基因治疗进展 & 生产支持

im体育APP’s consultative team of biopharmaceutical scientists have extensive experience supporting gene therapy programs for both IND and BLA regulatory pathways.

Not many therapeutic modalities can match the promise of gene therapies. 最近, life-changing results have been produced by gene therapies in late-stage trials, and the number of gene therapies approved by the US 食物 and Drug Administration (FDA) continues to increase. 然而,, while the industry faces mounting pressure to reduce cost-per-dose, 仍然迫切需要更多的指征.

Analytical testing is crucial to optimizing gene therapy development. Biopharmaceutical companies developing gene therapies need to identify a scientific partner that can meet the highest standards of safety and reliability, while meeting aggressive commercialization timelines. The ability of a contract research organization (CRO) and contract development and 制造业 organization (CDMO) to meet and accelerate commercialization timelines in support of one-of-a-kind regulatory paths with robust data collection and documentation is critical. This is particularly significant in an era of one-time functional cures, where competition within rare disease communities is common. 确定最优发展路径, 考虑到战略和承诺, 确保从基因到临床的快速战略, informed by solid and robust data delivered by im体育APP’s experts.

im体育APP’s consultative teams of scientific and regulatory experts understand that diverse projects have unique needs. 我们的灵活性, 结合我们深厚的知识基础, allows us to customize our gene therapy analytical 方法 to suite the specific needs of each project. Our QC-centric mindset informs optimized method development, allowing us to identify and overcome problems early and leapfrog challenges before they arise. Manufacturing the doses required for a therapeutic effect is exceptionally expensive, and efficiencies throughout gene therapy process development can significantly reduce costs and increase the program’s viability. Realize significant cost and time savings as workflows begin to scale, 通过优化和加速流程.

im体育APP works with our partners to determine what assays, 方法, and instruments will best help them reach their goals. We use a wide range of orthogonal analytical 方法 and capabilities to ensure the safety, 身份, 质量, 纯度, 以及基因疗法的力量, 包括一个哺乳动物细胞培养设备, 毛细管电泳, 稳定室自定义设置, 还有色谱法和质谱法, including Quadrupole Time of Flight (QTOF) and QExactive systems. Our comprehensive services support the entire product life cycle, from discovery to gene therapy process development, 制造业, 及以后.

基因治疗分析方法及服务

• 超高效液相色谱法测定空/满衣壳
• Determination of vector genome and total viral particle titer by SDS-PAGE or capillary gel electrophoresis
• 病毒载体纯度的测定
• 效力和转导的方法开发
• 肽图
• Residual polyethylenimine, iodixanol, benzonase, and host cell protein detection

Ensuring safety in gene therapy process development

毫无疑问, one of the biggest challenges to the clinical success of gene therapies remains patient safety. 多于1,000个临床研究正在进行中, 针对一系列疾病, including cancer and monogenic diseases like 𝛽-thalassemia and sickle cell anemia, patient safety must remain at the center of all development activities. 因此, while an efficient in vivo or ex vivo vector-strategy is important for successful genetic transfer, these considerations must come second to patient safety.

A careful analysis of viral vectors and transduction 方法 remains vital. im体育APP understands that accurate viral vector analysis, 例如腺相关病毒(aav), 发展对这一进程至关重要. We also understand that scientific confidence goes a long way towards ensuring gene therapies can hit their mark and cure difficult diseases. 我们融合了深厚的科学专业知识, 管理知识, and industry experience allows us to design custom gene therapy analytical 方法 and CMC strategies specific to the needs of each project while meeting the highest standards of safety and reliability.

im体育APP优势

im体育APP提供全面, phase appropriate GMP services to support the development and 制造业 of leading-edge gene therapies, supporting both IND and BLA regulatory submissions. Leverage the deep regulatory expertise of our biologic experts and purpose-built labs to advance your gene therapy 今天.

For more information about im体育APP’s advanced gene therapy services or to request a quote, im体育APP 今天.