Big analytics tools, like multivariate data analysis (MVDA) and design of experiments (DOE), can improve knowledge transfer capture to support technology transfer activities by making process information readily available to R&D, process development, commercial manufacturing, and other departments. In this presentation, Sartorius Data Analytics will present on how SIMCA® and SIMCA®-online can be leveraged to optimize technology transfer activities across the product lifecycle.

As current biological product pipelines become more diverse, product demand and cost pressures are increasing. To meet these demands, manufacturers often move towards process intensification. By making changes to unit operations, the process, or even the type of facility, our industry can identify areas of potential improvement that can increase productivity, reduce timelines, downsize process footprint, lower cost of goods, and/or unlock additional manufacturing flexibility.

Priyanka Gupta, Head of Market Entry Strategy PBT at Sartorius, will define intensified processing needs and will provide solutions using a decision criteria matrix to choose the optimal intensified strategy. The presentation will also shed light on the overall Cost of Goods analysis in selecting upstream and downstream intensified processes. She will show how selecting a specific process influences the overall facility footprint to satisfy throughput demands and how these solutions give multi-product facilities the flexibility to increase annual product turnover by up to 50%.

Covering the question of sustainability, Priyanka will highlight how water usage, energy, and HVAC will impact the overall PMI index concerning the process choices one makes.

Webinar Objectives:

• Obtaining a general overview of upstream and downstream Process Intensification
• Understanding cost implications in selecting various process intensification scenarios
• Discussing sustainability including energy, water and PMI depending on the process selection
• Facility footprint implications when moving towards an intensified process 

Speaker:

Priyanka Gupta
Head of Market Entry Strategy
Segment Marketing Protein-based Therapeutics
Sartorius

Silicones have been successfully formulated as pharmaceutical excipients in dermatological and topical drug delivery forms to improve their efficacy and acceptance by the patients, which are critical requirements in pain drug management.

The aim of this presentation is to promote the results of recent in-vitro studies where the benefits of DuPont™ Liveo™ silicone film former technologies on key parameters of topical forms, such as substantivity and skin permeation are discussed, as well as case studies to demonstrate their impact on the topical skin delivery of established pharmaceutical actives.

Speakers:

Monja Lampert
Product Manager Pharma
Biesterfeld Spezialchemie

Virginie Caprasse
Technical Specialist
DuPont

Harpoon is developing novel T cell engagers, including the TriTAC® and ProTriTAC platforms, that direct T cells to fight cancer and other diseases. Harpoon has four clinical stage TriTAC® drug candidates, with additional compounds in preclinical development. Harpoon’s ProTriTAC platform focuses on T cell engager prodrugs that are designed to be preferentially active in the tumor. In this talk, the unique challenges of expressing sufficient quantities of prodrug in CHO cells and the solutions to these challenges will be presented.

Speaker:

Susan Dana Jones, Ph.D.
Senior Vice President
Product Development
Harpoon Therapeutics

Best Practices for Streamlining Technology Transfer and Manufacturing for Early-Stage Clinical Products Abstract: Harpoon is developing novel T cell engagers, including the TriTAC and ProTriTAC platforms, that direct T cells to fight cancer and other diseases. Harpoon has four clinical stage TriTAC drug candidates, with additional compounds in preclinical development. Harpoon aims to move drug candidates from discovery to clinical development in an efficient and timely manner while working with various CDMOs and CROs to produce both Drug Substance and Drug Product. Lessons learned during technology transfer and manufacturing will be discussed.

Speaker:

