The Experts below are selected from a list of 309 Experts worldwide ranked by ideXlab platform

Christian K Schneider - One of the best experts on this subject based on the ideXlab platform.

  • the evolution of nonclinical regulatory science advanced Therapy medicinal products as a paradigm
    Molecular Therapy, 2013
    Co-Authors: Henrik Tang Vestergaard, Christian K Schneider, Lucia D Apote, Carla Herberts
    Abstract:

    Advanced Therapy medicinal products (ATMPs) comprise gene Therapy medicinal products (GTMPs), Somatic Cell Therapy medicinal products (CTMPs), and tissue-engineered products (TEPs). So-called combined ATMPs incorporate one or more medical devices as an integral part of the product. Because of their complexity and innovative nature, ATMPs pose new scientific and regulatory challenges to both developers and regulators.1 Many developers of such products request scientific advice from the Committee for Medicinal Products for Human Use (CHMP) at the European Medicines Agency (EMA). We have repeatedly been asked by stakeholders to analyze the scientific advice provided by the CHMP with regard to the identification of common principles that might be useful to share with product developers.

  • the committee for advanced therapies of the european medicines agency reflection paper on management of clinical risks deriving from insertional mutagenesis
    Human gene therapy. Clinical development, 2013
    Co-Authors: Alessandro Aiuti, Giulio Cossu, Pablo De Felipe, Maria Cristina Galli, Gopalan Narayanan, Matthias Renner, Axel Stahlbom, Christian K Schneider, Caroline Voltzgirolt
    Abstract:

    In the European Union, the Committee for Advanced Therapies of the European Medicines Agency takes the lead in the scientific assessment for marketing authorization applications for advanced Therapy medicinal products, which include gene Therapy medicinal products, Somatic Cell Therapy medicinal products, and tissue-engineered products. The Committee for Advanced Therapies also takes the lead in defining the scientific framework for the quality, nonclinical and clinical development of such products. This reflection paper represents the Committee's current thinking on management of clinical risks deriving from insertional mutagenesis. A multidisciplinary approach to insertional mutagenesis is provided. This reflection paper has been adopted by the committee in its April 2013 meeting.

  • clinical development of advanced Therapy medicinal products in europe evidence that regulators must be proactive
    Molecular Therapy, 2012
    Co-Authors: Romaldas Maciulaitis, Christian K Schneider, Lucia Dapote, Andrew Buchanan, Laura Pioppo
    Abstract:

    Advanced Therapy medicinal products (ATMPs), defined as gene Therapy medicinal products (GTMPs), Somatic Cell Therapy medicinal products, and tissue-engineered products (TEPs),1,2 constitute a major class of innovative therapeutics that are being investigated as treatments for several diseases. Despite the success of many such products in animal studies, few have reached more advanced regulatory milestones in Europe (Figure 1), such as ATMP Certification from the Committee for Advanced Therapies (CAT) of the European Medicines Agency (EMA),3 regulatory scientific advice from the EMA,4 or a marketing authorization application at the EMA.5 Therefore, there is a need to identify the major stakeholders in the development of such products and to investigate why they have been unable to move these products farther down the development pipeline. As members of the CAT and EMA, we analyzed the data in the European Union Drug Regulating Authorities Clinical Trials (EudraCT) database (https://eudract.ema.europa.eu). Analysis of 318 ATMP trials performed between 2004 and 2010 revealed that the main sponsors are academia, charities, and small companies. This may have implications for the further evolution of the regulatory framework for ATMPs given that such stakeholders often have limited financial resources or regulatory expertise, leading to a “translational gap” that must be proactively closed by regulators.

  • Erfahrungsbericht aus dem Ausschuss für neuartige Therapien (CAT)
    Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz, 2011
    Co-Authors: M. Reiss, I.c. Büttel, Christian K Schneider
    Abstract:

    Advanced Therapy medicinal products (ATMP) are highly innovative and complex medicines. They comprise gene Therapy medicinal products, Somatic Cell Therapy medicinal products, and tissue-engineered products (TEP). With the European Regulation on ATMP that came into force in 2008, a consolidated regulatory framework was created, where the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA) plays a central role. This article discusses pitfalls and challenges that the CAT has experienced in its discussions of various procedures. Often ATMPs are developed by small and medium-sized enterprises (SME) which also face nonscientific challenges. The CAT wishes to meet these challenges on a scientific and regulatory level during its 2010-2015 work program.

