment and collaboration across Europe are critical to transform the region into an environment where research and development can thrive, and where policy regulation facilitates timely access to innovative treatments, especially in areas of unmet medical need.
In rare disease, scientific innovation and access go hand in hand. If we want to facilitate timely access to innovative treatments such as gene therapy medicines, there will need to be some EU policy changes, for example in cross border healthcare. Novel and collaborative approaches to assessment and reimbursement are also critical to advance sustainable innovation and futureproof healthcare systems.
Boosting capacity and expertise
Gene therapy manufacturing is complex, requiring specialized technology and high levels of expertise. Common manufacturing challenges include specialized handling requirements, securing scarce raw materials, scaling up production and navigating stringent storage conditions.
As an example of this complexity, the average manufacturing process for our rAAV gene therapy technology – a method of gene therapy which uses a vector modeled after adeno-associated virus (AAV) – is around nine to 10 months from start to finish. What’s more, currently gene therapies must undergo additional tests to ensure they meet quality control requirements – which means batch size can be severely reduced during production.
This means that only a few manufacturers have the capacity to meet demand for gene therapies. Companies that do not have such capabilities must rely on contract manufacturing, and contract development and manufacturing organizations (CMOs, CDMOs). However, there is currently a significant industry backlog, with an average wait time of 16 months for CMOs to start new projects involving viral vectors.
For Europe to realize the full potential of gene therapy, we must focus on building manufacturing infrastructure, capacity and growing the expertise needed to produce these innovations at the quality, speed and scale required.
Enhancing infrastructure
At Pfizer, our rAAV gene therapies are manufactured in specialist facilities in North Carolina, USA. We then work with our expert distribution and packaging network to reach our patients – currently, those involved in our clinical trials around the world.
This is another complex story. Gene therapy medicine is fragile, so it’s stored in a specialized vial at minus 60 to minus 90 degrees Celsius – a process known as ultra-cold storage. This may sound familiar. Indeed, our real-world experience with optimizing cold-chain logistics for our COVID-19 vaccine will inform our strategies for distributing potential gene therapy medicines in Europe and beyond.
Gene therapies aren’t your average prescription, they are a potentially one-time treatment. For this reason, we’re investing heavily in Europe to ensure flawless distribution through our facility in San Sebastian de los Reyes, Madrid – one of three global packaging and distribution sites for our potential gene therapy medicines. Upon approval, the site in Madrid will be responsible for delivery direct to clinics throughout Europe, Latin America, and Canada. This is part of an $800 million global investment into our infrastructure for gene therapy.
Preparing for the future
With a renewed focus on health innovation, investment and excellence, Europe is poised to make the changes needed for gene therapy to reach its full potential. At Pfizer, we will continue to explore multiple areas of gene therapy science and apply our mRNA COVID learning in the field of rare disease. This includes progressing our rAAV technology, and following our recent collaboration with BEAM Therapeutics, we also to hope to advance several gene editing programs. We look forward to the day that all these options may become available to patients across Europe.
To learn more about gene therapy, including Pfizer’s rAAV manufacturing and what it takes to bring a gene therapy from concept to clinic, watch this video or tune in to our podcast series Science Will Win.
[1] European Commission. Rare diseases. Available at: https://health.ec.europa.eu/non-communicable-diseases/steering-group/rare-diseases_en Accessed August 2022. [2] Alliance for Regenerative Medicine. (2022). Regenerative Medicine: Disrupting the status quo. Available at: http://alliancerm.org/wp-content/uploads/2022/03/ARM_AR2021_FINAL-singles.pdf Accessed August 2022.
ABOUT THE AUTHOR
Dirk is a graduate from the Medical School of the Catholic University of Leuven (1988) and also holds a degree in Pharmaceutical Medicine and an MBA from theVlerick School for Management in Belgium. During his pharmaceutical career he also attended several executive leadership programs at Insead and Harvard Business schools.
After a few years in clinical practice, he joined the industry in 1993 as a clinical research physician. He joined Pfizer in 1997 as medical advisor and receivedincreasing responsibilities in medical affairs, regulatory and clinical development. He served as Country Medical Director for Pfizer Belgium and also headed the Pfizer Clinical Research Unit at the Erasme University of Brussels where he led a team of research professionals responsible for multiple First in Humans and First in Patients pharmacokinetic and pharmacodynamics studies.
Before assuming his current responsibilities as Vice President Medical Affairs International Developed Markets for Rare Disease, Dirk led the medical affairs teams in the Western European Markets for Specialty Care. Next to his role as IDM medical affairs lead, he’s a regular speaker at international events speaking on the evolving role of medical affairs and on policy issues thatare of importance to patients with rare diseases. He’s also an invited lecturer at the University of Leuven in the Pharmaceutical Medicine Curriculum for students in Biomedical science.
Dirk is a graduate from the Medical School of the Catholic University of Leuven (1988) and also holds a degree in Pharmaceutical Medicine and an MBA from theVlerick School for Management in Belgium. During his pharmaceutical career he also attended several executive leadership programs at Insead and Harvard Business schools.
After a few years in clinical practice, he joined the industry in 1993 as a clinical research physician. He joined Pfizer in 1997 as medical advisor and receivedincreasing responsibilities in medical affairs, regulatory and clinical development. He served as Country Medical Director for Pfizer Belgium and also headed the Pfizer Clinical Research Unit at the Erasme University of Brussels where he led a team of research professionals responsible for multiple First in Humans and First in Patients pharmacokinetic and pharmacodynamics studies.
Before assuming his current responsibilities as Vice President Medical Affairs International Developed Markets for Rare Disease, Dirk led the medical affairs teams in the Western European Markets for Specialty Care. Next to his role as IDM medical affairs lead, he’s a regular speaker at international events speaking on the evolving role of medical affairs and on policy issues thatare of importance to patients with rare diseases. He’s also an invited lecturer at the University of Leuven in the Pharmaceutical Medicine Curriculum for students in Biomedical science.
