Day 1 - July 8, 2020: Emerging Therapies
Manufacturing CRISPR RNPs for Clinical Applications
11:45 AM ET — Shawn Shafer, Genome Editing Platform Lead, Aldevron
Aldevron specializes in manufacturing Cas9 proteins, as well as custom protein development and manufacturing services. Although many vendors offer research grade CRISPR/Cas nucleases, these are not applicable to clinical programs due to construct design and quality grade. While Aldevron is the only manufacturer to offer GMP-grade Cas9 proteins and select vendors offer GMP-grade synthetic single guide RNAs (sgRNAs), there are currently no options for complexing the two as a ribonucleoprotein (RNP) in a GMP-certified process. In an effort to address this gap in manufacturing for therapeutic genome editing, Aldevron has created a method to complex, QC, and characterize RNP complexes under GMP conditions. We will review our findings in this presentation.
Technology Integrators in Cell and Gene Therapy — Increased Efficiency and Standardization
12:15 PM ET — Julien Meissonnier, VP & CSO, Catalent Biologics
The cell and gene therapy markets are seeing growth both in the use of allogeneic and autologous cell therapies as well as viral vectors for gene therapy and ex-vivo transduction of the cell therapies. Both require investments in disruptive technologies to enhance safety and activity as well as reduce production costs. Accelerated clinical trials for advanced therapeutics is on the rise and with many having fast track status designation, it is increasingly critical for innovators to have efficient and scalable production processes. As more programs shift to late-stage clinical development, optimized methodologies like Manufacturing by Design (MbD) and efficient and optimized manufacturing processing work to eliminate industrialization challenges in order to achieve commercial-ready processes. This process excellence combined with technical expertise across a variety of cell types, including emerging iPSC therapies, allow custom development and manufacturing organizations to be well-positioned to support this fast-moving industry.
Catalent’s deep and expansive cell and gene therapy global network is dedicated to evaluating new innovative technologies and process platforms across the entire scale-up and production process to meet the aggressive timelines of the clinical landscape. Focus areas such as improved upstream and downstream processes, enhanced bioanalytic characterization, high-capacity fill and finish platforms optimized for cell therapy batch efficiency, and integrated supply chains help to alleviate manufacturing bottlenecks thereby achieving more optimized and reproducible manufacturing processes.
Development of an Advanced Gene Therapy Platform
12:45 PM ET — Steve Pincus, PhD, Head of Science and Innovation FUJIFILM Diosynth Biotechnologies
The success of gene therapies has resulted in a surge in the number of companies that are entering this arena. Many of these companies seek advice from a CDMO on the system they should use to produce their product candidate. Fujifilm Diosynth Biotechnologies is developing a platform for AAV production to be offered to companies seeking help. Our platform will consist of 1) a suspension HEK293 cell line 2) GMP grade adenovirus helper 3) GMP rep/cap plasmid representing several AAV serotypes 4) a GOI plasmid that a candidate can be inserted in and 5) a selection of tools for in-process and release testing.
Meeting Regulatory Requirements for Cell and Gene Therapy Manufacturing
1:15 PM ET — Karen Magers, Head of Regulatory Affairs, Cell and Gene Technologies, and Rajesh Thangapazham, Head of Regulatory Strategy, Cell and Gene Technologies, Lonza Pharma and Biotech
Lonza is a global leader and well known contract development and manufacturing organization in the field of life sciences. Lonza was founded in 1897 and has more than 50 manufacturing sites worldwide in more than 30 countries employing close to 15,000 employees. Lonza’s mission is to industrialize manufacturing from concept to patient and therefore provides cutting edge solutions during development and manufacturing. Lonza’s vision for cell and gene therapy is to deliver the medicines of tomorrow, today. Lonza’s cell and gene technologies business unit is expanding its global foot print and increasing its manufacturing capacity through innovating in key areas such as process and analytical development and manufacturing. This webinar is intended to provide the audience with an understanding on the following topics;
- Existing regulatory landscape, guidance and regulations applicable to cell and gene therapy products
- Hot topics related to manufacturing facilities to be considered during regulatory filings
- Segregation measures and controls pivotal for a multiproduct facility
- Lonza’s regualtory capability and offering such as master files that sponsor’s can utilize to accelerate their regulatory timeline
Accelerating Clinical Development and Commercialization
with a Lentiviral Vector Platform Manufacturing Process for Cell Therapy
1:45 PM ET — Kevin Beck, Senior Manager, Miltenyi Biotec
Attendees will learn about:
- Enhancing predictability with lentiviral vector production platforms
- Use of serum-free suspension for large-scale lentiviral vector production
- The importance of robust and reproducible results in commercial viral vector production
- Streamlining project timelines while expediting the clinical phase and moving rapidly to the commercial phase.