  • Challenges with advanced Therapy medicinal products and how to meet them
    Nature reviews. Drug discovery, 2010
    Co-Authors: Christian K Schneider, Bernd Jilma, Paula Salmikangas, Bruno Flamion, Lyubina Racheva Todorova, Anna Paphitou, Ivana Haunerova, Toivo Maimets, Jean-hugues Trouvin, Egbert Flory
    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.

Gwendolyn L Kartje - One of the best experts on this subject based on the ideXlab platform.

  • Kinematic analysis of motor recovery with human adult bone marrow-derived Somatic Cell Therapy in a rat model of stroke.
    Neurorehabilitation and neural repair, 2012
    Co-Authors: Robynne G. Braun, Ellen M Andrews, Gwendolyn L Kartje
    Abstract:

    BACKGROUND The extent to which pharmaceutical and behavioral therapies following central nervous system injury may either deter or encourage the development of compensatory movement patterns is a topic of considerable interest in neurorehabilitation. However, functional outcome measures alone are relatively insensitive to compensatory changes in movement patterns per se. OBJECTIVE This study used both functional outcome measures and kinematic analysis of forelimb movements to examine the effects of human adult bone marrow-derived Somatic Cells (hABM-SCs) on motor recovery in a rat model of stroke. METHODS Adult male Long-Evans black-hooded rats (n = 12) were trained in a forelimb reaching task and then underwent surgical middle cerebral artery occlusion, producing a stroke that impaired the trained paw. One week poststroke, animals were randomly assigned to either a hABM-SC injection or control injection group. Reaching behaviors were then compared at baseline and at 10 weeks poststroke. RESULTS Both groups improved their outcome scores during the 10-week recovery period. However, the hABM-SC group recovered significantly more function than controls in terms of the number of pellets retrieved. Furthermore, the control group appeared to improve their functional performance by using compensatory strategies that involved an increased number of trajectory adjustments, whereas the hABM-SC group's kinematics more closely resembled prestroke movement patterns. CONCLUSIONS This study demonstrates that kinematic measures established in stroke research on humans are also sensitive to performance differences prestroke versus poststroke in the rat model, reinforcing the utility of this method to evaluate treatments that may ultimately translate to patient populations.

  • human adult bone marrow derived Somatic Cell Therapy results in functional recovery and axonal plasticity following stroke in the rat
    Experimental Neurology, 2008
    Co-Authors: Ellen M Andrews, Shihyen Tsai, Stuart Johnson, J R Farrer, J P Wagner, G C Kopen, Gwendolyn L Kartje
    Abstract:

    Stroke is the leading cause of adult disability in the United States. To date there is no satisfactory treatment for stroke once neuronal damage has occurred. Human adult bone marrow-derived Somatic Cells (hABM-SC) represent a homogenous population of CD49c/CD90 co-positive, non-hematopoietic Cells that have been shown to secrete therapeutically relevant trophic factors and to support axonal growth in a rodent model of spinal cord injury. Here we demonstrate that treatment with hABM-SC after ischemic stroke in adult rats results in recovery of forelimb function on a skilled motor test, and that this recovery is positively correlated with increased axonal outgrowth of the intact, uninjured corticorubral tract. While the complete mechanism of repair is still unclear, we conclude that enhancement of structural neuroplasticity from uninjured brain areas is one mechanism by which hABM-SC treatment after stroke leads to functional recovery.

Tim Farries - One of the best experts on this subject based on the ideXlab platform.

  • the genetically modified organism medicinal framework in europe united states and japan underlying scientific principles and considerations toward the development of gene Therapy and genetically modified Cell based products
    Human gene therapy. Clinical development, 2019
    Co-Authors: Houria Bachtarzi, Tim Farries
    Abstract:

    In vivo viral gene Therapy and Somatic Cell Therapy products (whether autologous, allogeneic, or xenogeneic) that have been subjected to an ex vivo gene transfer procedure will normally be classifi...

  • The Genetically Modified Organism Medicinal Framework in Europe, United States, and Japan: Underlying Scientific Principles and Considerations Toward the Development of Gene Therapy and Genetically Modified Cell-Based Products.
    Human gene therapy. Clinical development, 2019
    Co-Authors: Houria Bachtarzi, Tim Farries
    Abstract:

