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Table 1 Summary of the product development package for a MSC-based medicinal for the treatment of gonarthrosis.

From: Development of an advanced cell therapy product indicated for the treatment of gonarthrosis

GLP GMP GCP Study description Experimental system Dose; Route of administration
    Assessment of long-term effects of autologous MSC treatment in an ovine chronic model of OA [13] Ovis aries
(Ripollesa breed, ♀); surgically-induced OA
11x106 oMSC; intraarticular
X X   Biodistribution of hMSC in an immunodeficient mouse model Mus musculus
(NRG, ♀ and ♂)
4x105 hMSC; tail-vein injection
  X   Single-dose toxicology study of intraarticularly administered hMSC in athymic rats Rattus norvegicus
(NIH nude, ♂)
Up to 6x105 hMSC; intraarticular
X X   Dose-response study after intraarticular injection of hMSC in a rat model of OA Rattus norvegicus
(NIH nude, ♂);
MIA-induced OA
Up to 8x104 hMSC; intraarticular
X X   Analysis of protooncogen expression levels, hTERT activity, senescense, G-banding karyotype, and CGH arrays on clinical grade hMSC In vitro assays N/A
  X X Adult Stem Cell Therapy for Repairing Articular Cartilage in Gonarthrosis (EUDRA-CT: 2009-016449-24; ClinicalTrials.gov Identifier: NCT01227694) Human(♀ and ♂); grade II/III knee OA 40.9x106 hMSC; intraarticular
  1. GxP compliance of non-clinical and clinical studies, and for the production of batches of MSC are also indicated. GCP=Good Clinical Practice; GLP=Good Laboratory Practice; GMP=Good Manufacturing practice; MIA=monoiodoacetate; MSC=Mesenchymal Stromal Cell; OA=osteoarthritis; N/A=Not Applicable; NIH=National Institutes of Health; NRG=NOD.Cg-Rag1tm1Mom Il2rgtm1Wjl/SzJ.