- Ensuring effective process validation to support commercialization of cell therapies
Solutions for Rapid Development and Manufacturing Responses
to COVID-19 and Other Public Health Threats
2:15 PM ET — Richard W. Welch, PhD, Vice President Development Services, Emergent BioSolutions
The novel coronavirus SARS-CoV-2 and its related disease (COVID-19) have impacted many aspects of the daily lives of people around the globe, stressing healthcare and social systems and putting biotechnology at the forefront of this pandemic. Critical to the response to the pandemic is the ability to rapidly develop, scale, and manufacture novel, and effective, vaccines and therapeutics. Leveraging the lessons learned from the successful and ongoing production of early phase clinical trial material, Emergent is partnering with commercial and government teams to scale up and manufacture material capable of supporting late-stage clinical trials, as well as supplying material to support US and global efforts to fight this pandemic. Dr. Rick Welch will share lessons learned from developing drugs to treat people during epidemics and pandemics, such as COVID-19, and best practices for success in this unprecedented time.
Innovative Closed Process CAR-T Cell Therapy Platform
to Streamline Approach for Manufacturing with Great Predictability
2:45 PM ET — Tatiana Golovina, PhD, Senior Director Cell Therapy Process Development, WuXi Advanced Therapies
The presentation will showcase how to accelerate timeline and progress to market for CAR-T production that includes: in-stock raw materials with established batch records; regulatory and technical expertise; process and analytical development; full in-process and release testing; robust quality control and quality assurance oversight; and facilities that offer the capacity and flexibility to meet client timeline needs. The platform also offer a closed process that allow flexibility of clinical manufacturing in a shared pre-equipped suite model that will significantly increase efficiency while mitigating cost.
Economics of Stainless Steel and Single-Use Systems
3:15 PM ET — Justin Carbungco, Associate Director, Small Scale Manufacturing, Samsung Biologics
With the development of single-use systems for biomanufacturing, the lingering question is “Which system is better? Single-use or stainless steel?” For anyone that has looked into this topic, the answer is not black and white. In this presentation, we will focus on the cost comparisons for Stainless Steel and Single-Use Systems and some considerations for companies that are looking into clinical and contract manufacturing.
From Target to Hit: The WuXi HitS Platform
3:45 PM ET — Alex Satz, Senior Director DEL Strategy and Operations, WuXi AppTec
WuXi AppTec has recently launched the HitS (Hit Success) business unit to better serve early stage drug discovery. WuXi’s goal is to provide more and better hit molecules against targets of interest, and to help set the stage for follow-up Med-Chem optimization. We possess expertise in the production of all protein target classes, including solubilized membrane proteins and challenging protein complexes. A core strength of our HitS business unit is the WuXi DNA-Encoded Library platform (WuXi DEL). With a team of 40 library chemists and 6000 proprietary scaffolds, we have constructed more than 200 DEL libraries displaying >90 billion different small-molecule structures. Complementing our WuXi DEL approach, we have also built a “rule-of-three” compliant fragment screening platform which can be interrogated by MST, SPR, NMR, or X-ray crystallography. Hit confirmation may be accomplished with a number of different biophysical assays, and our team has particular expertise in label-free MST. Lastly, generation of ligand bound x-ray crystal structures (or Cryo-EM) facilitates structure-based medicinal chemistry optimization.
