Day 1 - July 21, 2021: Emerging Therapies

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BioProcess Insider Interview: Sam MacHour, Samsung Biologics

10:30 AM ET —

Sam MacHour, Senior Vice President & Chief Quality Officer at Samsung Biologics, spoke with BioProcess Insider editor Dan Stanton about the rapid response by both his company and the wider CDMO sector to biopharma's COVID-19 challenges. He also explained how Samsung Biologics has emerged as one of the largest CDMOs in just 10 years, and discussed future plans for the company in the CDMO space and beyond.

Enabling Pharma and Biotech Innovation Through an Open-Access Platform:
Targeted Protein Degradation

11:00 AM ET — Dave Madge, Vice President, Research Service Division, WuXi AppTec

This presentation will focus on the discovery and characterization of bifunctional molecules designed to leverage the ubiquitination pathway for degradation of a target protein. We will discuss strategies for the discovery of ligands for both the target protein and the ligase complex, determination of the kinetics of their interactions in binary and tertiary complexes, and the application of structural biology to design new ligands. We will also present methods for determining functional effects of these molecules, including efficacy in native cells, and discuss future prospects for application of alternative ligases.

Transient Viral Vector Solution

11:30 AM ET — Jason King, Business Development Manager, OXGENE, a WuXi Advanced Therapies Company

Although viral vector manufacturing for late stage clinical and commercial phases ultimately focusses of scalable solutions, all gene therapy projects begin with transient systems. The flexibility of these plasmid-based systems allows optimisation of the therapeutic transgene, the promoter driving its expression, various measures to reduce host immune responses and the targeting of the vector towards specific cell populations.

OXGENE has developed transient vector manufacturing systems for both adeno-associated and lentiviral vectors using its know-how in synthetic biology, cell line engineering and viral vector biomanufacturing. Whilst both systems generate high vector titres, the AAV system also produces capsids with a 30-70 % full rate, making the particles more effective. The LV system benefits from reduced levels of homology between viral vector sequences on different plasmids, making it less likely that replication competent virus is formed. Furthermore, the LV system dovetails nicely with OXGENE’s lentiviral packaging and producer cell lines so that users can transition to these scalable systems when the time is right. OXGENE’s AAV and LV plasmids are available, license-free from OXGENE.

Since joining forces in March 2021, Oxford (UK) based OXGENE and Philadelphia based WuXi Advanced Therapies are now using the same AAV and LV vector production systems making transitioning from preclinical to clinical grade manufacturing an even smoother process.

Applying the Allogeneic Process to Overcome Challenges in Cell Therapy

12:00 PM ET — Evan Zynda, Staff Scientist, Thermo Fisher Scientific

The commercial launch of approved cell therapies demonstrated to the industry and world alike, the transformative impact they can have on a patient’s life and the ability to revolutionize the treatment of human disease. As the industry is now turning to large-scale allogeneic therapies, which can impact a broader patient population, there is a focus on retaining more early memory T cells during ex vivo manufacturing. Examples of the advanced capabilities that will help us accomplish these goals include our state-of-the-art metabolic and proteomic understanding, robust T cell activation technologies and expansion media, and our deep understanding of how the workflow affects the cellular product. In this session, you will hear about the advantages of using allogeneic workflows and how to fine-tuning the manufacturing process can improve therapeutic performance.

TESSA Platform Delivers High-Quality, High-Titre, Multi Serotype rAAV Stocks

12:30 PM ET — Maria Patricio, Lead Scientist, Viral Design & Discovery, OXGENE, a WuXi Advanced Therapies Company

A significant challenge for Adeno-associated virus (AAV) -mediated gene transfer has been the capacity to produce recombinant AAV vectors of high quality, at high-titre and at large scale. OXGENE has addressed these challenges by developing a proprietary Tetracycline-Enabled Self-Silencing Adenovirus (TESSA) system that simultaneously provides Adenovirus (Ad) helper functions and encoded AAV Cap/Rep. When cells carrying an AAV ITR-flanked transducing genome are infected with TESSA-CapRep high-titre rAAV stocks are produced that are essentially free of contaminating TESSA vector.