Alpana Naresh, Ph.D.
Director CMC Operations
Harpoon Therapeutics

Recently the requirements of virus filtration are changing. For example, the protein concentrations of process solutions are increasing, and the virus filtration step is operated not only with pressurized vessels but more and more with pump-based system. In this 2-part presentation, we discuss about “Advanced Virus Filtration – High Protein Concentrations and Pump-Based Virus Filtration”. 　 Increasing cell culture productivity and improving column chromatography performance in the past several years have resulted in virus filtration feeds with higher protein concentrations. Feed solutions at higher protein concentrations can present some challenges, for example the potential increase in aggregation formation which in turn can affect filterability. However, one main benefit at higher concentration is the reduced filtration surface area needed and therefore the cost, providing that the nanofilter can handle this increase in concentration. Other benefits include reduced processing time and reduced footprint. Planova BioEX exhibits high throughput with high concentration mAbs and the addition of Virosart Max prefilter can further improve filterability by removing aggregates. Viral clearance validation studies are typically performed using small-scale filters operated by applying constant pressure to a feed vessel, while at large-scale it is favored to use pump-based systems utilizing single-use bags, which offer improved footprint and portability. Pump-based filtration processes present more process complexity, more potential for process fluctuations, and more challenges in scaling. We have performed studies to better understand pump-based virus filtration operations using Planova™ filters. We show that Planova 20N filters provide effective viral clearance even in the presence of large fluctuations in transmembrane pressure across all filter sizes and obtain comparable results with that of using constant pressure.

Speaker:

Mathithas Kandasamy
Senior Product Manager
Asahi Kasei Bioprocess Europe

Monoclonal antibody derivatives are widely used in therapeutic and diagnostic applications. Recently, antibody fragments have gained interest with some advantageous properties compared to their parent molecules. Downstream processing of mAb fragments typically starts with capturing by protein L affinity chromatography. In this talk performance parameters of several Protein L chromatography media will be compared and their influence on process economics will be evaluated. Further, we will demonstrate how transferring from a conventional batch mode to continuous affects the productivity of the antibody fragment capturing process.

Speaker:

Jonas Wege
Application Specialist
Tosoh Bioscience GmbH

Titer and process-related impurities levels are critical quality attributes (CQA) in biotherapeutic manufacturing of therapeutic drugs, viral vectors and vaccines. Executing accurate and precise bioassays of different types for large sample sets in downstream analytics can cause workflow bottlenecks. Gyrolab® systems perform automated immunoassays within nanoliter-scale microfluidic structures in a Compact Disk (CD) format, increasing speed while reducing sample and reagent consumption as compared to plate-based ELISA. We present case studies from antibody and viral vector bioproduction runs, showing how titer and multiple types of impurities are being assessed in a walk-away format, reducing hands-on time and increasing productivity in bioprocess analytics.

Speaker:

John Chappel
Director of Application Support EMEA and Asia Pacific
Gyros Protein Technologies

Adeno associated virus (AAV) is the leading vector in the field of gene therapy because of its low toxicity, good overall safety profile, and ability to maintain stable expression for long periods of time. It is therefore crucial to develop a robust and high efficiency platform for its manufacturing.

One of the key challenges in manufacturing viral vectors is to increase the ratio between empty and full capsids. The most efficient way is to design the USP to result in less than 10% of empty capsids. This can be realised by process optimisation using at-line HPLC to allow for analysis of the full and empty capsids ratio during the virus expression.

Fast and reliable in HPLC methods to allow for process optimisation USP and DSP, and assessing the purity of the final product using PATfix system will be presented.

Speaker:

Dr Ivana Petrovic Koshmak
Head of Upstream Process Development
BIA Separations

Buffers are the largest raw material products by type and volume that are used in the production of most modern biopharmaceutical therapies. In-house buffer manufacturing has been long-established due to the in-process volume required of materials, requiring permanent infrastructure for support in many facilities. In addition to the facility footprint required, there are contamination risks that must be mitigated in the buffer preparation process: Any deviation from the standard in these raw materials can require significant investigation, resulting in delays, loss of product, and other, dire effects on the manufacturing process as a whole.

The adoption of hydrated, pre-made liquid buffers and buffer concentrates is an alternative that can benefit biopharmaceutical manufacturers in a number of ways. When combined with technologies like in-line dilution, this method of buffer preparation can help reduce facility footprint, labor hours and overall cost-of-goods especially for multi-batch facilities and small-scale manufacturing. This presentation will give an overview of the challenges in raw material management with a risk/benefit analysis of liquid handling options to overcome manufacturing challenges.

Speaker:

Pranav Vengsarkar
Manager Process Development
Research Biopharma
Avantor

Big analytics tools, like multivariate data analysis (MVDA) and design of experiments (DOE), can improve knowledge transfer capture to support technology transfer activities by making process information readily available to R&D, process development, commercial manufacturing, and other departments. In this presentation, Sartorius Data Analytics will present on how SIMCA® and SIMCA®-online can be leveraged to optimize technology transfer activities across the product lifecycle.

