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Recent Progress in European Advanced Therapy Medicinal Products and Beyond
Tracy T L Yu1,2,3, Pravesh Gupta3, Vincent Ronfard4
1Centre for Craniofacial and Regenerative Biology, King's College London, London, United Kingdom.
Frontiers in Bioengineering and Biotechnology
|October 10, 2018
Summary
Cell and gene therapies are revolutionizing regenerative medicine, offering new hope for genetic diseases and malignancies. Recent advancements, particularly in immuno-oncology, show significant clinical and commercial progress in the European Union.
Area of Science:
- Regenerative Medicine
- Cell and Gene Therapy
- Immuno-oncology
Background:
- Cell- and gene-based therapies are foundational to regenerative medicine, with potential to treat genetic diseases, neurodegenerative disorders, and malignancies.
- Significant progress has been made in the last decade, leading to marketing authorizations and commercial products like Glybera®, Kymriah®, YESCARTA®, Holoclar®, and Luxturna™.
Purpose of the Study:
- To analyze developments in the regenerative medicine landscape, focusing on the European Union.
- To examine clinical and commercial successes and failures in regenerative medicine, including immuno-oncology.
- To discuss key issues such as unmet clinical need, challenges, economic evaluation, and policy improvements.
Main Methods:
- Analysis of developments in regenerative medicine and immuno-oncology within the European Union.
- Review of clinical and commercial outcomes, including successes and failures.
- Consideration of factors driving advancements in immuno-oncology.
- Reflection on industry insights regarding R&D, pricing, reimbursement, and technology adoption.
Main Results:
- Tangible outcomes achieved through unified efforts, resulting in approved commercial therapies.
- Identification of factors contributing to advancements in immuno-oncology.
- Emphasis on critical issues including unmet clinical needs, economic evaluations, and policy needs.
- Insights into industry perspectives on advanced therapy medicinal products (ATMPs) integration.
Conclusions:
- Cell and gene therapies represent a significant pillar of regenerative medicine with transformative potential.
- The European Union has witnessed notable clinical and commercial successes, alongside challenges, in regenerative medicine and immuno-oncology.
- Addressing key issues from clinical need to policy is crucial for the continued advancement and integration of innovative therapies.
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