Agenda

Registration open: Monday, 17 June 0730-1800 EDT.

Registration will also be open on Sunday, 16 June from 1300-1700 EDT.

Healthcare Equality - Technology and Innovation Applied in Service of Low- and Middle-Income Countries

The pandemic helped to highlight the inequitable access to vaccines that exists for patients in underserved communities worldwide.  Beyond vaccines, access to biopharmaceuticals including products like monoclonal antibodies and cell and gene therapies is also very limited.  As an industry, we can drive innovation, advanced manufacturing and commercial strategies in service of underserved communities.  Low- and Middle-Income Countries (LMICs) face major public health challenges and in most cases, the typical incentives for discovery, development and manufacturing of these life-saving products are not in place for traditional pharmaceutical and biotechnology companies. Unique partnerships and collaboration models between companies, funding bodies, non-profits and international organizations can help tackle the most devastating diseases.

Even with development funding, there are a few key challenges to enable LMIC access to biopharmaceuticals, including lowering cost of goods (COGS) and building capability, capacity and infrastructure for local and regional manufacturing.  There are a number of levers for reducing COGS in biological products to support use in LMICs. We are utilizing several of these in our Gates MRI late stage development programs.  In addition, we hope to engage in capability building with local manufacturers as we identify companies, often in LMICs, to ultimately produce our products for commercial sale.  We should also consider other factors such as policy, funding, market access, uptake and stakeholder acceptance that will help us deliver healthcare solutions to those in greatest need.

From Nanotechnology to mRNA Vaccines: How Overcoming Skepticism Led to New Medical Treatments and Ways to Tackle a Global Health Challenge

Advanced drug delivery systems are having an enormous impact on human health. We start by discussing our early research on developing the first controlled release systems for macromolecules and the isolation of angiogenesis inhibitors and how these led to numerous new therapies. This early research then led to new drug delivery technologies including nanoparticles and nanotechnology that are now being studied for use treating cancer, other illnesses and in vaccine delivery (including the Covid-19 vaccine). Finally, by combining mammalian cells, including stem cells, with synthetic polymers, new approaches for engineering tissues are being developed that may someday help in various diseases. These can also serve as a basis for tissues on a chip which can potentially reduce animal and human testing. Examples in the areas of cartilage, skin, blood vessels, GI tract and heart tissue are discussed.

Towards Individualized Medicine: Embracing Digital Twins and Digital Dependability in Pharma 4.0

A significant shift towards decentralization and modularization is currently unfolding in the field of pharmaceutical manufacturing, starting a new era characterized by increased flexibility and efficiency. This talk seeks to explore the critical role of automated manufacturing, digital twins, and digital reliability, while emphasizing the importance of enhanced interoperability and transparency inherent in Pharma 4.0. Central to this evolution is the adoption of modular processes, where different components undergo independent evaluation and certification, thereby enhancing the overall agility of the manufacturing ecosystem. By leveraging existing technologies such as digital twins, these modular processes are automated, allowing for seamless connectivity between systems and devices. This promotes interoperability and enables the system to dynamically adapt to specific requirements, such as personalized medicine. In addition, digital twins represent a cornerstone in the journey towards enhancing quality assurance in pharmaceutical manufacturing. As virtual replicas of physical processes, they offer manufacturers the ability to proactively simulate behavior, providing insight into both product quality and process effectiveness.

Imagine a future in which fully personalized medicines are manufactured quickly and safely, tailored to individual patient needs while maintaining necessary standards of quality and safety. Achieving this vision requires transparency and accountability especially for digital tools. Moreover, robust documentation of the manufacturing process, along with the strengthening of reliable software systems and the integration of explainable AI, is essential to ensuring regulatory compliance as well as confidence in the products procured.

This keynote explores approaches to realize digital transparency, advocating for automated documentation of data sources and decision-making processes. Utilizing different concepts proposed within the scope of Industry 4.0 systems, including the digital twin and adding concepts for product and patient safety, like “Digital Dependability Identities”. In addition, the discourse includes the integration of continuous engineering and simulation methodologies, highlighting their central role in optimizing manufacturing processes and ensuring product quality. By facilitating the coupling of various tools throughout the development lifecycle, these methodologies foster interactions between design, simulation, and engineering processes, thereby increasing overall efficiency and effectiveness.

