Journal Article10.1038/NRD3052
Challenges with advanced therapy medicinal products and how to meet them
Christian Schneider,Paula Salmikangas,Bernd Jilma,Bruno Flamion,Lyubina Racheva Todorova,Anna Paphitou,Ivana Haunerova,Toivo Maimets,Jean-Hugues Trouvin,Egbert Flory,Asterios S. Tsiftsoglou,Balázs Sarkadi,Kolbeinn Gudmundsson,Maura O'Donovan,Giovanni Migliaccio,J amacr,nis Anc amacr,Romaldas Ma ccaron,iulaitis,Jean-Louis Robert,Anthony Samuel,Johannes H. Ovelgönne,Marit Hystad,Andrzej M Fal,Beatriz Silva Lima,Anca Stela Moraru,Peter Tur ccaron,Robert Zorec,Sol Ruiz,Lennart Åkerblom,Gopalan Narayanan,Alastair Kent,Fabrizia Bignami,J. George Dickson,Dietger Niederwieser,María-Angeles Figuerola-Santos,Ilona Reischl,Claire Beuneu,Rosen Georgiev,Maria Vassiliou,Alena Pychova,Mette Clausen,Taina Methuen,Sophie Lucas,Martina Schüssler-Lenz,Vasilios Kokkas,Zsuzsanna Buzás,Niall MacAleenan,Maria Cristina Galli,Aija Lin emacr,Jolanta Gulbinovic,Guy Berchem,Mariusz Fr aogon,czek,Margarida Menezes-Ferreira,Nela Vilceanu,Mikuláš Hrubiško,Petra Marinko,Marcos Timon,Wing Cheng,George Andrew Crosbie,Nick Meade,Michelino Lipucci di Paola,Thierry VandenDriessche,Per Ljungman,Lucia D'Apote,Olga Oliver-Diaz,Isabel Büttel,Patrick Celis +68 more
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TL;DR: The CAT discusses some of the typical issues raised by developers of ATMPs, and highlights the opportunities for such companies and research groups to approach the EMA and the CAT as a regulatory advisor during development.
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Abstract: Advanced therapy medicinal products (ATMPs), which include gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer a major hope for various diseases for which there are limited or no therapeutic options. They have therefore been subject to considerable interest and debate. Following the European regulation on ATMPs, a consolidated regulatory framework for these innovative medicines has recently been established. Central to this framework is the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA), comprising a multidisciplinary scientific expert committee, representing all EU member states and European Free Trade Association countries, as well as patient and medical associations. In this article, the CAT discusses some of the typical issues raised by developers of ATMPs, and highlights the opportunities for such companies and research groups to approach the EMA and the CAT as a regulatory advisor during development.
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Citations
The risk-based approach to ATMP development – Generally accepted by regulators but infrequently used by companies
M. Kooijman,P.J.K. Van Meer,C.C. Gispen-de Wied,Ellen H.M. Moors,Marko P. Hekkert,Huub Schellekens +5 more
TL;DR: The risk-based approach facilitates the science-driven development of ATMPs, and the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) accept non-clinical development programs based on the risk- based approach.
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The Ethical Implications of Tissue Engineering for Regenerative Purposes: A Systematic Review
TL;DR: A systematic review of the ethical implications of the development and application of Tissue Engineering for regenerative purposes, as mentioned in the academic literature reveals that since 2008 a significant body of literature has emerged on how to design clinical trials for Tissue engineering in a responsible manner.
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On the edge of new technologies (advanced therapies, nanomedicines) ☆
TL;DR: Nanotechnology-based and advanced therapy medicinal products are at the cutting edge of innovation in translational drug development, potentially offering new treatment approaches for diseases with limited or no therapeutic alternatives.
Chimeric Antigen Receptor Based Cellular Therapy for Treatment Of T-Cell Malignancies
TL;DR: CAR-based cellular therapy seems feasible and effective for T-cell malignancies, however, the optimal design of CAR-based products is still unknown and long-term follow-up is needed for evaluation of their true potential.
Beyond chimerism analysis: methods for tracking a new generation of cell-based medicines
TL;DR: The toolkit of suitable labelling and detection methodologies for chimerism analysis is described and analyzed with actual examples along with a discussion on challenges ahead and potential solutions.
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References
Effect of gene therapy on visual function in Leber's congenital amaurosis.
James W B Bainbridge,Alexander J. Smith,Susie S Barker,Scott J Robbie,Robert H. Henderson,Kamaljit S. Balaggan,Ananth C. Viswanathan,Graham E. Holder,Andrew Stockman,Nick Tyler,Simon M. Petersen-Jones,Shomi S. Bhattacharya,Adrian J. Thrasher,Fred W. Fitzke,Barrie J Carter,Gary S. Rubin,Anthony T. Moore,Robin R. Ali +17 more
TL;DR: Three young adult patients with early-onset, severe retinal dystrophy were administered subretinal injections of recombinant adeno-associated virus vector 2/2 expressing RPE65 complementary DNA (cDNA) under the control of a human R PE65 promoter.
Progress and opportunities for tissue-engineered skin
TL;DR: In the laboratory, the use of tissue-engineered skin provides insight into the behaviour of skin cells in healthy skin and in diseases such as vitiligo, melanoma, psoriasis and blistering disorders.
1.1K
Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
Alessandro Aiuti,Federica Cattaneo,Stefania Galimberti,Ulrike Benninghoff,Barbara Cassani,Luciano Callegaro,Samantha Scaramuzza,Grazia Andolfi,Massimiliano Mirolo,Immacolata Brigida,Antonella Tabucchi,Filippo Carlucci,Martha M. Eibl,Memet Aker,Shimon Slavin,Hamoud Al-Mousa,Abdulaziz Al Ghonaium,Alina Ferster,Andrea Duppenthaler,Luigi D. Notarangelo,Uwe Wintergerst,Rebecca H. Buckley,Marco Bregni,Sarah Marktel,Maria Grazia Valsecchi,Pier Luca Rossi,Fabio Ciceri,Roberto Miniero,Claudio Bordignon,Claudio Bordignon,Maria Grazia Roncarolo +30 more
TL;DR: Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency and effective protection against infections and improvement in physical development made a normal lifestyle possible.
Donor-Derived Brain Tumor Following Neural Stem Cell Transplantation in an Ataxia Telangiectasia Patient
Ninette Amariglio,Abraham Hirshberg,Bernd W. Scheithauer,Yoram Cohen,Ron Loewenthal,Luba Trakhtenbrot,Nurit Paz,Maya Koren-Michowitz,Dalia Waldman,Leonor Leider-Trejo,Amos Toren,Shlomi Constantini,Gideon Rechavi +12 more
TL;DR: The findings here suggest that neuronal stem/progenitor cells may be involved in gliomagenesis and provide the first example of a donor-derived brain tumor complicating neural stem cell therapy.
Gene therapy: trials and tribulations
Nikunj V. Somia,Inder M. Verma +1 more
TL;DR: A key problem in gene therapy is the lack of a vector system that fulfils all the requirements for safety and efficacy, and viral vectors are the most promising vectors at this time.
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