Name: Quality and Compliance Requirements For Cell and Gene Therapy Products
Start: 2023-12-08T08:30:00Z
End: 2023-12-08T08:32:00.000Z
Location: BrightTALK
Rating: 0

BioPharma Webinars aims to keep its global audience abreast of all developments in the areas of Drug Development, Manufacturing, Quality Assurance, Outsourcing and Regulatory Affairs, with only the highest quality webinars, presented by the most respected people, working with companies in Pharmaceutical, Biopharmaceutical, Biologics and Biotech Industry.

Presented by Dr. Parthasarathy Sampathkumar, Head, Group Center for Operational Excellence at Biocon Biologics and Sara Jane Bresee, Managing Director of APAC at MasterControl

This presentation will cover The Opportunity Landscape & Methodology utilized at Biocon to drive OE, The Group Level Structure & glimpses of The OE Framework.

Opportunities Landscape & Methodology

At Biocon, we believe that the most fundamental way of identifying Opportunities is by virtue of “The 3Ms.” These being Mura, Muri & Muda. These are Japanese terms that stand for as Un-evenness or variability, Overburden or Under-capacity and Wastes, respectively.

The Group COE (Center of Excellence) Structure

The purpose of The COE Structure is to identify, exploit and translate the opportunity landscape into viable improvements under the umbrella of Business Excellence Framework providing Centralized Governance and driving Institutionalization of OE Leveraging Digital Transformation.

Operational Excellence Framework – An Introduction

The OE Framework is an Agile, Scalable, Holistic yet Flexible Model with a capability to Balance, Integrate and Assimilate all existing and any future initiatives under One Umbrella including Digital Interventions. This encompasses the Strengths of tried and tested International Excellence Frameworks with high internalization & contextualization to The Biocon Group in totality. The individual elements of The Framework will enable seamless delivery of Business Objectives with a strong linkage to robust Business Processes.

Leveraging Digital Transformation To Deliver Operational Excellence

Full title: Evaluation of Optimal Extraction Conditions for Polypropylene, PBT Resins, and Chlorobutyl Rubber Elastomer - Using a Variety of Solvents and Extraction Conditions

Chemical safety assessment is one necessary step to demonstrate that the polymer used is free from harmful chemicals. The assessment should start with adequate material characterization using state-of-the-art analytical methods, including various chromatography techniques most often hyphenated with mass-spectrometric detection. Since polymers are usually incompatible with direct analysis using chromatography-based techniques, an extraction step is required prior to analytical testing. This paper provides information for practitioners who need to understand how to develop science-based extraction conditions that enable the determination of the polymer composition with respect to volatile compounds. Analytical data sets for three types of polymers (polypropylene, polycarbonate, polybutylene terephthalate, and a rubber elastomer) are presented using three different solvents and three different extraction techniques. The extracts were collected at different times during extraction and analyzed by GC-MS. The differences observed between extraction techniques, conditions, and solvents are discussed to better understand the impact of these parameters in extractable studies.

Evaluation of Optimal Extraction Conditions for Polypropylene, PBT Resins...

Presented by Sethu Madhavan, Global Head of Insulin Operations at Biocon Biologics and Yvan Ruland, Head application APAC - Sartorius Chromatography Equipment

-Migrating Bioprocessing into the Existing Infrastructure with Carefully Controlled Risk Management Systems

-Addressing The Opportunities and Challenges Associated with Continuous Bioprocessing and The Importance Data Integrity and Management

-Discussion On The Challenges Specific To Continuous Manufacturing Such as Process Optimization, Process Characterization, Automation, In-Line Process Analytics

-Emergence Of Continuous Manufacturing As The Preferred Manufacturing Platform

Challenges And Opportunities Of Continuous Bioprocessing And Risk Management

Full title: Advanced Technologies for Media and Process Optimization for Biologics Manufacturing

Cell culture media composition and upstream bioprocess conditions are crucial factors affecting protein yield in the production of biologics and biosimilars. Recently, advanced new techniques were developed that use IT approaches, including computer simulation of bioprocesses, AI tools and ‘multi-omics’ methods for analysis of cellular metabolic pathways. These techniques allow optimization of both process and media composition for maximizing culture performance and protein production. In my talk, I will discuss the importance of cell culture media optimization and feeding conditions for achieving the most out of biologics pipelines. I will also discuss about other supplements that can help achieve faster cell growth and higher productivity.