In summary, the convergence of distributed manufacturing, digital twins, digital reliability, continuous engineering, simulation, and tool coupling holds the promise of revolutionizing the pharmaceutical industry. By leveraging the insights of these technologies and methodologies, the objective is to improve production, enhance safety, and stimulate innovation. Let's work towards a future where pharmaceutical manufacturing is not just efficient but becomes a model of reliability, transparency, and excellence, thereby advancing the accessibility and efficacy of healthcare on a global scale.

Centralized to Decentralized Manufacturing supporting a global market to avoid supply chain disruption. Beside the manufacturing challenge transfer the process to different location the regulations requirements may also be different. How to overcome decentralized Manufacturing challenges and how this could be support from the industry in dialog with the regulatory agencies.

AI has the potential to significantly impact pharmaceutical manufacturing. AI offers potential benefits to pharmaceutical manufacturers in the form of optimized process design and control, and smart monitoring and maintenance, to drive continuous improvement. In 2021 CDER announced to the public the FRAME (Framework for Regulatory Advanced Manufacturing Evaluation) initiative, which focuses on the policy elements and regulatory framework to enable the regulatory assessment and broader implementation of advanced manufacturing technologies, such as implementation of AI.  This talk will provide information on on-going efforts to implement a cohesive regulatory framework to support AI in drug manufacturing.

In order to address the many environmental challenges we face as a society, companies including biopharma companies have set ambitious sustainability targets for 2030 and beyond.  These targets, including reductions in energy & CO2, water and waste, require us to think differently about our operations in order to move towards a more sustainable model of resource use.  In this session we will discuss the various technologies and strategies that Sanofi is using and exploring to achieve reductions in energy and water use, carbon footprint and material used.

Considering shortages have been tied to quality related concerns, this session will focus on the practice areas highlighted in FDAs Quality Management Maturity prototype which integrate an ICH-based quality maturity framework to support industry's assessment and impact to the overall performance and of their quality maturity. The overall intent of the program is to further develop resilience to shortages which result from quality-based issues. The basis of each practice area which should be considered in developing a framework for assessment will be reviewed to provide a foundation to develop sustainable internal programs. All of the attributes within the protype support the development of a strong quality culture. An ideal quality culture is the ultimate goal for each organizations, the prototype provided by the FDA supports standardizing the process in which industry is able to assess culture and management maturity.

GSK’s EcoDesign Program: Enabling Sustainability Improvements in Biopharmaceutical Manufacturing

GSK has set aggressive corporate environmental sustainability goals to be net zero and net nature positive by 2030.  To achieve the 2030 goals and beyond, GSK needs to incorporate environmentally sustainable design practices in all of its product and process development work.  As further substantiation of this need, in 2014, the European Commission estimated that more than 80% of the environmental impact of a product is determined at the design stage of product’s life.  Accordingly, the GSK “EcoDesign” program, developed over the last two years, includes a new tool called the Product Footprint Calculator Tool (PFCT) that assists drug substance development scientists and product stewards to understand the characteristics across all manufacturing phases which significantly contribute to a product’s environmental footprint. In most cases, the API/drug substance manufacturing phase is the largest portion of a product’s footprint. PFCT is now being applied to the manufacturing processes being developed for both small and large molecules. 

PFCT assessments have been completed for several large molecule assets. Beyond understanding the key attributes of a manufacturing process that significantly impact sustainability, the EcoDesign assessment provides a baseline understanding to build key product lifecycle roadmap improvements that should be considered and addressed prior to committing to commercialization. This presentation will share the results of the product footprint calculations for several GSK Biopharmaceutical manufacturing processes, highlighting where design decisions and process details contribute to differential environmental impacts.

Join this discussion to learn how the Pathway Chief Quality Officer Team is working to advance the adaptability and agility of their organizations. In order to increase your ability to pivot for the patients who are counting on you, our team has integrated quality maturity into a larger enterprise-wide model. Take away insights related to key drivers such as supply chain capability mapping, “enterprise” risk management, infrastructure, investing in resources, ownership of quality, regulatory strategy and achieving employee empowerment.