Get to IND Faster: Accelerated and High-Performance Cell Line Development
4:15 PM ET — John Gill, Director of Cell Line Development, Samsung Biologics
Expediting development timeline is directly related to obtaining IND clearance and starting phase 1 study faster. This presentation describes how you can get to IND faster through our accelerated & high performance proprietary cell line. We will present our 3 month cell line development timeline and high quality cell line with outstanding productivity, the Faster & Better approach which can be applicable to your molecule's development path to IND.
Day 2 - July 9, 2020: Emerging Techniques, Technologies, and Strategies
The Role of New Technologies in Enabling Cell and Gene Therapies
10:00 AM ET — Chair: Doug Miller, Managing Director, BPTG
Daniella Kranjac, Co-Founder and Managing Director, Dynamk Capital LLC
Tim Kelly, President of Manufacturing, Asklepios BioPharmaceutical, Inc
Andy Ramelmeier, Executive Vice President of Technical Operations, Sangamo Therapeutics, Inc.
Ran Zheng, Chief Technical Officer, Orchard Therapeutics
A panel of experts will discuss product development and manufacturing challenges for cell and gene therapies, and technologies that are being developed to address these challenges. Panelists will include both end users that can discuss the needs for new technologies as well as technology providers.
The panel will discuss technologies for:
- Scalable and cost efficient production of viral vectors for gene therapy
- Non-viral approaches for gene therapy
- Scaling up to meet demand for autologous cell therapies
Road to Commercialization: A CDMO Perspective
11:00 AM ET — Richard Richieri, Chief Operations Officer, Avid Bioservices
The dynamic of biopharmaceutical industries to bring innovative product to the patients has evolved over the years. Avid Bioservices, as a CDMO with production scales including multiple 2kL SUBs, is an integral part of the supply chain to bring lifesaving drugs to commercialization. Avid has successfully completed 10 late stage cell culture based drug substance manufacturing campaigns for numerous clients. The presentation will focus on the lessons learned from various critical activities (such as process characterization) that supports the success of the “at-scale” process validation program which in turn enables our clients to submit for market approval.
Plasmid Manufacturing Excellence: The Foundation for Multiple New Modalities
11:30 AM ET — Mario Kraft, Head of Process Development, AGC Biologics
Gene and cell therapy as well as other new modalities, like RNA treatments and DNA vaccinations, are rapidly evolving and highly promising areas of drug development in the last decade. Increasingly, plasmid DNA (pDNA) is the starting material for these modalities. A highly reliable supply of non-GMP and cGMP grade pDNA is a crucial element in the future of bio-pharmaceutical development. AGC Biologics has established a comprehensive toolbox of host cells, fermentation procedures, alkaline lysis steps as well as purification platforms that can be optimally adapted to each specific customer need. With a 10-year proven track record in commercial plasmid production and a highly customizable approach for all plasmid needs, AGC Biologics is a center of excellence for high quality pDNA.
GPEx Boost: A Novel Approach for High-Expressing CHO Cell Line Engineering
12:00 PM ET — Gregory Bleck, PhD, Global Head of R&D, Biologics Catalent
Pharma companies are always examining ways to increase efficiencies and reduce cost of drug development. While cost of goods sold (COGS) can be high for small molecule drugs, biologic development can be even more costly due to the complex nature of the molecules. One way to reduce COGS during biologic development and manufacture is to increase the productivity of the cell line that is expressing the protein, typically a monoclonal antibody. Companies have multiple levers they can pull to attempt to increase productivity, but the cell line development process plays an important role in defining the upper limits of production.
GPEx® cell line development technology is a proven technology that generates highly stable, high titer production cell lines. Recently, GPEx technology and a glutamine synthase knock-out CHO cell line were combined in a unique way to create GPEx Boost. The new technology results in higher specific productivities, higher titers and improved cell growth characteristics compared to GPEx for most protein products. This talk will describe GPEx Boost technology in more detail and discuss additional case study comparisons.
