Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective
| العنوان: | Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective |
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| المؤلفون: | Peter Ulrich, Michael W. Bolt, Antonio R. Fernández de Henestrosa, Claudette L. Fuller, Prathap Kumar S. Mahalingaiah, David R. Compton, Laurence O. Whiteley, Jessica L. Lynch, Brian Lauritzen, Timothy K. MacLachlan, Binu K. Philip |
| المصدر: | Molecular Therapy: Methods & Clinical Development, Vol 19, Iss, Pp 89-98 (2020) Molecular Therapy. Methods & Clinical Development |
| بيانات النشر: | Elsevier BV, 2020. |
| سنة النشر: | 2020 |
| مصطلحات موضوعية: | 0301 basic medicine, medicine.medical_specialty, lcsh:QH426-470, Genetic enhancement, Harmonization, Viral vector, Toxicology studies, 03 medical and health sciences, 0302 clinical medicine, Genetics, Medicine, lcsh:QH573-671, Intensive care medicine, Molecular Biology, Gene, Modalities, lcsh:Cytology, business.industry, Safety pharmacology, Perspective (graphical), lcsh:Genetics, 030104 developmental biology, 030220 oncology & carcinogenesis, Molecular Medicine, Original Article, business |
| الوصف: | Nonclinical development strategies for gene therapies are unique from other modalities. The European Federation of Pharmaceutical Industries and Associates (EFPIA) Gene Therapy Working Group surveyed EFPIA member and nonmember pharmaceutical and biotechnology companies about their current practices for designing and implementing nonclinical toxicology studies to support the development of viral vector-delivered in vivo gene therapies. Compiled responses from 17 companies indicated that these studies had some variability in species selection, study-design elements, biodistribution, immunogenicity or genomic insertion assessments, safety pharmacology, and regulatory interactions. Although there was some consistency in general practice, there were examples of extreme case-by-case differences. The responses and variability are discussed herein. Key development challenges were also identified. Results from this survey emphasize the importance for harmonization of regulatory guidelines for the development of gene-therapy products, while still allowing for case-by-case flexibility in nonclinical toxicology studies. However, the appropriate timing for a harmonized guidance, particularly with a platform that continues to rapidly evolve, remains in question. Graphical Abstract The European Federation of Pharmaceutical Industries and Associates Gene Therapy Working Group conducted a survey on current practices associated with nonclinical toxicity studies conducted to support the development of in vivo viral vector-delivered gene therapies. Responses provide insight on studies conducted, endpoints evaluated, and challenges faced by biopharmaceutical/biotechnology companies. |
| تدمد: | 2329-0501 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e654392514a5dc89f2d528b12d218d5a https://doi.org/10.1016/j.omtm.2020.08.017 |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....e654392514a5dc89f2d528b12d218d5a |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 23290501 |
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