Development of Gene Therapies
portes grátis
Development of Gene Therapies
Strategic, Scientific, Regulatory, and Access Considerations
Sverdlov, Oleksandr; McIntosh, Avery
Taylor & Francis Ltd
05/2024
458
Dura
Inglês
9781032136554
Pré-lançamento - envio 15 a 20 dias após a sua edição
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1. Introduction: The Road to Gene Therapy 2. Driving AAV Drug Design to the Right Place, Right Amount, and Right Time 3. A Practical Guide to the Nonclinical Development of In Vivo Gene Therapies 4. Quantitative Systems Pharmacology Modeling of Adeno-Associated Virus Gene Therapies: Mechanistic Identification of Species-Translation Using Preclinical and Clinical Data 5. Bringing Gene Therapy to Patients: A Clinical Development Perspective Based on Brain and Neuromuscular Diseases 6. The Ethics of Gene Therapy 7. AAV Vector Immunogenicity in Gene Therapy: Mechanisms, Assessment, and Immunomodulation Strategies 8. Prenatal Somatic Cell Gene Therapy 9. Development of Gene Therapies for Ultra-Rare Disease 10. Statistical Innovation for Gene Therapy Development: Clinical Trial Design and Analysis Considerations 11. Biomarkers in Gene Therapy Development for Rare Diseases 12. Manufacturing, Analytical, and Process Comparability Challenges for Recombinant Adeno-Associated Virus (rAAV) Gene Therapy 13. Regulatory Considerations in the Development of Gene Therapy Products 14. Gene Therapy Clinical Safety Considerations: Short- and Long-Term 15. Development of Gene Therapies from an Academic Perspective 16. Commercial Models, Access Hurdles, and Health Economics of Gene Therapies 17. The Zolgensma Journey: A Groundbreaking Therapy for SMA 18. History and Development Story of Luxturna: Scientific and Regulatory Challenges 19. The Future of in vivo rAAV Gene Therapies for Rare Neurological Diseases
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Gene Therapy;in vivo rAAV;Regulatory Considerations;Clinical Development;Pharmacology;AAV Drug Design
1. Introduction: The Road to Gene Therapy 2. Driving AAV Drug Design to the Right Place, Right Amount, and Right Time 3. A Practical Guide to the Nonclinical Development of In Vivo Gene Therapies 4. Quantitative Systems Pharmacology Modeling of Adeno-Associated Virus Gene Therapies: Mechanistic Identification of Species-Translation Using Preclinical and Clinical Data 5. Bringing Gene Therapy to Patients: A Clinical Development Perspective Based on Brain and Neuromuscular Diseases 6. The Ethics of Gene Therapy 7. AAV Vector Immunogenicity in Gene Therapy: Mechanisms, Assessment, and Immunomodulation Strategies 8. Prenatal Somatic Cell Gene Therapy 9. Development of Gene Therapies for Ultra-Rare Disease 10. Statistical Innovation for Gene Therapy Development: Clinical Trial Design and Analysis Considerations 11. Biomarkers in Gene Therapy Development for Rare Diseases 12. Manufacturing, Analytical, and Process Comparability Challenges for Recombinant Adeno-Associated Virus (rAAV) Gene Therapy 13. Regulatory Considerations in the Development of Gene Therapy Products 14. Gene Therapy Clinical Safety Considerations: Short- and Long-Term 15. Development of Gene Therapies from an Academic Perspective 16. Commercial Models, Access Hurdles, and Health Economics of Gene Therapies 17. The Zolgensma Journey: A Groundbreaking Therapy for SMA 18. History and Development Story of Luxturna: Scientific and Regulatory Challenges 19. The Future of in vivo rAAV Gene Therapies for Rare Neurological Diseases
Este título pertence ao(s) assunto(s) indicados(s). Para ver outros títulos clique no assunto desejado.