Here we will discuss the opportunities and risks in the dynamic regulatory environment focusing on perand polyfluoroalkyl substances (PFAS). Removing certain PFAS from the pharmaceutical industry, where fluoropolymers are primarily used as membranes in fluid and gas filtration processes, would change how pharmaceutical filtration occurs due to their use during drug manufacturing to provide safe and effective therapies to patients. However, there may be opportunities to use existing materials as well as to innovate new materials to satisfy today’s applications in bioprocessing with technologies that could surpass current performance. Minimizing the use of persistent materials can lead to sustainable opportunities, and providing alternative solutions could lead to significantly reducing the environmental footprint for biomanufacturing to improve operational excellence through regulatory insights to enhance industry efforts to ensure reliable quality medicines are available to patients.

Quality Culture in the Pharmaceutical Industry (Case Studies)

The Quality Culture of regulated organisations is becoming increasingly more important. Although no regulations currently describe the expectations for Quality Culture, the regulatory focus is changing from essentially an evaluation of the state of compliance, towards an emphasis on the quality behaviours and mindset evident throughout an organisation. The outcome of poor Quality Culture includes risk to the patient and product, risk to the business, a decrease in trust by the customer and regulators, and uncertainty for quality and production leads. The Quality Department and regulators rely on the information provided to them to determine if a product will meet the specified quality attributes and marketing authorisation requirements. As such, the integrity of the individuals and teams performing the manufacturing and support functions, and the accuracy, integrity and completeness of information is of the utmost importance. This presentation looks at the impact of poor Quality Culture on the safety and integrity of the manufactured product, and how a company can apply strategies to implement an improved Quality Culture and benefit from the promotion and continuation of behavioural change throughout their organisation.

We encounter challenges when setting up quality management for a new business or revamping an existing quality system, such as facility, process, system, and resource management. In planning and due diligence evaluation, most firms take into account the factors mentioned above; however, the quality culture of the company is given the least attention, which determines the level of investment and development strategy of the top management.

The quality culture of some great matrix structured companies is not achieved to acceptable standards, particularly after the acquisition of facilities, which serves as an expansion for their business in various parts of the world. Underestimating the impact of quality culture in the workplace may lead to a failure model or the closure of pharmaceutical manufacturing facilities as a result of a chain of events affecting the management strategies. This presentation summarizes the importance of setting up a strong quality management system to achieve the competitive potential of the company and key fundamentals forgotten by the industry and the relationship between leadership commitment and quality culture, as well as the challenges associated with building a quality maturity model due to quality culture concerns.

This presentation will explore detergent uses in viral-vector and LNP therapeutics by examining the desired properties of industry standard detergents; long-term outcomes and considerations; process innovation by sourcing potential replacements for legacy detergents to achieve safer product and environmental outcomes. Detergent & solvent selections are crucial due to their influence on process and product efficiency and quality. It is important for thorough evaluation of detergents and solvents in the development stages of a product line because process changes after market entry impacts process validation and therefore initial regulatory filings. Detergent and solvent selection should be based around the following criteria: (1) risk of discontinuation as influenced or enforced by local and foreign environmental regulations; (2) potency; (3) risk of residuals and consequential removal procedures, and (4) waste product treatment.

St.Gallen presents results from the US FDA funded project “RiskSurve” which has been concluded last year. The presentation focuses on the role of culture and leadership to establish a sustainable quality management program and support quality and compliance. The results rely on 30 interviews with multiple health authorities, site quality leaders, and corporate quality leaders. The presentation will focus on the role of leadership in shaping a quality culture, related benefits, and expectations from industry and regulators. Especially, the presentation will elaborate on the multiple elements characterizing a good quality culture differentiating between regulator’s expectations and industry’s understanding. While the main outcomes are based on qualitative research we will triangulate the results with quantitative data from St.Gallen’s research. The presentation concludes with an overview on the key takeaways for manufacturers to master their quality culture under the lenses of quality management maturity.

Applying the Biomanufacturing Facilities Baseline Guide: Case Study Examples that Define the Basis of Design

This session will present two case study vignettes where companies used the Baseline Guide concepts to form the basis of design for a new manufacturing asset. The case studies focus on key Guide principles that identify the synergies between process closure, operation efficiency, contamination risk mitigation, and facility optimization. Specific references to the Guide will be provided as part of the interaction with the Audience.