Speaker:

Tiffany McLeod
Product Manager, Data Analytics
Sartorius Stedim Biotech

Biopharmaceuticals are complex in their very nature, and the manufacturing of biopharmaceuticals is an art of its own. Scientific expertise, longstanding experience and out-of-the box thinking are essential, but exceptional project management and consultation at every step along the value chain, including regulatory approval, are vital. Only then can we bring essential therapeutics to patients easily and faster. Learn how Rentschler Biopharma masters these complex challenges, together with clients and strategic partners, while growing strategically in an ever-changing biopharmaceutical landscape.

Speaker:

Federico Pollano
Senior Vice President Business Development
Rentschler Biopharma SE

As thermo-weldable solutions enabling quick connection and disconnection, TPE tubing have been valued for decades in several steps of biopharmaceutical processing. Strong from its heritage built on purity, quality and performance, DuPont™ Liveo™ is introducing a new ISO Class 7 manufactured TPE tubing with improved purity profile, excellent resistance to chemicals, high clarity and low spallation after pumping, as well as ensuring welding compatibility with existing TPE tubing. DuPont™ Liveo™ has conducted a comprehensive comparative test package to substantiate the high purity and high-performance claims for this new product and a comprehensive Qualification Guide in accordance with the industry USP and ISO standards will be available for faster qualification process at our customers’ facilities.

Speaker:

Stéphane Cornelis
TS&D/R&D Medical Devices Expert
DuPont Healthcare

Wacker Biotech is the microbial CDMO – your all-in-one partner of choice for GMP contract manufacturing of therapeutic proteins, vaccines, live microbial products and plasmid DNA. For each of these products Wacker offers outstanding microbial technologies enabling the production at highest quality standards, with boosted yields and reduced cost of goods. For example, Wacker Biotech’s ESETEC® technology, unique E. coli secretion system, allows the controlled release of correctly folded proteins in high yields into the culture broth. The technology has been successfully used for the production of a wide variety of recombinant proteins for clinical use, even those that are difficult to produce, including therapeutic Fabs, scaffolds and single-domain antibodies with yields of up to 14 g/L. With three times lower cost of goods sold (COGS), ESETEC® is the better alternative to mammalian systems for non-glycosylated proteins. Plasmid DNA (pDNA) is nowadays much in demand for nucleic acid-based gene therapies, for novel vaccines or as a starting point for innovative therapeutic agents, including messenger RNA (mRNA) and viral vectors. Wacker Biotech offers more than 20 years of experience and capacities for pDNA non-GMP and GMP manufacturing ranging from small scale up to 500 L stainless steel fermenters. Fermentation, downstream purification and pDNA quality control are part of our proprietary platform. A highly experienced, specialized team provides products and services that meet the highest quality and compliance standards.

Speaker:

Dr. Sebastian Schuck
Head of Business Development
Wacker Biotech GmbH

Join this webcast to learn how KBI approaches CMC development of a leading therapeutic modality, bispecific antibodies. Ulrich will touch on production facility design, CMC strategy, and pitfalls of bispecific development, sharing KBI’s experience working on over 40 bispecific programs.

Speaker:

Ulrich Valley
SVP Operations and Geneva Site Head
KBI Biopharma

Session Description Coming Soon!

Speaker:

Spearking Information Coming Soon!

This presentation will provide an overview of several case studies focusing on dynamics and interactions with CMOs when delivering technical programs and routine manufacturing. The presentation will explore best practices and lessons learned and provide recommendations for successful execution of technical programs, where both parties have ownership of the process.

Speaker:

Derek Lonergan
Director of Manufacturing Sciences
Jazz Pharmaceuticals

Adeno-associated virus (AAV) has become the workhorse of gene therapy over recent years, leaning extensively on the introduction of purification platforms and capture steps involving immunoaffinity resins such as POROS™ Captureselect™ AAVX. Nevertheless, enrichment of full capsids remains a challenge and a polishing step can only be developed with chromatographic tools offering unique properties like the POROS™ HQ50 does.

