About this Research Topic
This Research Topic is Volume II of a series. The previous volume, which has attracted 79 authors can be found here: Clinical Translation and Commercialisation of Advanced Therapy Medicinal Products.
Advanced Therapy Medicinal Products (ATMPs) went through a ‘fear of the unknown’ stage but are now proving themselves by saving lives and leading to their first commercial successes. However, to deliver the full potential of ATMPs, we have to achieve key milestones, such as advancing clinical translation for high unmet medical needs, to further improving scalability and manufacturability, and accelerating access to patients and commercial success.
Over the past decades, several major milestones have been achieved in the field of ATMPs, including the first approved and marketed products (such as the first FDA-approved CAR-T cell therapy in 2017), some of which have completely changed the treatment outlook of historically untreatable and often devastating diseases. What’s more, ATMPs most recently have stepped up to become a significant option for the pharmaceutical and biotech industry’s clinical and preclinical pipelines.
Nonetheless, most of the approved products and late-stage clinical trials still concentrate on some niche indications, such as rare diseases and liquid tumors. In order to fully unleash the potential of ATMPs, numerous efforts are being made across the whole healthcare ecosystem (such as in academic research, clinical translation, regulatory flexibilities & policy incentives, healthcare provider innovations, and much more). With this second volume of the Research Topic, we would like to present some of the most cutting-edge research, clinical translation advancements, and field-shaping opinions.
This Research Topic aims to discuss:
(1) key challenges hindering the successful translation and commercialization of ATMPs
(2) emerging concepts in ATMPs’ discovery, analytics, manufacturing, and administration
(3) regulatory landscape evolutions that enable successful clinical development
(4) current regulatory gaps for alternative approaches of non-living cell products or subunits (exosomes, secretomes, inactivated cells)
(5) learnings and new ideas that could boost clinical efficacy (such as combination therapies)
(6) key technology trends or breakthrough innovations (such as in-vivo generation of adoptive cell therapies or gene editing approaches and exosomes)
Dr. Yves Bayon is a Chief Scientist at Medtronic, Dr. Alain Vertès is affiliated with NxR Biotechnologies GmbH, Dr. Tracy Tong Li Yu is an employee of Merck Healthcare KGaA, Dr. Antoine Heron is Scientist and Strategy Consultant at Merck Life Sciences and Prof. Laurent-Applegate is the Founder of the spin-off, Elanix of the Lausanne University Hospital.
Advanced Therapy Medicinal Products (ATMPs) went through a ‘fear of the unknown’ stage but are now proving themselves by saving lives and leading to their first commercial successes. However, to deliver the full potential of ATMPs, we have to achieve key milestones, such as advancing clinical translation for high unmet medical needs, to further improving scalability and manufacturability, and accelerating access to patients and commercial success.
Over the past decades, several major milestones have been achieved in the field of ATMPs, including the first approved and marketed products (such as the first FDA-approved CAR-T cell therapy in 2017), some of which have completely changed the treatment outlook of historically untreatable and often devastating diseases. What’s more, ATMPs most recently have stepped up to become a significant option for the pharmaceutical and biotech industry’s clinical and preclinical pipelines.
Nonetheless, most of the approved products and late-stage clinical trials still concentrate on some niche indications, such as rare diseases and liquid tumors. In order to fully unleash the potential of ATMPs, numerous efforts are being made across the whole healthcare ecosystem (such as in academic research, clinical translation, regulatory flexibilities & policy incentives, healthcare provider innovations, and much more). With this second volume of the Research Topic, we would like to present some of the most cutting-edge research, clinical translation advancements, and field-shaping opinions.
This Research Topic aims to discuss:
(1) key challenges hindering the successful translation and commercialization of ATMPs
(2) emerging concepts in ATMPs’ discovery, analytics, manufacturing, and administration
(3) regulatory landscape evolutions that enable successful clinical development
(4) current regulatory gaps for alternative approaches of non-living cell products or subunits (exosomes, secretomes, inactivated cells)
(5) learnings and new ideas that could boost clinical efficacy (such as combination therapies)
(6) key technology trends or breakthrough innovations (such as in-vivo generation of adoptive cell therapies or gene editing approaches and exosomes)
Dr. Yves Bayon is a Chief Scientist at Medtronic, Dr. Alain Vertès is affiliated with NxR Biotechnologies GmbH, Dr. Tracy Tong Li Yu is an employee of Merck Healthcare KGaA, Dr. Antoine Heron is Scientist and Strategy Consultant at Merck Life Sciences and Prof. Laurent-Applegate is the Founder of the spin-off, Elanix of the Lausanne University Hospital.
Keywords: advanced therapy medicinal products, regenerative medicine, clinical translation, cell and gene therapy, market access, commercialization of ATMPs, regulatory landscape
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