BPI Theater

Cell and Gene Therapy Modalities: Business and Manufacturing Strategies Influencing the Decision to Develop One Therapy Type Instead of Another

Moderator Patricia Seymour opened the panel by asking each presenter to introduce himself and his company’s technology. Caribou Biosciences: Cell Therapies Panelist Justin Skoble (vice president of technical operations with Caribou Biosciences) is responsible for developing the company’s next-generation CRISPR genome-edited cell therapies. Before joining Caribou in 2019, he was chief development officer at Acton Therapeutics and served in positions of increasing responsibility in immuno-oncology and vaccine development at Aduro Biotech, Anza Therapeutics, and Syros. His experience encompasses development of…

Cell and Gene Therapy: Introducing New Technologies to Compress Timelines, Increase Capacity, and Reduce Risk

Moderator Nicholas Vrolijk (managing director, BPTG, BDO USA) opened the second panel on therapeutic cell and gene therapy by introducing the participants. David Chang (CEO, WuXi Advanced Therapies) has 30 years in the industry with prior experience at Celgene, Roche Genentech, Biogene, and BASF. He has worked in cell and gene therapy and biopharmaceutical technical development, manufacturing operations, engineering, and strategy. Chang: WuXi Advanced Therapies is a globally integrated CTDMO providing front-end research and discovery all the way through development,…

Platform Processes Accelerating AAV Vector Production from Gene to Clinic

Tom VanCott, PhD, global head of product development, Catalent Biologics. VanCott emphasized the difficulty of manufacturing cell and gene therapies, noting the large demands for such products from both companies and patients. Manufacturing complexities include the need for raw materials — plasmids, DNA, viral vectors, and cells. Not only is scale-up difficult, but current supply-chain interruptions are slowing access to reagents, media, and disposable bags and resins. VanCott described Catalent’s approach to accelerating delivery of cell and gene therapy treatments…

Building a Comprehensive Toolbox for Oligonucleotide Drug Discovery

Dave Madge, vice president, WuXi AppTec. Dave Madge oversees a number of WuXi AppTec’s preclinical discovery services from his base in Cambridge, UK. He spoke about his company’s capabilities in building new modalities, in particular for oligonucleotide therapeutics. Following an introduction on new modalities and oligonucleotides, he presented a workflow for discovery, chemistry, and biology for siRNA therapeutics, focusing on synthetic DNA and RNA therapeutics. Two main factors contribute to the importance of alternative modalities. First, within the small-molecule and…

The Future of AAV Gene Therapy Is Scalable

Ryan Cawood, chief scientific officer, WuXi Advanced Therapies. Cawood began by noting that WuXi Advanced Therapies supports clients throughout their journeys toward developing cell and gene therapies. With the acquisition of the UK-based contract research organization (CRO) Oxgene and its adenoassociated virus (AAV) and lentivirus platforms (known as the TESSA and LentiVEX platforms/technologies, respectively), WuXi Advanced Therapies now can scale processes up to good manufacturing practice (GMP) manufacturing through to commercial supply. After describing the work that takes place in…

Enabling Next-Generation Biomanufacturing Using Cell-Free Technology

Weston Kightlinger, director, cell-free protein synthesis, National Resilience. Kightlinger shared his company’s work to enable next-generation biomanufacturing, focusing on biologics and cell-free protein synthesis. Resilience was formed to address the need for robust manufacturing supply chains that remain stable amid times of disruption and enable design of therapeutics in pace with scientific advancements. Resilience is a technology-focused manufacturing company dedicated to democratizing access to complex medicines. It is building a network of high-tech manufacturing facilities: 11 sites, 1,700 employees, and a…

Innovation as the Key to Gene Therapy Manufacturing

David Backer, chief commercial officer, Oxford Biomedica. Backer described the business models of Oxford Biomedica and Oxford Biomedica Solutions. The public company, OXB, licenses its technology. It historically has focused on lentiviruses but has moved into AAV. End-to-end capabilities are offered at both the Boston and Oxford sites. The company has grown rapidly because even though it specializes in lentivirus production, it was asked to increase its capabilities to manufacture an adenovirus-vectored COVID-19 vaccine for AstraZeneca as part of a…

How a Robust, Fully Integrated, and Highly Vetted Cell-Line Development Platform Accelerates IND Timelines

Sherry Gu, senior vice president of CMC management, WuXi Biologics. Since 2020, WuXi Biologics has supported process development and manufacturing for more than 30 biopharmaceuticals associated with COVID-19. Those include three authorized/marketed products and multiple advanced clinical candidates representing modalities such as monoclonal antibodies (MAbs) and vaccines. Gu explained how her company’s integrated platform for cell-line development (CLD) has enabled customers to advance their programs rapidly. The company’s custom protein-generation services support research and development (R&D) activities, culminating in developability…

Accelerating Industrialization of Genomic Medicine

Clive Glover, general manager, gene therapy, Pall Corporation. Glover observed that demand continues to grow for genomic medicines such as virally delivered gene therapies and RNA-based vaccines and treatments. With several such products approaching regulatory approval and many more in the clinical pipeline, manufacturing delays are inevitable. One strategy for reducing bottlenecks involves standardizing production processes for viral vectors and lipid nanoparticles (LNPs) that are used to deliver genomic therapies. Glover described the work of his company and other Danaher…

Partnering with a CDMO: Past the Service Offering

Elie Hanania, PhD, vice president of process development for viral vector technologies, Avid Bioservices. Speaking as a representative of a global biopharmaceutical manufacturer, Hanania explored several considerations for identifying a suitable third-party manufacturer. Developers approach contract development/manufacturing organizations (CDMOs/CMOs) for different reasons. Biotechnology start-ups often lack internal capabilities for producing clinical-trial drug substance; thus, many such companies leverage CDMOs’ capacity and process-development expertise. Large, established pharmaceutical companies might work with contract partners to free up internal capacity for other products,…