Leadership’s commitment to quality is critical to upholding a strong quality culture. Leaders must ensure that an effective quality management system is in place as well as model the desired behaviors the sustain culture. This presentation will provide examples how quality management system program elements and behaviors go hand-in-hand to support robust quality culture. The presentation will describe how leadership responsibilities must include effective communication and presence as part of the quality management processes such as management review. The presentation will provide tips for leaders to evaluate culture. Leaders in the context of the presentation include technical and administrative leadership.

The ISPE Advancing Pharmaceutical Quality (APQ) Program has been developed by industry expert representatives, for industry use, to advance the current state of pharmaceutical quality by providing practical tools and approaches to enhance the effectiveness of the Pharmaceutical Quality System (PQS). The APQ program is a practical framework that an organization can use to first assess the maturity and then advance the state of quality within their organization. This session will provide an overview of the APQ program—its basis in ICH Q10, the Assess-Aspire-Act-Advance framework, and the guide series.

Beyond Boundaries: AI-Driven Efficiency in Modern Workspaces - Powered and led by ISPE Women in Pharma® 

The Goal of this session is to encourage all generations and cultures to fearlessly use AI during their daily business. In the whirlwind of fast-track projects, daily decision-making, and relentless multitasking, modern professionals find themselves at the intersection of innovation and efficiency. The demands of a dynamic work environment, accentuated by a constant barrage of meetings and pressing deadlines, necessitate a reevaluation of our working models. This panel discussion aims to explore how artificial intelligence (AI) can be seamlessly integrated into the fabric of our daily professional lives, offering solutions to enhance efficiency and work-life balance.

Join us in this exploration of the intersection between AI and the modern professional landscape, as we navigate the challenges of fast-paced projects, time zone differences, language barriers, and varying generational approaches. Discover how AI can revolutionize your daily work routine and propel your efficiency to new heights. Welcome to the future of work, where the fast lane meets intelligent innovation.

The advancement in technologies and innovative thinking are profoundly changing biopharmaceutical drug production industry holistically, giving rise to “the factory of the future.” What will the factory of the future with respect to its structure, technologies, processes, and drug product delivery to the patient be? What enablers will manufacturers need in terms of strategy, leadership, employee skills, IT infrastructure, regulatory oversight, and suppliers to make this a reality? The factory of the future is an innovation vision on how manufacturers should enhance production by transforming manufacturing in three dimensions: plant structure, plant digitization, and plant processes.

• Having a holistic vision of the process
• Designing and implementing a flexible framework
• Evaluating and implementing proper technology and skill
• Emphasizing safety, quality, efficacy and regulatory mandates
• The bottom line – delivering ROI

Good Practices to Achieve Data Quality and Data Maturity for the Product Life Cycle

High data quality, data integrity and data maturity are an essential prerequisite for process characterization and technology transfer. However, we still lack good practices, which are product and process agnostic, in the way we do data alignment, data contextualization, handling of data with different dimensionalities and frequencies. We show those tools embedded in automated and data integrity checked data import routines providing a holistic contextualized data hub. This allows for peeling out thorough process understanding and robust and holistic control strategies as they are key in process characterization. The shown approach will smoothen and therewith accelerate the tech transfer and validation activities significantly.

With the new Annex 1 being effective since August 25, 2023, the industry needs to be compliant to these requirements. One big topic of the Annex 1 is the Contamination Control Strategy (CCS) which shall bring all control measures together. The presentation will highlight how a quality by design approach of an aseptic filling line will benefit the CCS based on an example of a gloveless filling line. Besides regulatory requirements, ATMPs require a special way of manufacturing due to the fact that they need to be distributed to patient as fast as possible, highlighting a decentralised and local production. The presentation will have a close look on how gloveless fill and finish systems can benefit the future of manufacturing of ATMPs with a special focus on global scale out strategy.

