John Aunins view bio | back to agenda
Executive Vice President of Bioprocess & Manufacturing and Chief Technology Officer, Seres Therapeutics

Human Microbiome Therapeutics

The NIH Human Microbiome Project which ran from 2007-2012 resulted in a vast new appreciation for the role of microorganisms in health and disease – gut microbes are now thought of as a “forgotten human organ”. The role of the microbiome is increasingly evident: infectious, inflammatory, and metabolic diseases and even oncology treatment are all affected or caused by dysbiosis. The White House recently created a National Microbiome Initiative to acknowledge the importance of this field of research. Seres Therapeutics was founded in 2010 to explore the potential for live microbial ecologies (Ecobiotic^® Therapeutics) to be developed as treatments or prophylactics against human disease caused by dysbiosis. Seres currently has clinical-stage programs in Clostridium difficile and Ulcerative Colitis, and research programs in human stem cell transplantation (GvHD), immune-oncology treatments, and rare diseases (e.g. – urea cycle disorders). This talk will discuss the design, fermentation, processing, and analytical characterization of live microbial products as therapeutics for human disease, and the challenges posed to the Industrial Microbiologist and Biotechnologist.

Jeffrey C. Baker view bio | back to agenda
Deputy Director, Office of Biotechnology Products, CDER, FDA

Mirrors and Reflections: Perspectives on the Development and Manufacture of Biologic Medicines in the United States

We are now several years into a period of major changes in the regulation of Biotech products. The Affordable Care Act revised the definition of biological product and provided an abbreviated licensure path for biosimilars. The FDA Safety and Innovation Act provided a new timeline and structure for industry/FDA interactions in the development of biologics through BSUFA, introduced Breakthrough Product Designation, and clarified accountabilities for criminal adulteration of drugs in supply chains that are more complex and increasing global. The introduction of new technologies and business strategies continues to impact lifecycle management of approved biologics and we have seen an increase in submissions and a growth in the regulatory units responsible for review and regulation. This talk is not a deep dive into any of these topics, but rather a reflection upon how they may have impacted one another and upon the renewal of patient-centered, data-driven, risk-based decision making in the development and manufacturing of biologic products.

David Berry view bio | back to agenda
General Partner, Flagship Ventures

Entrepreneurial Innovation at the Frontier of Health

The start-up has emerged as playing a key role in innovation, but has the benefit of playing a unique role in the ecosystem. Entrepreneurial innovation is free from the encumbrances of large companies and even of the tenure system, which allows for rapid iteration and free movement between and across traditional fields. The result of the expansion of entrepreneurial innovation is the growth of novel technologies in a range of sectors, with notable impacts in biotechnology, where a multitude of cutting-edge capabilities have been recently enabled and used as the foundation of new product-platforms. Many of these new technologies have either yielded or required new manufacturing approaches. Rapid innovation in manufacturing has the benefit of translating these innovations into scalable products in therapeutics and beyond, but also is enabling to the fields more broadly, directly and indirectly. The combination of deep scientific insights with unfettered innovation is well positioned to continue producing impactful new products and platforms and the concomitant advances in engineering.

James Bouressa view bio | back to agenda
Senior Director, Clinical Manufacturing, Pfizer

Facility Considerations – Planning for the Future

The biotech industry is experiencing an increase not only in recent approvals. We’re also seeing an increased number of programs progressing to late stage clinical trials. The introduction of biosimilars is also placing additional demand on existing manufacturing facilities.

In this talk, we’ll review five capital expansion projects currently being undertaken at Pfizer to meet our projected demand for both clinical and commercial manufacturing in the year 2020 and beyond. Consideration will be given to existing capacity, current facility layouts, and ease of expansion as well as greenfield initiatives. Bioreactors considered in this presentation range from 1,000 Liters to 12,000 Liters in scale.