Main topics of my talk:

DoE approaches for media formulation and feeding optimization

‘Digital Twins’ bio-simulations for reducing wet data requirements and allowing rapid optimization of feeding manner

Multi-omics and metabolic flux analysis for identification of metabolic bottlenecks and critical media components, for development of enhanced basal and feed media

Supplements for productivity enhancement

Advanced Technologies for Media and Process Optimization for Biologics Manu...

Full title: Navigating The Complexities of AAV Scale Up to Keep Pace with the Increasing Demand For these Viral Vector Delivery Systems

Presented by Seth Levy, Director of Bioprocess Development at Modalis Therapeutics and Gael Nicolas, Senior Technical Sales Specialist at Refeyn

Modalis’ epigenetic editors are designed to efficiently target genes and control chromatin conformation by coupling an engineered Cas9 variant with epigenetic effector domains; referred to as GNDM (Guide Nucleotide Directed Modulation), which is delivered via a single AAV vector.

Most AAV capsids used for systemic treatments require high doses,

leading to safety concerns. To overcome such obstacles, Modalis has evaluated numerous novel AAV capsids allowing for reduced dosing, increased safety margins, and higher

therapeutic efficacy. These novel capsids do not always play nicely with standard manufacturing techniques and available reagents, so creative process development and analytics are needed to bring these promising therapeutic molecules to patients.

Navigating The Complexities of AAV Scale Up to Keep Pace with the Increasing...

Full title: Validation of Test Result Equivalence for Alternative BET assays, Including Recombinant Methods

Both FDA’s 2012 Guidance for Industry and USP <1223> specifically use the term, “equivalence” as a requirement for validating an alternative test. Traditional methods, such as the Limulus amebocyte lysate (LAL) assay, are widely accepted and have a proven record of assuring parenteral drug and device safety, but they have limitations, including reagent variability and sustainability concerns. As a result, new methods using a number of innovative platforms, including recombinant technology (rFC and rCR), have emerged. The concept of test result equivalence bridges the gap between old and new technologies, offering cost-effective, environmentally friendly, and reproducible means of endotoxins detection, advancing the industry's ability to ensure continued product quality and patient safety.  This webinar will explore the concepts of equivalence, comparability, and statistical methods used to evaluate them.

Validation of Test Result Equivalence for Alternative BET assays...

It is a regulatory requirement (ICH Q5A Revision 2) to demonstrate viral safety of biotechnology product derived from in vitro cell culture, including viral-vector derived products like recombinant Adeno-Associated virus used as a delivery medium for gene therapy products. Viral clearance in gene therapy product poses different challenges from the other non-viral biotechnology product in the viral removal strategies. In this webinar, we will present a case study with a clinically relevant AAV8 downstream process:
1 The rationale of the study design: the choices of virus panel, surrogate and the unit operation for demonstrating viral clearance.
2 Present the clearance achieved in a scaled down model of the process

The Importance of Viral Clearance and Characterization in Gene Therapy Products

Presented by Pallavi Karnik, Analytical lead, Cell and Gene Therapy, Intas Pharmaceuticals Ltd.

Analytical Characterization of Viral Vectors and Plasmids

Full title: Current State of PAT Applications for a Reliable and Effective PCM Equipment Operation

PAT is an integral part of every Pharmaceutical Continuous Manufacturing (PCM) control strategy. On the other hand, PAT sensors can cause equipment and process inefficiencies due to the complex nature of those instruments. Sensor technology, data volume, sensor maintenance or the interface to the process contribute to operational complexity.

Nonetheless, PAT is required to measure Critical to Quality product Attributes (CQA), on-line or at-line and is enabling Real Time Release strategies. Furthermore, PAT sensors are also used during process and predictive model development, CQA monitoring, diverting Out of Specification (OOS) material and thus are essential for Real time Release testing applications.

Consequently, the selection of PAT applications is a critical step in every PCM business case assessment and during the entire engineering phase of a PCM system. This presentation will provide an overview of commonly used PAT sensor technologies and their selection criteria. Furthermore, it will revisit process design criteria for the optimal positioning of those sensors within the given process flow.

The overall control strategy defines what level of PAT implementation is necessary to run a successful continuous operation. All examples will be supported by real world PAT applications in operating PCM installations. 

Finally, this presentation should provide confidence in available PAT technologies. Innovation will aim for new even more sophisticated sensor technologies and new analytical software tools, but today PCM drug substances and drug product applications are covered and allow lead users to achieve the expected Overall Equipment Effectiveness (OEE) and required Net Present Value (NPV).

Current State of PAT Applications for a Reliable and Effective PCM...