In the pursuit of enhancing the cadence and efficiency of technology transfer in biopharmaceutical manufacturing, GSK is transitioning to a recipe centric approach to drive our Data Science-Assisted efforts in this Biopharmaceutical Tech Transfer. Some of historical perspective in clinical manufacturing, our digital vision of tech transfer, and some successes to date will be used in an engaging presentation. This will cover several case studies sharing GSK’s experience in this area, including plant-specific initiatives to enable data availability and flow; cross-site engagement to enable digital technology transfer ways of working, and data use modelling activities including MSPM and model-predictive control. The presentation will conclude with a forward-looking perspective on the need for improved and enduring data infrastructure.

CAR-T landscape is witnessing much awaited FDA approvals. Newer therapeutic applications continue to emerge at a rapid pace. Having established the scientific foundation, the question of affordability becomes the next challenge that the industry must overcome. The presentation discusses end-to-end, fully automated, closed manufacturing as the key strategy towards a more affordable CAR-T drug product. The presentation includes a discussion of the following: Contributors to COGs including raw material and labor. Focus on three manufacturing related contributors: facility, labor and raw material Cost reduction approaches for manufacturing and facility solutions

Regulatory Challenges for a Machine Learning Solution in the GxP Space

The presentation will show the implemented approach for the initial validation upon the Life cycle implemented for the development, release and maintenance of the Machine learning embedded in the SW Solution, focusing upon the determination of model performance (e.g., prediction accuracy and model sensitivity) and the adequate sizing of the dataset for the associated evaluation. In addition, the presentation will describe the established mechanisms by which the performance of the model is monitored and the criteria which may trigger a model update, in case data drifts are observed. The implemented Life Cycle allowed to create and maintain the required qualification documentation of Machine Learning to be embedded in the Validation documentation, which allows to meet the current regulatory requirements ensuring the Accuracy of the outputs generated by the Machine Learning solution and ultimately the compliance against the ALCOA+ expectations for the entire ecosystem.

In the face of persistent global challenges posed by microbial and viral pathogens, adapting vaccine production to emerging threats demands a shift towards flexible and platform-oriented approaches. An innovative solution is the use of low-energy electron irradiation (LEEI) in liquid suspensions.

This technique reliably and safely inactivates a spectrum of organisms, spanning eukaryotic, microbial, and viral entities relevant to both veterinary and human diseases. The LEEI platform offers key advantages: 1) Continuous operational capability, 2) Seamless integration into high-security laboratories, 3) Dependence on electrical energy, eliminating the need for toxic chemicals, and 4) Affordability, enabling vaccine research and production campaigns in emerging markets. Collaborating with the Fraunhofer Research Society, we successfully demonstrated the proof-of-concept for the LEEI approach in initial animal studies. Case studies on the Zika virus and TBEV underscore the immunological relevance of LEEI-treated viral vaccines.

Beyond vaccine manufacturing, LEEI technology holds promise for diverse applications, including processing serum and blood products and treating infectious liquid waste. This multifaceted approach not only addresses pressing challenges in global vaccine production but also extends its impact to broader fields of healthcare and environmental sustainability.

End-to-end digital twins describe the entire manufacturing process with all relevant unit operations. They enable predictions of process parameter influence of any process step on critical quality attributes on drug substance level. This is how they differentiate from classical models of individual unit operations that cannot make statements on final product quality. In this talk we want to showcase the first time how such end-to-end process models, that are formed in process characterizations, can be applied to manufacturing use cases. We will demonstrate how an end-to-end model can predict intermediate and drug substance quality based on process performance. Such a model can aid in creating impact assessments, allows for early detection of events, and acts as a copilot in the monitoring of manufacturing processes.

Automated Intelligence (AI) is a mature product that has been used for almost a decade in quality vision systems. AI is branching out into others parts of the manufacturing processes and for many of the front end paperwork required for cGMP. This presentation will show real world applications that are being used in 2024 for both manufacturing and legal areas to ensure compliance. AI is reducing the time to create quality documentation: FAT, SAT, IQ, OQ and PQ as well as SOPs: Cleaning, Calibration, Preventative Maintenance and Training. AI is also being used to help with monitoring counterfeiting on a global scale.

