US Pharmacopeia (USP) · Diane McCarthy, Vice President, Global Biologics · Analytical Methods

Emerging analytical platforms use different measurement principles, making direct comparison difficult and leading to lack of concordance. Bridging between methods requires comparative studies and a well-characterized reference material or control; outlines considerations for the design and analysis of such studies.

新兴分析平台采用不同的测量原理，导致方法间难以直接比较、结果缺乏一致性。方法桥接需要开展比对研究并使用充分表征的对照品或对照，介绍此类研究的设计与分析考量。

US Pharmacopeia (USP) · Ben Clarke, Senior Scientist · Gene Therapy CMC & Analytics

USP is developing <1067> Best Practices for the Manufacture and Quality Control of Recombinant AAV Gene Therapy Products, plus standards, reference materials and tools covering AAV impurities, titer and capsid content; attendees learn to integrate these into a robust analytical QC strategy.

USP 正在制定 <1067>《重组 AAV 基因治疗产品生产与质量控制最佳实践》，并配套涵盖 AAV 杂质、滴度与衣壳含量的标准、对照品与工具；与会者将学习如何将其整合进稳健的分析质控策略。

US Pharmacopeia (USP) · Anthony Blaszczyk, Senior Scientist, Global Biologics · Gene Therapy CMC & Analytics

Lentiviral vectors underpin ex vivo (e.g., CAR T) and increasingly in vivo therapies; their size and complexity make characterization challenging. USP has developed LVV standards for vector copy number, residual HEK293 DNA quantification and physicochemical characterization to strengthen confidence in LVV quality.

慢病毒载体支撑离体（如 CAR T）及日益增多的体内疗法；其尺寸与复杂性使表征颇具挑战。USP 已制定慢病毒载体标准，涵盖载体拷贝数、残留 HEK293 DNA 定量与理化表征，以增强对慢病毒载体质量的信心。

USP (United States Pharmacopeia) · Huiping Tu, Senior Principal Scientist, Microbiology / Global Biologics · Cell Therapy Analytics

Traditional mycoplasma testing is constrained by long turnaround, volume requirements and specialized labs. USP <77> introduces nucleic acid amplification tests (NATs) for rapid qualitative mycoplasma detection with a risk-based implementation framework, outlining assay controls and validation parameters.

传统支原体检测受限于周转时间长、样品量需求高、依赖专业实验室。USP <77> 引入核酸扩增检测（NAT）以实现快速定性支原体检测，并提供基于风险的实施框架，阐述检测对照与验证参数。

US Pharmacopeia (USP) · Diane McCarthy, Vice President, Global Biologics · Oligonucleotide & Peptide CMC

Outlines quality frameworks for therapeutic oligonucleotides and peptides, emphasizing how public standards strengthen CMC development. Covers impurity control, starting-material characterization and analytical consistency across the lifecycle, with updates on new and upcoming USP documentary and physical reference standards including phosphoramidites.

阐述治疗性寡核苷酸与多肽的质量框架，强调公开标准如何强化 CMC 开发。涵盖杂质控制、起始物料表征及全生命周期分析一致性，并更新包括亚磷酰胺在内的 USP 文字与实物对照标准的最新进展。

NIST (National Institute of Standards and Technology) · Melis Kant, Biochemist, Biomaterials Group · Cell Therapy Analytics

Cell characterization and testing are critical for bioprocess monitoring. Standards are urgently needed to establish common practices, interoperability and translation. Describes the current standards infrastructure for cell characterization and recent trends in process analytical technologies (PAT) for cell therapies, including label-free approaches.

细胞表征与检测对生物工艺监控至关重要。亟需标准来确立统一规范、互操作性与成果转化。介绍当前细胞表征的标准基础设施，以及细胞治疗过程分析技术（PAT）的最新趋势，包括无标记方法。

US Pharmacopeia (USP) · Sarita Kattel, Principal Scientist · RNA & LNP Production and Formulation

The rapid growth of mRNA therapeutics demands consistent, science-based quality standards for raw-material, manufacturing and release testing. USP is developing an integrated framework of documentary and physical standards to support reliability, comparability and regulatory confidence across the mRNA-LNP lifecycle.

mRNA 疗法的快速发展要求建立一致、基于科学的质量标准，用于原材料、生产与放行检测。USP 正在构建文字与实物标准的整合框架，以支撑 mRNA-LNP 全生命周期的可靠性、可比性与监管信心。

Novartis · Nripen Singh, Executive Director and Site Head, Process Development, TRD CGT · Gene Therapy CMC & Analytics

In vivo CAR T could eliminate patient-specific ex vivo manufacturing and enable scalable, off-the-shelf therapies; success will be largely determined by CMC readiness. Evaluates in vivo CAR T through a CMC lens, focusing on consistency, scalability, formulation stability, analytical control and comparability.

