Points to consider for non-clinical safety evaluation of <i>in vivo</i> gene therapy products
نویسندگان
چکیده
Advances in gene therapy have led to a wide range of products. Hence, it is challenging use standard non-clinical safety tests, which are typically used evaluate chemically synthesized Therefore, case-by-case evaluation based on the unique characteristics each product necessary. In Japan, fundamental considerations for evaluating products outlined “Ensuring Quality and Safety Gene Therapy Products”. Notably, when designing studies, essential consider factors such as efficiency vector transduction, tissue/cell tropism vector, species differences expression level biological activity transgene target tissue/organs. However, also crucial understand limitations animal studies that arise due assess vectors expressed genes thorough literature review their properties. The aims this present approaches Japanese guidelines key appropriate vivo
منابع مشابه
"Points to consider" regarding safety assessment of biotechnology-derived pharmaceuticals in non-clinical studies (English translation).
Regulatory and industrial scientists collaborated to publish a "points to consider" document regarding the safety assessment of biotechnology-derived pharmaceuticals in non-clinical studies in 2002 (Pharmaceutical Non-clinical Investigation Group, 2002). The collaboration team intended to clarify the interpretation of ICH-S6 guideline and furthermore share recent Japanese practices on this matt...
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ژورنال
عنوان ژورنال: Translational and regulatory sciences
سال: 2023
ISSN: ['2434-4974']
DOI: https://doi.org/10.33611/trs.2023-002