%PDF-1.3 1 0 obj << /Type /Catalog /Outlines 2 0 R /Pages 3 0 R >> endobj 2 0 obj << /Type /Outlines /Count 0 >> endobj 3 0 obj << /Type /Pages /Kids [6 0 R 22 0 R 24 0 R 26 0 R 28 0 R 30 0 R ] /Count 6 /Resources << /ProcSet 4 0 R /Font << /F1 8 0 R /F2 9 0 R /F3 10 0 R /F4 11 0 R >> /XObject << /I1 12 0 R /I2 13 0 R /I3 20 0 R /I4 21 0 R >> >> /MediaBox [0.000 0.000 595.280 841.890] >> endobj 4 0 obj [/PDF /Text /ImageC ] endobj 5 0 obj << /Creator (DOMPDF) /CreationDate (D:20220609101127-04'00') /ModDate (D:20220609101127-04'00') /Title (Gene Therapy: Development, Design of Studies, and Approval Process - BLOG) >> endobj 6 0 obj << /Type /Page /Parent 3 0 R /Annots [ 14 0 R 16 0 R 18 0 R ] /Contents 7 0 R >> endobj 7 0 obj << /Length 5797 >> stream 1.000 1.000 1.000 rg 34.016 34.016 527.249 773.859 re f q 441.000 0 0 97.500 45.016 710.374 cm /I2 Do Q 0.000 0.000 0.000 rg BT 45.016 699.385 Td /F1 9.0 Tf [(Published on )] TJ ET BT 99.547 699.385 Td /F2 9.0 Tf [(IVT Network)] TJ ET BT 149.056 699.385 Td /F1 9.0 Tf [( \()] TJ ET BT 154.555 699.385 Td /F1 9.0 Tf [(http://www.ivtnetwork.com)] TJ ET 0.000 0.000 0.000 RG 0.18 w 0 J [ ] 0 d 154.555 698.234 m 259.081 698.234 l S BT 259.081 699.385 Td /F1 9.0 Tf [(\))] TJ ET 0.620 0.620 0.620 rg 45.016 686.888 506.159 0.750 re f 0.000 0.000 0.000 rg BT 45.016 662.142 Td /F3 15.0 Tf [(Gene Therapy: Development, Design of Studies, and Approval Process - BLOG)] TJ ET q 45.016 610.283 506.159 33.750 re W n 0.800 0.800 0.800 rg 45.016 643.658 13.005 0.750 re f 0.000 0.000 0.000 rg BT 45.016 623.669 Td /F1 9.0 Tf [(By)] TJ ET 0.800 0.800 0.800 rg 58.021 643.658 70.533 0.750 re f 0.000 0.000 0.000 rg BT 58.021 625.544 Td /F4 9.0 Tf [(Kaiser Jay Aziz)] TJ ET 0.800 0.800 0.800 rg 128.554 643.658 102.051 0.750 re f 0.000 0.000 0.000 rg BT 128.554 625.544 Td /F4 9.0 Tf [(Jul 6, 2021 7:00 am EDT)] TJ ET Q BT 45.016 483.794 Td /F2 9.0 Tf [(This article was originally published in Journal of Biotechnology & Bioinformatics Research, July 10, 2021, Volume 3\(3\): 4-4. It )] TJ ET BT 45.016 470.058 Td /F2 9.0 Tf [(is being reprinted here with the permission of the author. IVT Network is republishing this work to assist in bringing the latest )] TJ ET BT 45.016 456.322 Td /F2 9.0 Tf [(research and developments to our audience, as well as to support the great works of researchers like Kaiser Aziz.)] TJ ET BT 45.016 431.717 Td /F4 10.5 Tf [(ABSTRACT)] TJ ET BT 45.016 408.514 Td 0.808 Tw /F1 9.0 Tf [(Genome editing can be applied to various areas of medical diagnosis and treatments. Gene therapy pre-market applications )] TJ ET BT 45.016 394.778 Td 0.497 Tw /F1 9.0 Tf [(comprise of systematically assessing a products design controls, manufacturing process controls, and proposed protocols for )] TJ ET BT 45.016 381.042 Td 0.355 Tw /F1 9.0 Tf [(post-marketing surveillance. Quality risk management principles have been described in various FDA regulatory guidances for )] TJ ET BT 45.016 367.305 Td 0.791 Tw /F1 9.0 Tf [(several aspects of good manufacturing practices \(GMPs\) such as several stages of process validation and verification in the )] TJ ET BT 45.016 353.569 Td 1.101 Tw /F1 9.0 Tf [(genome