Stuart Bussell view bio | back to agenda
Senior Director, Process Development, Sutro Biopharma

Sutro’s Cell Free Manufacturing Systems - Platforms Well Suited for the Flexible Manufacture of the Protein Therapeutics of Today and Tomorrow

Sutro’s cell free manufacturing systems separate fermentation operations from target protein production. Fermentations generate cell free extract and other shared custom reagents that can be industrially manufactured, released, and stored for later use during cell free production of all target proteins. This naturally leads to easier implementation of distributed manufacturing strategies when compared to cell-based production systems. When combined with the platforms’ facility for protein engineering and site-specific conjugation, Sutro’s cell free manufacturing systems are well suited to meet the product design and manufacturing challenges posed for products like bispecifics and ADCs.

Jie Chen view bio | back to agenda
Vice President, CMC Operations, WuXi Biologics

Scale-out with Disposable Manufacturing

The success of therapeutic biologics in past decades has reshaped the pharmaceutical industry landscape. Today, thousands of biopharmaceutical drugs have been approved with more than ten thousand in pipeline at various preclinical and clinical development stages. The diverse biological molecule structure design and wide range of predicted market, together with increasing dynamic biotech business models, result in many new challenges to biomanufacturing fields. Without sacrificing quality, new generation biomanufacturing facilities emerge with “value (not volume) driving design” focusing on flexibility, scalability, efficiency, safety, and regulatory compliance.

WuXi Biologics has constructed the world’s largest mammalian cell culture manufacturing facility using disposable bioreactors with 2x1000L perfusion and 14x2000L fed-batch capacity. The upstream scale-out strategy allows the facility to by-pass the scale-up limitation from the disposable bioreactor size, thereby maximizing the disposable technology benefits while minimizing the scale-up risks associated with product quality and process performance. Downstream scale-out by cycle provides flexibility in manufacturing facility for easy integration of continuous process technology. In addition, thoughtful hybrid combination of disposable and stainless steel media/buffer tanks offers additional manufacturing cost saving. Traditional room classification and campaign operation ensures the safety and regulatory compliance for multiproduct facility. Such facility design presents better scalability and flexibility on API manufacturing to accommodate biopharmaceutical drugs with different productivity levels and wide range market demands.

Eliana Clark view bio | back to agenda
Vice President, Manufacturing Sciences, Biogen

Biogen’s Approach to Advanced Process Control in Biomanufacturing

Biogen is taking a holistic approach to develop and implement an end-to-end advanced process control (APC) strategy for monoclonal antibody drug substance manufacturing with the goal of improving process robustness, product quality consistency, and operational efficiency. Under this APC paradigm, raw material variability and its impact on process performance are well understood through extensive raw material and process characterization, resulting in a raw material control strategy. Inline and at line measurements, and dynamic feedback and feed forward controls are used in the production bioreactor and selected purification steps to improve product and process consistency and robustness. Rapid analytical technologies including multi-attribute assays are deployed to support right time release at selected intermediate process steps instead of the final drug substance step. Finally, consistency and predictive models are utilized to support product quality assessment and batch release. This presentation will describe how these components are integrated to deliver a more robust process control and efficient manufacturing over traditional process control.

Parrish M. Galliher view bio | back to agenda
Chief Technology Officer, Upstream, GE Healthcare Life Sciences

Manufacturing Strategy and Facilities for a Diverse World - Avoiding Facility Obsolescence

As biotech pipelines grow in size and diversity, manufacturers are increasingly challenged to adapt their facilities to different capacities, drug types, and changes in process architectures. This increasing diversity comes on top of the uncertainty of predicting manufacturing capacity in the face of unpredictable clinical trials and the normal chaos of the biotech industry. Given these uncertainties, many facilities have to be modified before they are complete or are obsolete before they are validated. The presentation will review the growing diversity of the biotech industry and the probability of any one facility surviving intact until it is validated for use, followed by an examination of manufacturing and facility strategies and designs that can mitigate capacity uncertainty risk and extend facility usefulness, productivity and lifetime.

Shekar Ganesa view bio | back to agenda
Executive Director, Process Development, Amgen

Advanced Process Control Opportunities for Biologics

The development of a commercial product manufactured according to the Quality Target Product Profile (QTPP) will ensure that it will meet its desired Target Product Profile (TPP) (i.e. quality, in vivo safety and efficacy). In this presentation we will share examples of process analytical technologies (PAT) and real-time monitoring of product attribute control (PAC) during process development.