体内 CAR T 有望免除针对患者个体的离体生产，实现可规模化的现货型疗法；其成败在很大程度上取决于 CMC 就绪度。从 CMC 视角评估体内 CAR T，聚焦一致性、可规模化、制剂稳定性、分析控制与可比性。

Bristol Myers Squibb · Stephan O. Krause, Executive Director Analytical Quality, BMS Cell Therapies · Cell Therapy Analytics

Illustrates an enhanced analytical strategy execution roadmap focused on method-performance optimization and automation across product development and commercialization. Reviews two critical guiding principles and an execution roadmap, then deep-dives into an integrated analytical development program and state-of-control process with case studies.

展示一套增强型分析策略实施路线图，聚焦贯穿产品开发与商业化的方法性能优化与自动化。回顾两项关键指导原则与实施路线图，并结合案例深入剖析整合的分析开发项目与受控状态工艺。

Immatics Biotechnologies GmbH · Divya Ravirala, Director, CMC Analytical Development · Cell Therapy Analytics

Potency is a critical quality attribute essential for product release and licensure. Establishing scientifically sound, mechanism-based, phase-appropriate potency strategies remains key through development and commercialization; presents a structured, risk-based approach to potency-assay development and validation to ensure functional consistency.

效价是产品放行与获批所必需的关键质量属性。在开发与商业化全程建立科学合理、基于机制、分阶段适用的效价策略至关重要；介绍一种结构化、基于风险的效价检测开发与验证方法，以确保功能一致性。

Sanofi · Jill Bradley-Graham, Principal Scientist, GMU BioAnalytics Characterization · Gene Therapy CMC & Analytics

AAV vectors have unique stability challenges and poorly understood structure-function relationships. Comprehensive stress testing (heat, pH, oxidation, light) using potency assays, mass photometry and peptide mapping showed sensitivity to temperature, light and pH extremes — informing formulation optimization and CQA identification.

AAV 载体存在独特的稳定性挑战，其结构-功能关系尚不清晰。采用效价检测、质量光度法与肽图谱开展全面强制降解试验（热、pH、氧化、光照），显示其对温度、光照与极端 pH 敏感——为制剂优化与 CQA 识别提供依据。

Paul Ehrlich Institute · Erika Friedl, Senior Quality Expert, Haematology, Cell and Gene Therapy · Formulation & Delivery of Biologics

Process-related impurities are critical quality attributes that can severely impact product quality and patient safety. Problematic host cell proteins (HCPs) must be identified, characterized and removed; regulatory guidance should be consulted to meet expectations and avoid market-access delays. An effective HCP control strategy is essential.

工艺相关杂质是可严重影响产品质量与患者安全的关键质量属性。须识别、表征并去除有问题的宿主细胞蛋白（HCP）；应参考监管指南以满足预期、避免市场准入延误。建立有效的 HCP 控制策略至关重要。

ARPA-H · John E. Schiel, Program Manager, Scalable Solutions · RNA & LNP Production and Formulation

Manufacturing personalized and individualized (P/I) genetic medicines is constrained by centralized, complex, costly production. The GIVE (Genetic Medicines and Individualized Manufacturing for Everyone) program will enable a distributed, multi-site, multi-product RNA biomanufacturing network integrating automated manufacturing with rapid QC.

个体化（P/I）基因药物的生产受制于集中化、复杂且高成本的生产模式。GIVE（人人可及的基因药物与个体化制造）项目将构建分布式、多站点、多产品的 RNA 生物制造网络，整合自动化生产与快速质控。

Genezen / ARPA-H / NIIMBL · Susan D'Costa (moderator); John E. Schiel (ARPA-H); Chris Williams (NIIMBL, Co-Lead Viral Vector) · Gene Therapy CMC & Analytics

Panel on regulatory expectations and emerging guidance for demonstrating comparability, control and product-specific quality in highly personalized runs; ultra-flexible GMP-compliant platforms that pivot batch to batch; the patient as a central manufacturing input; and supply-chain orchestration for N-of-1.

圆桌讨论高度个体化批次中演示可比性、控制与产品特异质量的监管预期与新兴指南；可逐批切换且符合 GMP 的超柔性平台；将患者作为核心生产输入；以及 N-of-1 的供应链协同。

USP Biologics — Cell and Gene Therapy Expert Committee (Independent Consultant) · Kok-Seong Lim, Independent Consultant; Member, USP Biologics — Cell and Gene Therapy Expert Committee · Gene Therapy CMC & Analytics

In 2025, Baby KJ became the world's first patient to receive personalized CRISPR therapy in six months; the next step is scalable, cost-effective standard care. Explores strategic and technical foundations for scalable gene-editing platforms, regulatory innovations enabling rapid deployment, and reshaping the economics of personalized CRISPR therapy.

