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
217
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.
read more
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.
read more
Chat with Paper
AI Agents for this Paper
Find similar papers on Google Scholar, PubMed and Arxiv
Write a critical review of this paper
Analyze citations of this paper to find unaddressed research gaps
Citations
[Regulatory framework of innovative therapies : From bench to bedside].
TL;DR: For planning as well as operational realization of the initial clinical trial involving ATMPs, specific requirements that need to be addressed are discussed in this paper.
1
Fettes Potential – Stammzellen des Fettgewebes in der regenerativen Medizin
Daniel Engel,Pamela Fischer-Posovszky +1 more
- 01 Feb 2019
TL;DR: Dieser Artikel behandelt die Vor- and die Nachteile sowie das Anwendungsspektrum dieser Stammzellen.
EBMT Registry of Nonhematopoietic Stem Cells and Regenerative Therapy (Cellular and Engineered Tissue Therapies in Europe)
Helen Baldomero,Ivan Martin,Katarina Le Blanc,Jan J. Cornelissen,Jakob Passweg,Dietger Niederwieser +5 more
- 01 Jan 2012
TL;DR: It is shown that donor lymphocyte infusion was successfully introduced in the 1990s to cure patients suffering from disease relapse after HSCT to establish cellular therapy as a new therapeutic option for tissue repair, tissue replacement, and immune modulation.
•Dissertation
Urogenital tissue engineering using new hybrid biomaterials
P.K.J.D. de Jonge
- 01 Jan 2018
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.
776