CRISPR Library Screens for Oncology Target Discovery at WuXi AppTec
12:30 PM ET — Yong Cang, PhD, Scientific Advisor, Oncology and Immunology Unit, WuXi AppTec and Professor, Cancer Cell Biology, ShanghaiTech University (Shanghai)
Tumors acquire genetic mutations to escape immune elimination to grow and metastasize. This webinar will share case studies highlighting application of customized CRISPR/Cas9 libraries to screen for genes that control cancer cell sensitivity to target therapeutic agents and T cell-mediated elimination in vitro and in vivo. These genes are potential targets for therapeutic intervention.
Attend to discover:
- Synthetic lethal screens for mutations with chemical inhibitors or immune checkpoint blockers
- Application of in vivo CRISPR screen for metastatic drivers in cancer cells against host immunity
- Novel approaches for oncology and onco-immunology target discovery
An End-to-End Integrated Solutions Approach to Commercial Viral Vector Manufacturing
1:00 PM ET — Jeremy Rautenbach, Global Product Manager Biotech Integrated Solutions, Pall Biotech
With several recent FDA approvals and a strong drug pipeline, gene therapy is coming of age. With this comes the requirement to ensure that there are robust manufacturing processes in place in order to scale with demand and to make these therapies readily accessible to those who need them. However, current manufacturing processes for gene therapies have often been developed with limited scalability in mind and large shifts in technology have to take place to enable industrialization. This also has to be done while keeping costs in mind. In this talk we will discuss the importance of establishing robust, scalable manufacturing operations and demonstrate its impact on future manufacturability of gene therapies.
New Business and Funding Models To Accelerate the Development of Life-Preserving Therapies
1:30 PM ET — Jonathan Freeman, PhD, Founder & COO, Anthos Therapeutics and Senior Advisor Blackstone Life Sciences; and Abdelaziz Toumi, PhD, Head of Ibex™ Design & Develop Customer Solutions, Lonza Pharma & Biotech
In this presentation, Anthos Therapeutics & Lonza Pharma & Biotech will explore how innovative business models and strong partnerships can help accelerate the clinical development process to bring vital, but underfunded, drugs to patients. The partners will discuss how their program across the development and manufacturing of MAA868, an antibody directed at Factor XI and Xia, is designed to deliver quality with agility, speed and an investor focus.
A Holistic and Integrated CMC Development Approach: Committing to Quality, Reliability and Speed
2:00 PM ET — Jerry Yang, PhD, SVP and General Manager, HJB International
Securing Your Manufacturing Capacity During the Pandemic
2:30 PM ET — Richard Lee, Head of Drug Product Business Unit, Samsung Biologics
As COVID-19 pandemic has spread globally, market forecast has become completely unpredictable. The capacity modeling for manufacturing drug substance and drug product will become the one of the most pivotal variables in the coming years. Rise and fall of pharmaceutical companies will depend not only on types of treatments and vaccine development but on product delivery and making them available.
Leveraging Infrastructure Investments & Innovation to Accelerate Biologics Development
2:45 PM ET — Paul Jorjorian, Vice President and General Manager – Biologics, Thermo Fisher Scientific
With a growing pipeline of both traditional and regenerative biopharmaceutical drugs and increasing cadence of approvals for novel biologics and biosimilars, there is a need now more than ever for readily available solutions that will translate the promise of a remarkable discovery into a successful therapy. This webinar will showcase how Thermo Fisher Scientific is leveraging infrastructure investments and innovation to deliver platform and bespoke solutions meant to accelerate biologics development.
Protein or Not? Advanced High-Throughput Aggregate Analysis with the Aura™
In protein-based formulations, distinguishing aggregated API from other particle types is important for understanding the root cause of instability. Until now, existing methods have been either unreliable or too cumbersome to use in many workflows. Here we introduce the Aura™ 96-well low-volume aggregate and particle imaging system, which can rapidly size, count, and characterize particles and identify them as proteins, non-proteins, hydrophobic, or other types of molecules.