Hugh A. Haydon view bio | back to agenda
President, Kentucky BioProcessing

Manufacturing Biologics Using Tobacco Plants

Recent advances have brought renewed attention to the use of tobacco plants as a host organism for the production of biopharmaceutical proteins. Since its founding in 2006, Kentucky BioProcessing has established itself as a world leader in developing and executing scalable and cGMP-compliant processes for manufacturing products in this novel system.

This presentation will provide a brief history of the system while focusing on the unique advantages offered by the use of tobacco plants in protein expression. Information will be provided on the manufacturing process with emphasis on upstream steps that differentiate the tobacco system from traditional cell-based production methods. Case studies will be cited to illustrate the speed offered by transient plant-based gene expression as well as the flexibility afforded by use of the plant as an entire complex organism as production host.

Gregory Hiller view bio | back to agenda
Associate Research Fellow, Culture Process Development, Pfizer

Upstream Options for Process Intensification: Perfusion, Metabolism Meddling for Growth Inhibitor Control, Hybrid Processes, and Anything Else to Get More Cells for More Stuff

We will discuss methods of process intensification that our group has been developing over the last decade. Building on our technique of glucose-limited nutrient feeding to fed-batch cultures, we have recently identified and quantitated a significant number of previously unreported small molecular weight metabolites that accumulate in CHO cell cultures and work synergistically to slow cell division and cap peak cell densities. These growth inhibitors, combined with the practical limits of feed volume addition and the problem of amino acid counter ion and miscellaneous osmolyte accumulation, serve to limit productivity in the industry-standard fed-batch bioreactor. To break these barriers, we have begun to investigate mixed modes of bioreactor operation, some using novel methods for cell-controlled perfusion, which can substantially reduce the volumes of perfusion medium required, suppress lactic acid formation, shorten the time to reach peak cell densities, and reduce the complications associated with cell retention devices. Our methods of high-intensity perfusion and mixed-mode bioreactor operation are also more compatible with the move to continuous downstream processing as the stream delivered is more concentrated and provides a more continuous daily mass of product to allow for reduced affinity chromatography scale.

Nathan C. Ihle view bio | back to agenda
Vice President, CMC Strategy & Management, Seattle Genetics

Manufacturing Challenges for Antibody-Drug Conjugates: Past, Present and Future

Antibody-drug conjugates (ADCs) are an important new class of pharmaceuticals in the treatment of serious diseases, especially cancer. They consist of three distinct units: a monoclonal antibody which provides a targeting mechanism; a small molecule “drug” which is responsible for inducing a pharmacologic effect within a target cell or tissue; and a “linker” which is responsible for covalently attaching the drug to the antibody. Manufacture of these hybrid molecules relies upon traditional chemical and biological technologies, but also presents some unique challenges. Conjugation processes must accommodate the physicochemical and reactivity properties of all the components, which are typically quite distinct. Most manufacturing facilities are not equipped to deal with these unique materials and processes, and most manufacturing personnel are not trained in both chemical and biological processing. In addition, most ADCs currently under development utilize highly potent cytotoxic drugs, and this adds complexity to the manufacturing manipulations as extra precautions must be instituted in order to assure worker safety.

We will discuss some of our manufacturing and regulatory experiences from past and present ADC projects. We will also highlight areas where challenges remain, and suggest areas for future development of manufacturing technologies related to ADCs.

Avak Kahvejian view bio | back to agenda
Co-founder and Chief Executive Officer, Rubius Therapeutics

Engineering Red Blood Cells for Therapeutic Function

Rubius Therapeutics has built a platform for producing allogeneic Red Cell Therapeutics (RCTs) – stem cell-derived red blood cells genetically modified and expanded ex vivo to possess biotherapeutic capabilities. RCTs are able to harbor active intracellular as well as extracellular proteins, ranging from enzymes and cell targeting moieties to agonists and antibodies. As such, RCTs represent a transformational therapeutic platform in their ability to address a wide array of indications including metabolic disorders, autoimmune diseases, and cancer. In oncology, they enable multiple distinct modalities including tumor starvation, enhanced apoptotic signaling, and immune checkpoint inhibition, among others. Rubius is currently evaluating multiple candidates in parallel preclinical studies.

