REGULATORY PERSPECTIVE FOR GENE THERAPY PRODUCTS IN THE USA, EU AND JAPAN AND FUTURE ASPECTS

Gene Therapy Products (GTPs) hold immense promise in revolutionizing medical treatments by altering genetic expressions to address various diseases. This study gives a summary of gene therapy products and their prospective uses, their historical development and several treatment options, as an in-depth exploration of the regulatory considerations for GTPs in the United States of America (USA), European Union (EU), and Japan, along with insights into future aspects of this field. A comprehensive discussion follows detailing the regulatory landscape and approval pathways in the USA, EU, and Japan. Programs that are exclusive to each area, such as PRIME (Priority Medicines) in the EU, USA's RMAT (Regenerative Medicines Advanced Therapy) designation and the Sakigake system in Japan are examined. Milestone meetings, approval requirements, and specific regulatory guidelines for GTPs in each region are also thoroughly covered. A list of approved GTPs and a glimpse into the future of the field. Anticipated trends include increasing investments, challenges related to production costs, expansion of manufacturing capabilities, and regulatory updates. The various regulatory strategies in each area and their efforts to balance patient access and safety will have a big impact on GTPs marketed in the future. Japan is well-positioned to maintain it’s as a global leader in regenerative medicine and cell treatments because of its favourable regulatory environment and government backing.

qPCR在重组腺相关病毒产品大片段宿主细胞残留DNA检测中的应用 / 中国生物制品学杂志

@#Objective To investigate the feasibility of using quantitative PCR(qPCR)technology to detect large fragments of host cell residual DNA(HCD)in recombinant adeno-associated virus(rAAV)gene therapy products.Methods Four different serotypes of rAAV were extracted for the nucleic acids,two fragment sequences of 244 bp and 562 bp within the long terminal repeat sequence(LTR)in the genome of host cells HEK293 were specifically quantified by qPCR,and the proportion of HCD in the total nucleic acids was calculated.Results Large fragments of HCD in qPCR quantifiable range were detected in four different serotype rAAV products,with the abundance ranging from 0. 3% to 5. 4%. As the length of the detected fragment increased,the abundance of HCD fragments showed a decreasing trend.Conclusion qPCR technology can be used to determine the presence of large fragments of HCD in rAAV products.