Full title: Navigating the Digital Transformation for Biopharmaceutical Process Development: Harnessing the Power of Microbial Systems and Innovative Technologies

In this webinar, we will explore biopharmaceutical process development using microbial systems, such as bacteria and yeast. Key aspects include optimization of target protein expression, downstream process development, and high throughput approaches for rapid parameter screening.

The innovative smart Process Design platform will be central to the webinar, streamlining workflows and considering capacity limitations during process development. The importance of automation, parallelization, and integrated process models will be highlighted, enhancing efficiency, and facilitating data-driven decision-making for continuous improvement in bioprocess development.

The webinar will emphasize the unwavering commitment to leveraging advanced technologies and data-driven strategies in delivering innovative bioprocess solutions. This dedication drives the digital transformation of the biopharmaceutical industry, ensuring its position at the forefront of scientific and technological advancements.

Attendees will gain insights into the latest trends and best practices in biopharmaceutical process development using microbial systems. The webinar will underscore the significance of adopting a forward-thinking approach to process development, leading to more efficient, cost-effective, and sustainable solutions for the biopharmaceutical industry.

Navigating the Digital Transformation for Biopharmaceutical Process...

Full Title: De-bottlenecking Challenges in Large Scale Viral Vector Manufacturing for Gene Therapy Applications

The rush to develop an affordable and accessible COVID-19 vaccine not only accelerated research and development of therapeutic modalities like viral vector, mRNA and pDNA based vaccines but also challenged the bioprocess community to solve for unique scale-up related challenges to manufacture these important life-saving medicines. 

Adenoviral vectors are attractive vectors for delivering genes for other gene therapy applications. These vectors can accommodate large transgenes, transduce quiescent and dividing cells, and do not integrate into the host’s genome. Adenoviral vector technology using HEK293 host cells has been around for a long time and the manufacturing process includes upstream processing (HEK293 cell culture and viral infection), followed by series of clarification, filtration, and chromatography steps to produce drug substance with high purity. Although, the process unit operations seem uncomplicated, manufacturing at large scale in single use systems has remained a major challenge due to poor and inconsistent cell growth in suspension culture during scale-up, low viral titers, product aggregation, inefficient separation of empty and filled viral capsids, as well as complexities related to handling large volume viral infection. Hence, there is a strong requirement to build robustness and scalability in early process development to minimize unnecessary late-stage process changes, as well as design cGMP facility and strong quality culture to handle operational complexities and reduce risks associated with viral vector manufacturing. The webinar will illustrate key questions and challenges in large -scale manufacturing of viral vectors from a process robustness, facility design and product quality standpoint as well as our experiences in debottlenecking these challenges to scale adenoviral vector technology at 2000L and beyond.

De-bottlenecking Challenges in Large Scale Viral Vector Manufacturing...

Full title: A systematic approach to deliver data science solutions for bioprocess development leveraging digital infrastructure and data management solutions

A streamlined approach to analytics can play a crucial role in optimally using digital infrastructure and data management solutions to realize the full potential of advanced analytics for process development. We will discuss our systematic approach to develop scalable advanced analytics solutions including multivariate statistics, digital twins based on AI/ML, mechanistic or hybrid approaches, and more.  Emphasis will be laid on the strategy to democratize and decentralize development efforts and empower citizen data scientists to use these methods to bring new medicines to patients. A diversity of skill sets required to deliver these solutions, including collaboration with our IT partners will be presented. We will also share our experience in employing cloud-based analytics platforms to build an eco-system of data science products that are widely accessible for end-user consumption as well as to inspire other potential citizen data scientists.

A Systematic Approach to Deliver Data Science Solutions for Bioprocess...

Full title: A Systematic approach to define operating space and MRT for continuous manufacturing ER tablets: Opportunities and Challenges

Presented by Ahmed Zidan, Senior Staff Fellow (Pharmacologist) Center for Drug Evaluation and Research at FDA, Owen Rehrauer, President of Sentronic US Corp and Rodolfo J. Romañach, Ph.D. Center for Structured Organic Particulate Systems (C-SOPS) at the University of Puerto Rico

Twin-screw wet granulation is an emerging continuous manufacturing (CM) technology for solid oral dosage forms. Compared to batch operation, this technology offers several advantages including reduced processing time, uniform granule size distribution and shape, and real time process control. Continuous granulation process has been successfully employed for commercial manufacture of IR tablets. However, the high polymer content in the ER formulations has poised challenges by narrowing the design space of screw configuration and operating parameters. This design space defines the flow dynamics within the barrel and plays a crucial role in determining the granule properties. This work pursues a systematic approach for defining the optimum operating space and mean residence time (MRT) through understanding the interaction effects of the screw design and processing parameters on the critical characteristics of granules and tablets. The study identified further the opportunities and challenges of using CM granulation for manufacturing of ER formulations containing high load of swellable polymers.