In order to support the pre-clinical studies of an AAV gene therapy product, hear a case study from KU Leuven, who has developed a 2-step chromatographic process for the purification of AAV vectors of different serotypes. POROS™ Captureselect™ AAVX and POROS™ 50 HQ were tested for capture and polishing, respectively, of AAV2, AAV9 and a novel engineered capsid to achieve vectors with high purity and significant empty:full ratio improvement.

Speakers:

Nicolas Laroudie
Senior Field Application Specialist Purification
Thermo Fisher Scientific

Duy Tien (Ryan)
Research associate
Laboratory of Viral Cell Biology and Therapeutics

Viral vectors are being increasingly used for gene therapy. Adeno Associated Virus (AAV) is fast becoming the leading viral vector that is widely used in the development of gene therapy. Different AAV production platforms can be used and here, we present results from process development of AAV2 and AAV5 production by using triple plasmid transient transfection using polyethylenimine (PEI) in HEK293T suspension cells. We used Design of Experiment (DoE) strategy to be able to optimize and evaluate a number of different parameters in small scale (shake flask). Key for a successful upstream process is high number of viral genome and full capsids in the harvest material. Furthermore, we also present a scalable, robust and reproducible AAV production, that was developed from small scale shake flask production up to 25L in different single-use bioreactor systems.

Speaker:

Ann-Christin Magnusson
Staff Research Engineer
Protein and Viral Production R&D
Cytiva

Adeno-associated virus (AAV) is often the vector of choice for gene therapies; in fact, AAV represents around 37% of the current advanced therapies market. And when it comes to manufacturing AAV for in vivo gene therapies, developers must pursue triplicate goals of quality, quantity, and speed. At OXGENE and WuXi ATU, our ambition is to revolutionize AAV manufacture in two ways. First, by improving existing transient expression systems and manufacturing platforms. To this end, we present the quality, titer, and regulatory advantages of OXGENE’s AAV plasmid system, the key quality considerations of WuXi Advanced Therapy (WuXi ATU)’s plasmid manufacturing processes, including a real-world example of accelerated manufacture and delivery timelines, and finally demonstrate the performance of the AAV plasmid system at scale in WuXi ATU’s AAV suspension platform, standardization and optimization of which allows for consistently high results and accelerated time to market. Secondly, we implement wholesale innovations in AAV manufacture to overcome the key industry challenges of scalability, process robustness and cost of goods. Here, we describe how rethinking AAV manufacture ‘from the ground up’, allowed OXGENE™ to manipulate AAV’s natural relationship with adenovirus to address these challenges with their novel TESSA™ technology. TESSA™ vectors, like wild-type adenovirus, provide high quality help for AAV replication, but unlike the wildtype virus are unable to produce adenoviral structural proteins, thereby reducing adenoviral contamination by 99.9999% in a manufacturing run. Integration of the AAV rep and cap genes into the TESSA™ vector means that everything required for AAV production, except the AAV genome, can be provided in a single viral vector. Meanwhile, the AAV genome can either be encoded within a second TESSA vector, in a plasmid, or within an AAV particle itself. Once this first AAV seed stock has been produced, co-infecting cells with this AAV alongside another TESSA vector can further amplify the AAV in a simple, reproducible and scalable manner, removing the reliance on expensive and limiting plasmids for AAV manufacture.

Speaker:

Dr Ryan Cawood
CSO OXGENE
WuXi Advanced Therapies

In this session, you will discover the different technologies and solutions available for viral production and the new approaches in bioprocessing that can lead to sustainable gene therapy while reducing development time.

Speaker:

Hanna Lesch
Chief Technology Officer
Exothera

Session Description Coming Soon!

Speaker:

Tom Wall
Global Planning & Logistics Lead
Spark Therapeutics

Scalability and affordability are two of the main prominent challenges for the AAV gene therapy supply. To meet the need for increased viral vector manufacturing, we propose to establish standardized AAV viral vector and plasmid manufacturing and analytics to ensure consistency of the productivity and to accelerate the release of GMP materials. Transfection efficiency, selection of producer cells, upstream production and downstream purification by chromatography as well as packaging plasmid manufacturing will be discussed. In addition, the newly launched AAV reference standards for AAV testing and analytics and their application for the standardized AAV testing will be overviewed.