We have now examined the ability of TESSA to support production of a diverse range of AAV serotypes. The Cap coding region of AAV serotypes 1-9 were independently engineered into the E1 region of TESSA and the quality, physical and infectious titre of the rAAV produced by this system were characterised. For all rAAV serotypes, physical titres and infectious titres were invariably greater than that observed for conventional plasmid-based rAAV systems. For these serotypes, the ratio of full to empty particles is greatly improved. Moreover, for some AAV serotypes, the TESSA system delivered infectious titres 1000-fold greater than the plasmid-based helper-free systems.

Importantly, TESSA-derived rAAV serotypes were competent for reporter gene and therapeutic gene transfer into a multiplicity of human cell types in vitro and a variety of model animal tissues in vivo. OXGENE have therefore developed a versatile system that can be applied to a range of naturally occurring AAV serotypes, and also to experimentally engineered AAV capsids with novel tissue targeting phenotypes. This system provides unique opportunities for robust high-quality scale up of rAAV manufacture that will reduce costs and facilitate rapid progression to clinical application of novel rAAV-based gene therapies.

Comprehensive Target Discovery and Validation at HDB-WuXi AppTec

1:00 PM ET — Shelly (Fang) Zhang, PhD, Director II, Exploration Biology Team Leader, HD Biosciencs - WuXi AppTec

Novel target discovery and validation are the most crucial steps and prerequisite for successful drug development. I will present our evolving genetic manipulation platforms propelled by various new technologies, and extension to the challenging field of immunology, as well as expansion and utilization in multiple new modalities.

• Advanced genetic manipulation platforms propelled by CRISPR and other new technologies
• Gene editing in various human and mouse primary immune cells
• From new tech to new modalities (PROTAC/TPD, TCR-T/CAR-T, AOC/ASO etc)

A Behind-The-Scenes Look at Lykan Bioscience’s State-of-the-Art,
Purpose-Built Cell Therapy Facility

1:30 PM ET — Patrick Lucy, President & CEO, Lykan Biosciences

With decades of experience in biopharmaceutical production and, more specifically, in cell therapy manufacturing, the team at Lykan Bioscience has leveraged their expertise to develop a world class, purpose-built, multi-product cell therapy manufacturing facility. Advanced and integrated software systems enable real time monitoring of the process and product, complete with electronic batch records that will support real time testing and release of product from each of the eight clinical and commercial cGMP manufacturing suites. Strategically located in the proximity of three international airports provides Lykan with many options for the efficient receipt of patient material and delivery of product back to the patient. In this video, Patrick Lucy, Lykan’s President and Chief Executive Officer, takes you behind the scenes of Lykan’s state-of-the-art, purpose-built cell therapy facility located in Hopkinton, MA. Learn more about the process development and manufacturing services that Lykan Bioscience offers and how Lykan is partnering with leading biotechnology companies to develop, manufacture and deliver life-saving cell-based therapies to patients worldwide.

 

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Day 2 - July 22, 2021: Emerging Techniques, Technologies, and Strategies

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BioProcess Insider Interview - Marc Hummersone, Astrea Bioseparations

10:30 AM ET —

Marc Hummersone, Senior Director of Research & Development at Astrea Bioseparations, sat down (virtually) with BioProcess Insider editor Dan Stanton to discuss his company’s past, present and future. The firm has 30 years of experience in bioprocessing but with the backing of KKR and a growing number of sister companies under the Gamma Biosciences portfolio, Astrea is looking to be the market leader in the purification solutions space across all modalities.