    In vivo viral gene Therapy and Somatic Cell Therapy products (whether autologous, allogeneic, or xenogeneic) that have been subjected to an ex vivo gene transfer procedure will normally be classified as genetically modified organisms (GMOs) in Europe, not just the gene transfer vectors used in their construction. These products are, therefore, expected to fulfill certain environmental requirements with regard to the biosafety aspects of their clinical use (which may be subject to review by government departments responsible for environmental affairs). In the European Union (EU), clinical trials using GMOs generally require three levels of review (in addition to local review processes), which are often performed by separate national agencies. In this study, the principles under which certain EU member states control use of the GMOs in clinical trials under the definitions of either "contained use" or "deliberate release" will be discussed and evaluated from a scientific and a regulatory perspective, with comparisons with non-EU expectations as described by the U.S. Food and Drug Administration and the Japanese living modified organisms (LMOs) regulations. For the latter, an understanding of the criteria under which LMOs exemptions apply, notably with respect to the nature of the viral construct used, the manufacturing process, and demonstration that there is no detectable residual replication-competent virus in the final gene-modified Cells, is of paramount importance. Building on the existing European, U.S., and Japanese experience with GMOs/LMOs within the context of experimental gene and Cell therapies, a through reflection on, and harmonization of, the current global GMO framework is needed to avoid unnecessary delays in clinical development and to ensure a smooth and a rapid access by patients to innovative life-saving therapies.

Houria Bachtarzi - One of the best experts on this subject based on the ideXlab platform.

  • the genetically modified organism medicinal framework in europe united states and japan underlying scientific principles and considerations toward the development of gene Therapy and genetically modified Cell based products
    Human gene therapy. Clinical development, 2019
    Co-Authors: Houria Bachtarzi, Tim Farries
    Abstract:

    In vivo viral gene Therapy and Somatic Cell Therapy products (whether autologous, allogeneic, or xenogeneic) that have been subjected to an ex vivo gene transfer procedure will normally be classifi...

  • The Genetically Modified Organism Medicinal Framework in Europe, United States, and Japan: Underlying Scientific Principles and Considerations Toward the Development of Gene Therapy and Genetically Modified Cell-Based Products.
    Human gene therapy. Clinical development, 2019
    Co-Authors: Houria Bachtarzi, Tim Farries
    Abstract:

    In vivo viral gene Therapy and Somatic Cell Therapy products (whether autologous, allogeneic, or xenogeneic) that have been subjected to an ex vivo gene transfer procedure will normally be classified as genetically modified organisms (GMOs) in Europe, not just the gene transfer vectors used in their construction. These products are, therefore, expected to fulfill certain environmental requirements with regard to the biosafety aspects of their clinical use (which may be subject to review by government departments responsible for environmental affairs). In the European Union (EU), clinical trials using GMOs generally require three levels of review (in addition to local review processes), which are often performed by separate national agencies. In this study, the principles under which certain EU member states control use of the GMOs in clinical trials under the definitions of either "contained use" or "deliberate release" will be discussed and evaluated from a scientific and a regulatory perspective, with comparisons with non-EU expectations as described by the U.S. Food and Drug Administration and the Japanese living modified organisms (LMOs) regulations. For the latter, an understanding of the criteria under which LMOs exemptions apply, notably with respect to the nature of the viral construct used, the manufacturing process, and demonstration that there is no detectable residual replication-competent virus in the final gene-modified Cells, is of paramount importance. Building on the existing European, U.S., and Japanese experience with GMOs/LMOs within the context of experimental gene and Cell therapies, a through reflection on, and harmonization of, the current global GMO framework is needed to avoid unnecessary delays in clinical development and to ensure a smooth and a rapid access by patients to innovative life-saving therapies.

Antonio Vallano - One of the best experts on this subject based on the ideXlab platform.

  • Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States.
    Frontiers in pharmacology, 2019
    Co-Authors: Carolina Iglesias-lopez, Antònia Agustí, Mercè Obach, Antonio Vallano
    Abstract:

    Advanced Therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development. For both regions, ATMPs fall under the regulatory framework of biological products, which determines the legal basis for their development. Sub-classifications of advanced therapies are different between regions, while in EU, there are four major groups, i.e., gene Therapy, Somatic Cell Therapy, tissue-engineered therapies, and combined advanced therapies; in US, the sub-classification covers two major groups of products, i.e., gene Therapy and Cellular Therapy. The inclusion criteria that define a gene Therapy are equivalent in both regions, and the exclusion criteria are directly related to the indications of the product. In the EU, there is a clear differentiation between Cell- and tissue-based products regarding their classification as advanced therapies or coverage by other legal frameworks, whereas in US, there is a broader classification about whether or not these products can be categorized as biologic products. Both in EU and in US, in order to classify a Cell- or a tissue-based product as an advanced Therapy, it must be ensured that the processing of the Cells implies a manipulation that alters their biological characteristics, although the term of manipulation in US differentiates between structural and non-structural Cells and tissues. The regulatory terminology used to define ATMPs and their sub-classification reveals some differences between EU and US.