This presentation will showcase a systematic approach for optimizing the operating parameters and process understanding of a twin-screw granulation to manufacture ER tablets. Examples of failure modes will be discussed, along with the implementation of quality-by-design coupled with PAT tools to monitor the critical quality attributes of granules.

A Systematic approach to define operating space and MRT...

Full title: Applications of Next Generation Sequencing for Viral Safety in Light of ICH Q5A R2

Applications of Next Generation Sequencing for Viral Safety...

Solvent Selection for Controlled Extraction Studies of Pharmaceutical Packaging Components: An Experiment-Based Approach

The 2006 recommendations from the Product Quality Research Institute (PQRI), Leachables and Extractables Working Group stated that extraction studies accomplished on pharmaceutical container closure/delivery system components should include “multiple extracting solvents of differing polarity”. This recommendation was later incorporated into USP General Chapter <1663>.  The intent of the recommendation was to ensure that extraction studies were comprehensive, and therefore had the greatest opportunity of discovering all potential leachables that could accumulate in a drug product over its shelf-life.  Since the laboratory studies accomplished by this PQRI working group focused on inhaled drug products and particularly on metered dose inhalers, the solvents used were dichloromethane, isopropyl alcohol, and hexane.  A second PQRI working group studied injectable and parenteral drug products which are aqueous-based, and therefore their laboratory work included water (with bracketing pHs) as an extracting solvent.  This presentation will summarize the scientific basis for extracting solvent selection for packaging components for all drug product types, including a discussion of the use of polarity index and other physical-chemical parameters.  Data obtained from extraction studies on various plastics and elastomers will be discussed. The possibility of developing standard methods for extraction studies will also be explored.

Solvent Selection for Controlled Extraction Studies of Pharmaceutical Packaging

Presented by Karen Zink McCullough, Owner, Principal Consultant at MMI Associates and Allen L. Burgenson, Global Subject Matter Expert, Testing Solutions Lonza Walkersville, USA, Timothy Francis, Senior Technical Specialist for the LAL Division of FUJIFILM Wako Chemicals U.S.A. Corporation

Variability, Limits, and Other Considerations for the Next Generation of Pyrogen

Presented by John F. Cipollo, Ph. D. Team Lead and Senior Principal Scientist, Global Biologics and Khaled Yamout, Senior Director, Analytical Services and Quality Control at TriLink Biotechnologies.
Following the rapid and successful deployment of mRNA vaccines during the COVID-19 pandemic, over 150 mRNA vaccines are in development, with further therapeutic applications in oncology, cardiovascular and genetic diseases and forecasts predict a high level of growth in products using this drug manufacturing platform. USP engaged with stakeholders in government, drug development, pharmaceutical companies and other experts to build consensus on relevant quality attributes for mRNA-based products and appropriate analytical methods. As part of this effort, USP worked with vaccine experts to generate draft guidelines (Analytical Procedures for mRNA Vaccine Quality) and solicited feedback from stakeholders to update and evolve the guidelines. This presentation will provide an overview of USP updated guidelines and plans for further assessment and qualification of analytical methods presented therein. Physical, chemical, and structural elements of drug substance and drug product and how they relate to product quality attributes will also be discussed.

mRNA Quality Assessment and USP Guidelines

Full title: A Digital Transformation Journey in Process Development – Building Automated Data Flows, from Equipment to eLN to Advanced Analytics
Sanofi CMC/Process Development is implementing a comprehensive Digital Transformation program to improve our productivity and reduce our development timelines. We are deploying a standardized digital workflow across our development sites globally (target: 2,000 users) and building automated data access to ensure scientists can document, visualize, and analyze their experimental work. We will discuss the progress, successes, and pain-points encountered by the development teams as we progress towards our digital ambition.

A Digital Transformation Journey in Process Development...

This presentation will outline Quality and Compliance Requirements For Cell and Gene Therapy Products which incudes”:

- Facility compliance

- GMP quality Management system

- Consideration in IPQA checks and batch release procedure

- Consideration in “ Patient to product tracking system”

- Temperature-controlled clinical trial supply chain

Quality and Compliance Requirements For Cell and Gene Therapy Products

Cell Therapy

Facility compliance

Gene Therapy

batch release procedure

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Quality and Compliance Requirements For Cell and Gene Therapy Products

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