During these roundtables, experts will explore the benefits and challenges for implementation and highlight the "WHY" for using these respective technologies in the pharmaceutical industry:  Model validation & verification, Accelerating Product and Model Life Cycle from Process Characterization until Manufacturing, Data Governance, and New Data science Horizon for Tech Transfer.

After this interactive session, the outcomes will be discussed and connected through a panel discussion with the track speakers.

Pandemic vaccine distribution from COVID-19 has revealed many gaps related to logistics, costs, cold storage and shipment time. To address these issues, modular mRNA vaccine facilities have been condensed into shipping containers that were installed in Africa. Although this solves the access logistics, it cannot be extended to resource limited settings. To overcome these limitations, we propose an innovative approach: transforming a shipping vessel into an on-sea manufacturing facility dubbed MoSeS (Manufacturing On-Sea and Ship). This concept entails producing vaccines onboard while en route to destination countries/ports. This presentation delves into the broader possibilities of this approach including adaptation of flexible and modular facility components, quality assurance and quality control measures, and regulatory requirements for successful implementation of this on-sea manufacturing facility. This concept is a potential rapid response strategy for future global health crisis without border restrictions.

Over the last decade as there has been continuous growth and development in mAb production science and technology. Increases in cell line productivity and improved purification capacity science and technology offer a unique opportunity for a major shift in the biopharmaceutical industry towards accelerated product supply, flexibility, high throughput, lower cost per gram, and lower carbon footprint. This presentation provides critical research metrics on a number of future NGB Facility Concepts across a wide range of global mAb supply (500 – 20000 kg/year).

This presentation will feature a case study highlighting a methodology for the evaluation and selection of a new platform technology across multiple scales to accelerate technology transfer and commercialization of a portfolio of similarly behaving products. The proposed methodology is unique because it is thorough, while also sparing API and valuable analytical resources through the use of surrogate materials. It emphasizes applying experiential knowledge in identifying worst-case attributes within a portfolio of products, selecting surrogates based on these attributes and defining acceptance criteria so that technology candidates can be comprehensively vetted and confirmed to be robust enough to meet current and future portfolio needs.

OEM equipment is the foundation of a modular facility; it enables a faster time to market with high-quality validation-ready technology that reduces the overall risk on your facility's construction and qualification. However, each OEM has a different approach to their application software design that can lock a producer into a plant constructed out of operational and digital silos. This makes OEM coordination paramount for producer's ability to achieve their Pharma 4.0 vision. In this presentation learn how to collaborate effectively with your OEMs and automation technology partners to implement your Pharma 4.0 vision using practical specifications and the latest plug and produce technology.

Biology centered therapies generated in the last decade created a completely new classes of modalities as agents in service of target and personalized treatments for disease caused by metabolic, functional, or genetic condition in new ways. In the quest to make new drugs accessible faster to patients, new “ways of working” continue to challenge old paradigms in manufacturing of new generation drugs that are injectables. One of them is acquisition of e-data at product/equipment interface at time of introduction into production at any scale, any site or manufacturing trains. The signature of product/process interaction is easy to generate, is measurable, it correlates with processing parameters and is transferable as part of the technology transfer knowledge e-data package.  This presentation will describe the power and value of the unique type of knowledge acquired using SmartSkin’s digital replicas of vials, syringes, or cartridges in speeding up activities and integrating critical to quality requirements across Stage 1, Stage 2 and Stage 3 validation. Case studies will illustrate the enhanced agility to technology transfers across sites and type of equipment used in production.

In this presentation, we will explore a novel concept known as a Unified Knowledge Space (UKS) for Pharmaceutical Manufacturing.  We will first share the research performed on the issues currently affecting Lifecycle Management, Tech Transfers and Process Start-Up. This research consists of over 100 one-on-one interviews across all functions and levels of manufacturing.  We will then examine how other industries have addressed similar problems in their contexts and their successes with these novel lifecycle management methods. The derived concept of a Unified Knowledge Space will then be explained along with its traits and benefits to enable more resilient lifecycle management and to accelerate products to market.

As the landscape of biopharmaceuticals continues to evolve, the imperative to design advanced therapy manufacturing facilities that are both innovative and regulatory-compliant has become increasingly complex. A successful design requires a combination of tangible and intangible aspects. What are those aspects and how can you start collecting the necessary data?