Speakers:

Jeffrey Hung, Ph.D.
General Manager
Vigene Biosciences, a Charles River Company

Tony Hitchcock
Technical Director at Cobra Biologics
Charles River Company

Viral safety and risks for cell and gene therapy: how to improve pathogen safety of cell and gene therapy products.

Speaker:

Sebastian B. Teitz, Ph.D.
Cologne Technical Center
Asahi Kasei Bioprocess Europe

Cell and Gene Therapies continue to offer exciting avenues as curative therapies for unmet patient needs in the ultra-rare, rare, and beyond rare clinical indications. To serve these unmet needs, cell and gene therapy companies are developing multiple products that are expected to rapidly advance from pre-clinical stages to clinical trials.

Although development of every product may present a unique set of challenges, leveraging a manufacturing platform can help overcome specific barriers to the clinic. FUJIFILM Diosynth Biotechnologies’ (FDB) AAV manufacturing platform offers clients access to an end-to-end service, including a fully characterized, suspension adapted, clonal HEK293 cGMP cell line, research and cGMP-grade Rep/Cap, Helper, and GOI (backbone) plasmids, accelerating process development, in addition to a comprehensive array of analytical methods to support phase-appropriate product characterization and batch release testing. FDB’s AAV platform and process development & manufacturing approaches to other viral vectors will be discussed in this presentation.

Speaker:

Vishruth Gowda PhD
Senior Scientist in the Process Development Team
FUJIFILM Diosynth Biotechnologies

Some side effects of the vaccination might be attributed to the impurities in the vaccines. The vaccines should therefore be as pure as possible. Constant improvement of the impurity detection methods followed by enhancement of the vaccine purity and is therefore necessitated. In this work we present novel adenoviral vectors purification manufacturing process based on monolith chromatography supported by fast and sensitive HPLC analytics. The novel industrial process secures better purity at higher yields. The robustness of the process was verified using different upstream materials. Last but not least, higher capacity of the columns used in this process overcomes the raw material supply bottlenecks.

Speaker:

Ales Strancar
BIA Separations
Managing Director
Sartorius Company

Session Description Coming Soon!

Speaker:

Stefano Menegatti
Associate Professor
North Carolina State University

The fast-growing gene therapy industry is in pressing need for fast and reliable analytical methods to accelerate bioprocess development and manufacturing of viral vectors. In this presentation you will hear how the MSAT analytical development team at MeiraGTx, a clinical-stage gene therapy company with six ongoing programs in clinical development, streamlined bioprocess analytics of adeno-associated viral vectors (AAV) by developing and implementing a panel of titer and process impurities assays using the automated nanoliter-scale Gyrolab® immunoassay technology. The assays are now being transferred to GMP regulated environment for product release testing.

Speaker:

James Norton
MSAT Analytical Development Lead
MeiraGTx

Lentiviruses and gammaretroviruses are the most popular vectors used in gene and cell therapies with more than 300 clinical trials currently underway in Phase II and Phase III. Quantifying quality attributes of these retroviral vectors is of increasing importance as they enter into the late development stages. In this webinar we demonstrate the use of two analytical techniques, batch dynamic light scattering (DLS) for rapid, low-resolution measurements and multi-angle light scattering in combination with field-flow fractionation (FFF-MALS) for extended characterization of viral vectors. We will discuss methods for evaluation of the size distribution and physical titer, impurity profiling, in-depth assessment of aggregation, and lot-to-lot variability.

Key Learning Objectives:

• How DLS and MALS determine particle size and concentration
• How high-throughput DLS rapidly screens processes and formulations for titer, purity, aggregation and stability
• How FFF- and SEC-MALS are used to compare different viral vector lots and purification protocols, assess stability and quantify impurities
• How DLS and FFF-MALS can be used in viral vector production and lot release assays

Speaker:

Anatolii Purchel
Application Scientist
Wyatt Technology

Bio-Rad has over 60 years of experience supporting life science research and clinical diagnostics. Our innovative products are available as standalone solutions or combine into your processes that are optimized to minimize downtime and to provide the right answer the first time. Bio-Rad is dedicated to offering tools and services that empower the development, analysis, and manufacturing of cutting-edge gene therapies.

Speaker:

Andrew M. Prantner
Field Marketing Specialist
Bio-Rad Laboratories