Full GMP-Compliant Validation of Bone Marrow-Derived Human CD133 (+) Cells as Advanced Therapy Medicinal Product for Refractory Ischemic Cardiomyopathy.

According to the European Medicine Agency (EMA) regulatory frameworks, Advanced Therapy Medicinal Products (ATMP) represent a new category of drugs in which the active ingredient consists of cells, genes, or tissues. ATMP-CD133 has been widely investigated in controlled clinical trials for cardiovas...

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Publicado en:BioMed Research International Vol. 2015; pp. 1 - 11
Autores principales: Belotti, Daniela, Gaipa, Giuseppe, Bassetti, Beatrice, Cabiati, Benedetta, Spaltro, Gabriella, Biagi, Ettore, Parma, Matteo, Biondi, Andrea, Cavallotti, Laura, Gambini, Elisa, Pompilio, Giulio
Formato: research tables/charts Journal Article
Publicado: Wiley-Blackwell 10/1/2015
Acceso en línea:Ver este registro en EBSCOhost
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      dt: 10/1/2015
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      pub: Wiley-Blackwell
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        10.1155/2015/473159
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        atl: Full GMP-Compliant Validation of Bone Marrow-Derived Human CD133 (+) Cells as Advanced Therapy Medicinal Product for Refractory Ischemic Cardiomyopathy.
      aug:
        au:
          Belotti, Daniela
          Gaipa, Giuseppe
          Bassetti, Beatrice
          Cabiati, Benedetta
          Spaltro, Gabriella
          Biagi, Ettore
          Parma, Matteo
          Biondi, Andrea
          Cavallotti, Laura
          Gambini, Elisa
          Pompilio, Giulio
        affil: Laboratory of Cell Therapy “Stefano Verri”, Azienda Ospedaliera San Gerardo, Via Pergolesi 33, 20900 Monza, Italy
      sug:
        subj:
          Cell Culture Techniques
          Bone Marrow
          Myocardial Diseases Therapy
          Myocardial Ischemia
          Human
          Drug Design
      ab: According to the European Medicine Agency (EMA) regulatory frameworks, Advanced Therapy Medicinal Products (ATMP) represent a new category of drugs in which the active ingredient consists of cells, genes, or tissues. ATMP-CD133 has been widely investigated in controlled clinical trials for cardiovascular diseases, making CD133(+) cells one of the most well characterized cell-derived drugs in this field. To ensure high quality and safety standards for clinical use, the manufacturing process must be accomplished in certified facilities following standard operative procedures (SOPs). In the present work, we report the fully compliant GMP-grade production of ATMP-CD133 which aims to address the treatment of chronic refractory ischemic heart failure. Starting from bone marrow (BM), ATMP-CD133 manufacturing output yielded a median of 6.66 × 10(6) of CD133(+) cells (range 2.85 × 10(6)-30.84 × 10(6)), with a viability ranged between 96,03% and 99,97% (median 99,87%) and a median purity of CD133(+) cells of 90,60% (range 81,40%-96,20%). Based on these results we defined our final release criteria for ATMP-CD133: purity ≥ 70%, viability ≥ 80%, cellularity between 1 and 12 × 10(6) cells, sterile, and endotoxin-free. The abovementioned criteria are currently applied in our Phase I clinical trial (RECARDIO Trial).
      pubtype: Academic Journal
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      ougenre: Article
    language: English
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