Embarking on the design of a state-of-the-art facility necessitates a meticulous exploration of both tangible and intangible aspects. By engaging key stakeholders through workshops, we dissect the tangible components, including quantitative parameters such as modality, patients per year, process steps, equipment, operations, and storage requirements. Simultaneously, we delve into the intangibles, envisioning the qualitative impacts on society and patients, talent acquisition and retention, cultural development, human wellness, and the inspiration required to accomplish unprecedented achievements.

Closed Systems allows the opportunity for innovation when designing new biologic facilities, as the need for environmental control is reduced. Closed systems technology has been readily available for some time; however, they are still being used in cleanrooms with high classification by using the principles outlined in the BioPhorum Closure Playbook users can put closed systems within a controlled not classified (CNC) space making everything quicker and cheaper. Fundamentally they offer greater patient protection and provide a higher quality of product for patients.

We will also give details and examples from our new paper—A risk-based approach to filter integrity testing requirements for biologics drug substance manufacturers which we wrote because the updated Annex 1 is relatively explicit about FIT requirements for drug product facilities, no clear guidance is available on FIT requirements in drug substance manufacturing the papers aims to fill that gap with a risk-based, value-driven approach for FIT testing of sterilizing grade filters used to manufacture low bioburden drug substances, which are so important for Closed Systems.

To accelerate commercialization, the necessary production processes must be developed quickly without compromising safety and risk management. However, due to their inherent complexity, the risks in the processes are often manifold and can only be identified with a considerable amount of time and resources - this delays the market launch of important drugs and is often associated with major corrections, as some process weaknesses remain unidentified, jeopardizing the entire commercialization process. The main reason for the high-effort and error-prone nature of process design and risk assessment is that they have to be created largely manually. The presentation will introduce a novel process simulation approach that utilizes a generic database around so called "process-frames" (process-microsteps) to develop data-driven recommendations for process design and autogenerated risk management insights with unprecedented accuracy. By linking the "process frames" with numerous production data, a self-learning software has been created that also identifies potential risks for new process ideas and helps to achieve robust process design and CCS more efficiently. The presentation presents case studies from JnJ and Moderna and shows how "Frame-by-Frame Risk Profiling" has significantly accelerated process design and risk management - both prerequisites for faster plant and operational readiness.

With the rise of antibiotic resistant bacterial strains, therapeutic bacteriophages are emerging as both a potential alternative to antibiotics and as an antibiotic-synergistic treatment of bacterial infections. Bacteriophages are viruses that only infect and replicate in bacteria, often with very targeted infection of specific strains. Emerging therapies require CGMP manufacturing of host bacterial culture in large-scale (10L - 1000L) bioreactors and downstream processes scaled appropriately.

Manufacturing at-scale involves examining key process input variables for their effect upon key process outputs for all unit operations. For upstream operations this involves examining such variables as bioreactor temperature, time of infection, multiplicity of infection, and agitation for their influence on such outputs as bacteriophage yield, infection kinetics, and quality of phage. Downstream processes begin with separating the bacterial debris from the newly replicated phage particles through techniques including centrifugation or filtration.

Facility design begins with translating optimized net API yield potential to the equipment and processes determined to provide the mass/activity required. Then, prokaryotic culture facility design is established considering such factors as the selected mode and scale of each operation throughout the process train, and such options as the use of either single-use or stainless-steel equipment to implement them.

A holistic and integrated view in terms of culture but also digitalization and modelling is required to break up these silos and pave the road towards sustainable, affordable and accessible drugs. Over the last 7 years we have developed an end-to-end modelling framework that enables pharmaceutical companies to model processes from the first manufacturing step until the last, include patient models and grow those models along the product life cycle to achieve business goals like increased product quality and space time-yields more efficiently. This requires the mutual action of multiple stake holders such as USP, DSP, analytical, clinical development and manufacturing but also the combination of appropriate modelling techniques such as mechanistic, statistical and hybrid modelling under one umbrella. With this platform we demonstrate in case studies and scientific papers to decrease time-to-market and reduce costs of goods.