products life cycle including critical quality attributes \(CQAs\) and monitoring critical process parameters \(CPPs\). A )] TJ ET BT 45.016 339.833 Td 0.647 Tw /F1 9.0 Tf [(CPP is defined as a process parameter whose variability has an impact on a CQA of genome product and, therefore, should )] TJ ET BT 45.016 326.097 Td 1.023 Tw /F1 9.0 Tf [(be monitored or controlled to ensure that the manufacturing process produces an end product of the desired quality. FDAs )] TJ ET BT 45.016 312.360 Td 1.498 Tw /F1 9.0 Tf [(mission is to facilitate the premarket review and evaluation of new genomic products for clinical use. The FDA guidances )] TJ ET BT 45.016 298.624 Td 0.329 Tw /F1 9.0 Tf [(emphasize a quality management approach to the design of studies by providing oversight and objective review based on risk-)] TJ ET BT 45.016 284.888 Td 0.434 Tw /F1 9.0 Tf [(benefit analysis of new genomic products. FDA reviews, evaluates, verifies and validates the implementation of the regulatory )] TJ ET BT 45.016 271.152 Td 0.871 Tw /F1 9.0 Tf [(design-control requirements which are applied to the control genomic products quality throughout the total product life cycle )] TJ ET BT 45.016 257.415 Td 0.000 Tw /F1 9.0 Tf [(\(TPLC\) [1-5].)] TJ ET BT 45.016 232.811 Td /F4 10.5 Tf [(INTRODUCTION)] TJ ET BT 45.016 209.608 Td 1.832 Tw /F1 9.0 Tf [(One of the primary functions of a genomic firms research project team is to coordinate the various literature references )] TJ ET BT 45.016 195.872 Td 3.568 Tw /F1 9.0 Tf [(necessary for the successful development of the genomic product under consideration. This coordination is usually )] TJ ET BT 45.016 182.135 Td 2.926 Tw /F1 9.0 Tf [(accomplished by preparing a detailed genomic drug development plan and process controls. This requires analyzing, )] TJ ET BT 45.016 168.399 Td 0.319 Tw /F1 9.0 Tf [(evaluating and constructing study design as they relate to the proposed genomic products chemical entity for disease therapy )] TJ ET BT 45.016 154.663 Td 1.560 Tw /F1 9.0 Tf [(\(i.e., Cardiovascular, cancer, CNS indications, diabetes, etc.\). This includes types and duration of therapies \(i.e., acute or )] TJ ET BT 45.016 140.927 Td 3.164 Tw /F1 9.0 Tf [(chronic situations with one or a few doses adequate for treatment modes. Its also important to consider routes of )] TJ ET BT 45.016 127.190 Td 0.305 Tw /F1 9.0 Tf [(administration \(i.e., intravenous, or infusion or non-intravenous such as oral, pulmonary, subcutaneous, intramuscular, dermal, )] TJ ET BT 45.016 113.454 Td 0.246 Tw /F1 9.0 Tf [(etc.\). The timelines for the various studies and their integration into a formal therapeutic drug development plan are compound )] TJ ET BT 45.016 99.718 Td 1.329 Tw /F1 9.0 Tf [(specific and dependent on the availability of resources within the various departments of the sponsor firm and approval of )] TJ ET BT 45.016 85.982 Td 1.525 Tw /F1 9.0 Tf [(sponsors management. At the same time, the designation of pertinent milestone events and the critical path are genomic )] TJ ET BT 45.016 72.245 Td 0.997 Tw /F1 9.0 Tf [(compound specific and firm-specific. 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