The Human Gene Therapy Applications Laboratory (HGTAL)-National Core will continue to serve as a national resource for all NHLBI supported investigators pursuing clinical gene therapy applications. Since its initiation, the HGTAL has produced and certified 7 retroviral producer cell Master Cell Banks (MCBs) and Clinical retroviral supernatant derived from 4 of these MCBs have been used in Phase 1 and 2 clinical trials. For Adenoviral vectors, the HGTAL-National Core has produced and certified a MCB and a Master Working Cell Bank (MWCB) of HEK-293 cells, produced two different Adenoviral vector MVBs and produced an Ad.iNOS Final Product (FP). Also, the core has produced and certified a MCB of HSV trans-complementation cells, two different HSV Master Viral Banks (MVBs) and is currently producing HSV vector FP. Moreover, the methods for clinical production and purification of clinical grade AAV serotypes 6 and subsequently 8 are being developed by the HGTAL that will allow production of AAV FP within the first year. The facility will be able to produce up to three of each type of vector per year. Thus the primary function of the HGTAL-National Core will remain the production of clinical grade recombinant Viral Vector Final Products (FP) for all eligible NHLBI supported investigators pursuing gene therapy clinical trials. The HGTAL-National Core is able to produce for NHLBI supported investigators, as needed, clinical grade Retoviral, Herpes Simplex and Adenoviral Vectors and within the next year will be able to produce adeno-associated virus (AAV) serotypes 6 and 8. It anticipated that the Core will be able to produce up to three of each type of vector per year. The HGTAL also is able to manufacture and certify all necessary clinical intermediate products such as Master Cell Banks (MCB), Master Viral Banks, and plasmid stocks needed to produce clinical grade viral vector FP and has developed scalable methods of vector production that can be used to produce these materials under compliance with the cGMP directives of the 21Code of Federal Regulations (21CFR). The Core also has developed validated lot release assays, as required by the FDA, for clinical grade recombinant Retroviral, Adenoviral, and Herpes viral Final Products and is developing lot release assays for Adeno-Associated Virus serotypes 6 and subsequently 8 final products. Finally, the Core is able to assist investigators in preparing regulatory submissions of their studies to include IBC, RAC, FDA, and their IRB.
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