Stephen Kennedy view bio | back to agenda
Chief Technology Officer, Histogenics

Manufacture of Cell and Tissue Therapy Products: Cartilage Regeneration

Several cell and tissue therapy products have been developed for articular cartilage regeneration and repair over the past two decades. NeoCart is a late-stage autologous product that has been demonstrated clinically to be effective in repairing and regenerating cartilage, and represents the next generation technology for articular cartilage repair. Chondrocytes isolated from individual patient cartilage biopsies are expanded then embedded in a Type 1 collagen scaffold to produce cartilage tissue with biomarkers and biomechanical characteristics like native cartilage. When implanted, NeoCart integrates with native tissue to repair cartilage defects, resulting in improved function and reduced pain for the patient. Significant manufacturing and logistical challenges present major impediments for reliable supply of NeoCart to the tens of thousands of patients that could potentially benefit from NeoCart annually in the US. Scale-out technologies, including multi-unit tissue bioreactors, will be developed to overcome manufacturing challenges. Allogeneic technologies utilizing iPS cells are also under development.

Konstantin Konstantinov view bio | back to agenda
Senior Vice President, Manufacturing & Process Sciences, Codiak BioSciences

Toward Commercialization of Exosome Technology

Codiak BioSciences is developing a broad platform for the delivery of macromolecules to the cytoplasm of diverse cells and tissues, to create a proprietary pipeline of novel, targeted medicines for diseases with high unmet medical need. Our technology capitalizes on the unique properties of exosomes which have been shown to deliver nucleic acids, proteins, lipids, and small molecules to various cell types. Codiak will build and expand upon the natural tropism of exosomes for delivery of payloads to specific cell and tissue types, to solve the challenges of delivering macromolecules across biological membranes. To realize the full potential of exosomes, Codiak is pioneering the development of industrial-scale production. Our efforts are aimed at optimizing the collection of exosomes through cell-culture processes, and then scaling the purification process for commercial production. We are developing existing biological production and manufacturing technologies in novel and proprietary ways to produce exosome therapeutics on an industrial scale.

René Labatut view bio | back to agenda
Vice President, Technology Innovation, Sanofi Pasteur

Paradigm Shift: Perspective of mRNA in Vaccine

mRNA is an emerging new class of drug with multiple applications foreseen in treatments related to infectious disease vaccination, oncology, and others, such as replacement therapy for single-gene deficiencies. This new product approach leverages the translation mechanism of eukaryotic cells. The final immunogenic or therapeutic molecule is the protein generated by the patient’s cells themselves. Beyond opening up several new therapeutic fields, mRNA could be considered as a vectorised way that drastically increases drug efficiency by obtaining a similar effect with a significantly lower dose of injected product compared to classical protein products. It should also be seen as a technology platform boosting CMC development of new specialties. This could also significantly change the manufacturing paradigm and its economy. This talk will have a look at this field overall and focus on anti-infectious vaccines to exemplify how far it could go.

J. Christopher Love view bio | back to agenda
Associate Professor, Department of Chemical Engineering, MIT

Challenges and Opportunities for Distributed Manufacturing of High-Quality Biopharmaceuticals

Recombinant proteins are an important class of biopharmaceuticals. Manufacturing strategies for these drugs have relied predominantly on centralized facilities with large capital and operating costs to generate drug substance and to assure quality of the final product. Regional or localized manufacturing could provide benefits for patients in emergency situations, in rare diseases, or in underserved areas of the world. This talk will consider some of the techno-economic challenges faced by distributing production, and highlight opportunities for enhancing technologies that could enable robust and reproducible manufacturing of high-quality proteins.