Separate registration required. To learn more and register, visit this link: https://ispe.org/training/course/gamp-basic-principles-2-day-training-course

This classroom or online course has been updated to include the new revised GAMP® 5 Second Edition. This fundamental course introduces participants to regulatory requirements for computerized systems in the pharmaceutical, biotech, or medical device industry and explores tried, tested, and internationally recognized methods of meeting those requirements. GAMP guidance provides a pragmatic and effective framework for achieving computerized systems that are fit for intended use and meet current regulatory requirements, by building upon existing industry good practice in an efficient and effective manner.

Separate registration required. To learn more and register, visit this link: https://ispe.org/training/course/apq-quality-management-maturity-training-course

As the industry faces increasing scrutiny from Health Authorities on the assessment of Quality Management Maturity and Continual Improvement, it is more important than ever to have practical tools and approaches to enhance the effectiveness of the Pharmaceutical Quality System (PQS).

The ISPE Advancing Pharmaceutical Quality (APQ) Program has been developed by industry representatives, for industry use, to provide a practical framework that organizations can use to assess and advance the state of quality within their organization. The APQ program recognizes that the ability to advance the maturity of quality management lies within the industry itself and provides a range of sustainable and practical quality management improvement strategies.

This facility tour is sold out and will not be accepting additional registrants.

On Wednesday, 19 June, Buses will arrive at 8:00 am EDT at the main entrance outside the front of the Westin Copley Place Hotel and depart at exactly 8:30 am. 

Ultragenyx recently opened a state-of-the-art gene therapy manufacturing facility (GTMF) in Bedford, MA. The 110,000ft2 facility was designed for the manufacture of clinical and commercial Pinnacle PCL and HEK platform gene therapy products. The facility is comprised of a drug substance manufacturing suite, a drug product manufacturing suite, central services and support areas, a cGMP warehouse, satellite Quality Control labs, mechanical spaces, and offices. The GTMF was designed with patients in mind, focused on delivering treatments to rare disease patients as quickly as possible. This is demonstrated by the implementation of single-use equipment and utility panels in ballroom-style processing areas for greater flexibility and quick turnaround of the manufacturing areas. Tour attendees can expect to see an operational AAV gene therapy manufacturing facility, from warehouse operations to drug substance and drug product manufacturing areas. They will learn a little about what makes the GTMF facility design unique and some challenges that we faced along the way [COVID-19] to deliver the facility on time and on budget.

Transportation and Lunch are provided! To reserve your spot, be sure to add this to your registration for $75 or stop at the onsite registration desk. Attendance limited to 24. 

Important Notice: No photos permitted.

Separate registration through the chapter required. To learn more and register, visit this link:  https://www.ispeboston.org/events/?eventID=1389 

Join the ISPE Boston Chapter for their 2nd Annual Juneteenth Celebration—an evening where culture, education, and networking converge in a historic setting. Dive deep into the rich narratives and pivotal moments of African American history, guided by a knowledgeable historian who brings the past to life.

Location:
Museum of African American History
46 Joy Street, Boston, MA  02114

Separate registration required. To learn more and register, visit this link: https://ispe.org/training/course/gamp-basic-principles-2-day-training-course

This classroom or online course has been updated to include the new revised GAMP® 5 Second Edition. This fundamental course introduces participants to regulatory requirements for computerized systems in the pharmaceutical, biotech, or medical device industry and explores tried, tested, and internationally recognized methods of meeting those requirements. GAMP guidance provides a pragmatic and effective framework for achieving computerized systems that are fit for intended use and meet current regulatory requirements, by building upon existing industry good practice in an efficient and effective manner.

Separate registration required. To learn more and register, visit this link: https://ispe.org/training/course/apq-quality-management-maturity-training-course

As the industry faces increasing scrutiny from Health Authorities on the assessment of Quality Management Maturity and Continual Improvement, it is more important than ever to have practical tools and approaches to enhance the effectiveness of the Pharmaceutical Quality System (PQS).

The ISPE Advancing Pharmaceutical Quality (APQ) Program has been developed by industry representatives, for industry use, to provide a practical framework that organizations can use to assess and advance the state of quality within their organization. The APQ program recognizes that the ability to advance the maturity of quality management lies within the industry itself and provides a range of sustainable and practical quality management improvement strategies.