Tailoring the Cell Line Development Process to the Specific Molecule and Program Goals

11:00 AM ET — Gregory Bleck, Vice President of Research & Development, Catalent Biologics

Speed and efficiency remain major drivers for developers bringing their protein-based biologics to first in human studies. The right cell line development approach can help streamline the path to clinic for both standard monoclonal antibodies and more complex modalities such as bispecifics. During this presentation, we share the latest data on how the GPEx® suite of technologies enables high titers, short timelines and flexibility in cell line development for mammalian-expressed proteins.

CGMP Manufacturing Facility Design and Evolution

11:30 AM ET — Mike Alston Jr., Director, Project Engineering, and Will Leonardi, PhD, Senior Project Manager, Avid Bioservices

The dynamics of the biopharmaceutical industries have evolved over the years to ensure the innovative and safe manufacturing of clients products. As a Contract Development Manufacturing Organization (CDMO), Avid Bioservices has a long standing track record of manufacturing safe commercial products for its clients. To best serve the growing industry needs, Avid has begun expansion of its facilities. This expansion will result in more than a 2 fold increase of bioreactor capacity for Avid. This presentation will discuss lessons learned and key factors to consider when building new single use facility.

SYMPHONX: A Multifunctional Single-Use Purification System for Connected and Integrated Continuous Processing

12:00 PM ET — Charles Heise, PhD, Senior Staff Scientist, Bioprocessing Strategy & Development, FujiFilm Diosynth Biotechnology

The downstream purification system SymphonXTM uses a disposable, generic flow path to operate multiple downstream process unit operations (e.g. Chromatography, Tangential Flow Filtration (TFF), Single-Pass TFF, Viral Inactivation and Viral Filtration). This presentation outlines the functional flexibility of the system and its advanced Buffer Management capabilities (in-line conditioning and in-line dilution), all contained within one propriety multi-functional hardware, flow path and operating software and its deployment in a continuous process. We present data from a successful non-GMP operation of multiple connected, integrated SymphonXTM skids in FUJIFILM Diosynth Biotechnologies’ continuous monoclonal antibody production platform, MaruXTM. The SymphonX™ design simplifies the single-use consumable supply chain, allows plug and play flexibility across all downstream unit operations and provides a generic operator interface to de-risks execution of complex continuous bioprocessing operations in a multi-product facility.

Embrace Molecule Complexity With Optimized Expression

12:30 PM ET — Anil Sebastian, PhD, Group Leader, Cell Culture Development, and Joachim Klein, PhD, Head of Microbial Strain Development, Lonza Pharma & Biotech

Biologic drugs are increasingly complex, creating numerous challenges for biopharmaceutical companies from higher candidate failure rates to expression and manufacturing obstacles. Our Mammalian and Microbial expression systems and processes address the needs of more complex candidates including vector design, cell line and strain selection and analytics to measure product quality attributes.  Case studies will be discussed demonstrating the range of expression capabilities across mammalian and microbial derived molecules.

Don't Let the Immune Response Blind-Side You:
The Importance of Early Immunogenicity and Immunotoxicity Assessment

1:00 PM ET — Noel Smith, Head of Immunology, Lonza Pharma & Biotech

Harnessing the power of the immune system has revolutionized the treatment options for patients with serious illnesses in fields such as oncology and autoimmune disease. However there are concerns about unwanted immune responses leading to serious adverse events.  Regulatory agencies throughout the world require a thorough assessment of a biotherapeutic’s potential to trigger these unwanted responses, a requirement complicated by the fact that “traditional” animal studies may not be highly predictive in this area. Assessing the potential for immunogenicity and immunotoxicity early in candidate selection helps you make better decisions, evaluate safety and can accelerate development time. This presentation will focus on solutions to address the potential for unwanted immune responses both in early candidate selection as well as for improving existing drugs.

bioCERTIFIED: Turn Your Quality Management System into a Competitive Advantage

1:30 PM ET — Jigisha Patel, Vice President of Regulatory Compliance & Technical Services, Spectrum Chemical