Aura Mackenzie view bio | back to agenda
Senior Principal, Global Pricing and Product Strategy, Precision for Value

Global Market Access Manufacturing Considerations for Transformative Products

Globally, payers recognize that biopharmaceuticals, most topically the cell and gene therapies, are the next big step in the evolution of medicine. Unsurprisingly, the upfront cost of these treatments to payers and uncertainty over long-term outcomes are the major pricing and market access hurdles. However, many manufacturers fail to appreciate sufficiently early in development how healthcare funding processes and manufacturing logistics can also present significant market access constraints. These include center of excellence site selection, manufacturing site selection, reimbursement pathway constraints, and cross-border dynamics. We will present a series of relevant commercial and market access strategic considerations that manufacturers should factor into their development plans for these transformative products.

Samantha Maragh view bio | back to agenda
Leader, Genome Editing Program, NIST

Targeted Genome Editing: Overview and Measurement Needs for Cell and Gene Therapies

Genome editing technologies enable targeted DNA sequence changes to be made in the cells of virtually any organism. Genome editing is being actively pursued to transform precision medicine and bioscience in areas such as cell therapy, synthetic biology, novel antimicrobials and antivirals, protein therapeutic biomanufacturing, agriculture, and global food production.

In response to high demand from stakeholders, NIST organized a special Genome Editing Standards Workshop on May 2, 2016, in partnership with the American Society of Gene and Cell Therapy and sponsored by Microsoft and Editas Medicine, to bring together experts in government, academia, industry, and informatics to begin identifying measurement assurance and standards: barriers, gaps, and potential solutions for the genome editing field, and to chart a path forward for increased confidence in assessing genome editing. This talk will give an overview of genome editing and its applications as well as the NIST workshop and NIST’s developing program to support the measurement and technology innovation needs to support the translation of genome editing to cell and gene therapies.

PJ Millan view bio | back to agenda
Head of Process Automation, R&D Platform Technology & Science, GlaxoSmithKline

Game of Clones

From cell line to commercial manufacturing, a platform, scale-out approach to managing the supply chain of process knowledge: Critical to the success of R&D-driven companies such as ours is to have an effective and efficient New Product Introduction process; yet historically and currently, document and people-centric information and manual processes dominate. Information associated with new and existing products is scattered throughout the company in information silos of various formats. We need a single version of the truth! At GSK, we have begun building and integrating such a solution with a Process Design-centric approach that leverages a scale-out approach for both equipment and technology.

Peter Nguyen view bio | back to agenda
Technology Development Fellow, Laboratory of James Collins, Wyss Institute of Biologically Inspired Engineering, Harvard University

Just Add Water: Portable, On-Demand Biomolecular Manufacturing

Synthetic biology uses living cells as molecular foundries for the biosynthesis of drugs, therapeutic proteins, and other commodities. However, the need for specialized equipment and refrigeration for production and distribution poses a challenge for the delivery of these technologies to the field and to low-resource areas. Here, we present a portable platform that provides the means for on-site, on-demand manufacturing of therapeutics and biomolecules. This flexible system is based on reaction pellets composed of freeze-dried, cell-free transcription and translation machinery, which can be easily hydrated and utilized for biosynthesis through the addition of DNA encoding the desired output. We demonstrate this approach with the manufacture and functional validation of antimicrobial peptides and vaccines and present combinatorial methods for the production of antibody conjugates and small molecules. This synthetic biology platform resolves important practical limitations in the production and distribution of therapeutics and molecular tools, to both the developed and developing worlds.

Rajeev Ram view bio | back to agenda
Professor, Department of Electrical Engineering and Computer Science, MIT

Advances in Microbioreactor and Analytic Platforms for Bioprocess Support

Two technologies that have recently transformed biopharmaceutical manufacturing are process analytics for rapid on-line monitoring of critical quality attributes (CQA) and tools for high-throughput process development. We will consider several novel approaches to analysis including optical assays developed in our lab, nanofluidics developed by the Han Lab, and nanoparticle-based sensors developed in the Strano Lab. These technologies together offer the potential for comprehensive measurement of CQA in real-time. Recently, a microfluidics-based bioreactor system was developed for continuous and perfusion processes. This microbioreactor’s smaller 1-2 mL volume enables long-term continuous experiments with negligible media usage. On-line cell density and precise flow-rate control enabled controlled genetic switching of Pichia pastoris in a perfusion microbioreactor. Commercial versions of perfusion microbioreactors are now available for high-density CHO cell cultue. A mature perfusion microbioreactor platform has the potential to fill a critical need in the evolving landscape of continuous biomanufacturing.