2025 年，Baby KJ 成为全球首位在六个月内接受个体化 CRISPR 疗法的患者；下一步是实现可规模化、低成本的标准治疗。探讨可规模化基因编辑平台的战略与技术基础、支撑快速部署的监管创新，以及重塑个体化 CRISPR 疗法的经济模式。

Advanced Cell & Gene Therapy LLC · Scott R. Burger, Principal · Cell Therapy CMC

Outlines FDA's flexible approach to CMC requirements for cell and gene therapy products seeking BLAs under 21 CFR Part 601, showing how FDA maintains rigorous standards while adapting expectations to complex development challenges to bring therapies to patients with serious or life-threatening conditions more quickly.

阐述 FDA 对依据 21 CFR Part 601 申请 BLA 的细胞与基因治疗产品在 CMC 要求上的灵活做法，说明 FDA 如何在保持严格标准的同时调整预期，以更快地将疗法带给重症或危及生命的患者。

Denali Therapeutics Inc · Charles Morgan, Executive Director, Regulatory CMC · CMC for ADC & Next-Generation Conjugates

Talk on regulatory CMC considerations for antibody-drug conjugates. // TODO: detailed abstract not published on the CHI agenda page (research/bioprocessing-summit-2026/digest-regulatory-quality.md).

关于抗体偶联药物监管 CMC 考量的演讲。// TODO：CHI 议程页面未发布详细摘要（research/bioprocessing-summit-2026/digest-regulatory-quality.md）。

Bayer US LLC · Jigesha Dholakia, Director, CMC Analytical Strategy, DA01 Analytical Workstream Lead · Cell Therapy Analytics

As Bayer and BlueRock advance toward commercial readiness following RMAT designation from the FDA, phase-appropriateness becomes increasingly important. In transitioning to late-phase development and ensuring process comparability, assays must effectively capture critical quality attributes (CQAs) during release and extended characterization.

随着 Bayer 与 BlueRock 在获得 FDA RMAT 资格后迈向商业化就绪，分阶段适用性愈发重要。在过渡到后期开发并确保工艺可比性时，检测方法须在放行与扩展表征中有效捕捉关键质量属性（CQA）。

Sangamo Therapeutics · Santoshkumar L. Khatwani, Senior Director, Analytical Development and CMC · Gene Therapy CMC & Analytics

Strategies for a late-stage AAV program for accelerated CMC development in support of the approval pathway; a case study and data will be presented.

围绕一个后期 AAV 项目的加速 CMC 开发策略，以支持获批路径；将展示一个案例研究与相关数据。

AbbVie · Lingling Xia, Principal Research Scientist I, Purification Process Development—ADC · Purification & Recovery

Product-related impurities must be managed through a comprehensive strategy aligned with ICH Q6B guidelines. New modalities such as bispecific antibodies and DVDs increase impurity-control complexity due to unique structures and an expanded impurity spectrum; the talk focuses on low molecular weight species and their control.

产品相关杂质须通过符合 ICH Q6B 指南的综合策略加以管理。双特异性抗体、DVD 等新型模态因结构独特、杂质谱扩大而增加杂质控制复杂性；演讲聚焦低分子量物种及其控制。

Biologics CMC Consulting · Kevin Zen, Principal Consultant · Training Seminars

One-day training seminar giving a comprehensive overview of phase-appropriate CMC activities for biotherapeutic products and introducing the brand-new CTD Quality guidance (2026). Covers bioprocessing (cell-line and process development, qualification, manufacturing) and analytical activities (development, validation, reference-standard qualification, formulation).

为期一天的培训研讨会，全面概述生物治疗产品分阶段适用的 CMC 活动，并介绍全新的 CTD 质量指南（2026）。涵盖生物工艺（细胞系与工艺开发、确认、生产）与分析活动（开发、验证、对照品确认、制剂）。

Gilead Sciences · Anthony R. Mire-Sluis, Senior Vice President, Global Quality · Plenary Keynote — Quality

Digitalizing quality systems and AI could revolutionize quality work but need careful planning and execution. Appropriate use cases, change management, training and streamlining processes before digitalizing are essential — adding complexity just results in digital complexity. AI implementation must follow GxP principles in what is currently a vague regulatory framework.

数字化质量体系与 AI 有望变革质量工作，但需谨慎规划与执行。在数字化之前确立合适的用例、变更管理、培训并精简流程至关重要——盲目增加复杂度只会带来数字化的复杂度。在目前尚模糊的监管框架下，AI 的实施必须遵循 GxP 原则。

MasterControl Inc. · Jake Ure, Senior Product Manager · Continuous Processing

Sponsored vendor talk. Bioprocessing manufacturers range from paper-based to digitally mature. Presents strategies for implementing purpose-built, compliant AI that delivers measurable ROI — reducing quality-review time by ~30% while keeping humans central — and balancing innovation with compliance in manufacturing.

赞助商演讲。生物工艺生产商从纸质流程到数字化成熟度参差不齐。介绍如何实施专门构建、合规的 AI，以带来可衡量的投资回报——将质量审核时间缩短约 30%，同时保持以人为核心——并在生产中平衡创新与合规。