Presenter: Bernardo Cordovez, Founder and Chief Scientific Officer, Halo Labs
The Evolving Role of the Outsourcing Manufacturer: Why CDMO Is the New Normal
Throughout the pharma industry, third-party manufacturing partners have become more than simple contract supplier. No more is this evident than in the biologics manufacturing space as outsourcing firms respond to the needs of the end-user and increase their offerings and technologies to provide progressively end-to-end services. This panel looks at how ‘CMOs’ have evolved into ‘CDMOs’ in the biomanufacturing space and how the importance of offering developmental and auxiliary services has shaped the outsourcing landscape.
Chair: Dan Stanton, BioProcess Insider
Panelist: Magnus Schroeder, PhD, VP Process Development, Avid Bioservices
Panelist: Daniel Slone, VP & Head of Downstream Manufacturing, Samsung Biologics
Having In-House Manufacturing Capabilities Gives Cell and Gene Therapy Developers a Competitive Advantage — Discuss
As cell and gene therapies progress through the clinic, developers are looking to secure manufacturing capabilities. Within the CDMO space, unprecedented demand for services is driving facility expansions and a whole new M&A focus. But a growing number of end-users are opting to move away from outsourcing partners and invest in their own cell and gene networks. Pfizer is building a $500 million NC plant, Novartis bought its CAR-T CDMO CellForCure, and Astellas acquired Audentes for $3 billion, citing the addition of an AAV facility as motivation. So will having inhouse capabilities provide an advantage as the sector matures? This panel hopes to find out.
Chair: Dan Stanton, BioProcess Insider
Panelist: Joe Rininger, Latham Biopharm Group
Panelist: Felix Hsu, Chief Commercial and Chief Business Officer,
WuXi Advanced Therapies
BioProcess Insider Interviews: Julien Meissonnier, Vice President and Chief Scientific Officer, Catalent Biologics
Julien Meissonnier, Vice President & Chief Scientific Officer at Catalent, sits down with Bioprocess Insider editor Dan Stanton to talk about his firm’s recent jump into the cell and gene therapy space, how new modalities are affecting the CDMO sector, and how industry is increasingly turning to innovative third-parties to tackle everything from advanced therapy complexity to COVID-19 therapeutics.
BioProcess Insider Interviews: Kevin Sharp,
Director Business Development, Global Business Development Center, Samsung Biologics
Kevin Sharp, director of Business Development, Global Business Development Center at Samsung Biologics, sat down with Bioprocess Insider editor Dan Stanton to discuss his firm’s response to the COVID-19 pandemic, the evolving role of the outsourcing manufacturing firm, and their own plans to expand going forward.
BioProcess Insider Interviews: Felix Hsu, Chief Commercial and Chief Business Officer, WuXi Advanced Therapies
Felix Hsu, Chief Commercial and Chief Business Officer, spoke with Bioprocess Insider editor Dan Stanton to talk about the technical struggles in developing and manufacturing advanced therapies and how the current emergence of process technologies available to support speed-to-market will evolve into fewer, more efficient platform as the sector matures.
BioProcess Insider Interviews:
Steve Burton, Chief Executive Officer, Astrea Bioseparations
CEO Steve Burton talks to Bioprocess Insider editor Dan Stanton about Astrea Bioseparation’s streamlined strategy following its recent acquisition by KKR Gamma Biosciences. He also explains why, 30 years on, antibody makers continue to demand solutions to the bottlenecks in the downstream process, and how chemical and small molecule-based adsorbents can offer a more customized and cost-effective alternative to recombinant ligands and the ‘gold standard’ Protein A.
BioProcess Insider Interviews: Timothy Compton,
Chief Commercial Officer, Avid Bioservices
Timothy Compton, Chief Commercial Officer at Avid Bioservices sits down with Bioprocess Insider editor Dan Stanton to discuss how the veteran biologics manufacturer has embraced life as a pureplay CDMO, and how facility and offering expansions will place it in good stead to service the high demand from the biopharma industry.