Rahul Singhvi view bio | back to agenda
Senior Vice President and Chief Operating Officer, Global Vaccine Business Unit, Takeda Pharmaceuticals International

Impact of Vaccine Manufacturing Excellence on Global Health

Vaccines have proven to be excellent tools to prevent infectious disease. Small pox has been eradicated and polio is on the verge of eradication. Disease burden of many other infectious diseases, such as measles, diphtheria, tetanus, and rotavirus, has been reduced dramatically post introduction of an effective vaccine. In spite of these successes, the potential impact of vaccines has been stunted due to lack of affordable and timely access of vaccines globally. There are many reasons for this but manufacturing cost and capacity is one of the chief reasons. This presentation will investigate the root cause for the incessant manufacturing issues plaguing vaccine production as well as the lack of affordable supply. Fundamental analysis of the vaccine industry and unique features of vaccines reveal that addressing the manufacturing issues at the root level requires rethinking of the vaccine development model, including global partnerships and collaboration with stakeholders ranging from policy makers to industry and academia. A comprehensive solution is offered to address the issue of access and affordability while maintaining sustainability of the industry.

Ariel Dora Stern view bio | back to agenda
Assistant Professor of Business Administration, Harvard Business School

The Impact of the Entry of Biosimilars: Evidence from Europe

Biologic drugs represent a large and growing share of drug spending in the United States, accounting for less than 1% of prescriptions filled but nearly 28% of drug spending. Whereas small-molecule drugs have historically faced competition from generic drugs after patent expiration, biosimilars – biologic drugs that have been shown to be therapeutically equivalent to an already approved original biologic – have only been approved in the United States since 2015. Europe, however, has had biosimilar entry since 2006 and we study the first eight years of biosimilar competition in 23 European countries. A major contribution of this project is the completion of a detailed survey, allowing us to characterize European biologic drug procurement institutions over time. Using data from three classes of biosimilars, we analyze how market features and public policies predict entry, market prices, and penetration. We find significant heterogeneity across countries and drug classes in all of these outcomes. While we observe that effective buyer institutions (in particular, committed tenders) are associated with increased biosimilar entry and penetration, price patterns are more difficult to glean from the available data. Our estimates can inform ongoing policy discussions on both sides of the Atlantic about the economic implications of biosimilar policies.

Christopher Stevens view bio | back to agenda
Senior Director, Operations, Planning, and Strategy - Biopharm and Cell & Gene Therapy, GlaxoSmithKline

Game of Clones

From cell line to commercial manufacturing, a platform, scale-out approach to managing the supply chain of process knowledge: Critical to the success of R&D-driven companies such as ours is to have an effective and efficient New Product Introduction process; yet historically and currently, document and people-centric information and manual processes dominate. Information associated with new and existing products is scattered throughout the company in information silos of various formats. We need a single version of the truth! At GSK, we have begun building and integrating such a solution with a Process Design-centric approach that leverages a scale-out approach for both equipment and technology.

Jorg Thommes view bio | back to agenda
Senior Vice President, Engineering & Technology, Biogen

Next Generation Manufacturing in Biogen’s Hybrid Biomanufacturing Network

Over the last 30 years, the Biopharma Industry has had remarkable successes and has delivered tremendous value to society through the development of innovative medicines. During this time our industry has also matured to an extent that the traditional paradigm of value creation solely through development and commercialization of innovative science may change. The economics of developing a drug pipeline became more challenging, the reimbursement environment is changing, biosimilars and increasing globalization add opportunities and competition. For the longest time, manufacturing was not part of the value equation; the transformation going on in our industry, however, requires that operations becomes part of the value chain. This presentation will discuss how biologics manufacturing can be turned from a necessary evil to a competitive advantage.

Dramatic improvements in productivity over the past decade coupled with the development of robust platforms have enabled our industry to break with the paradigm of large stainless steel manufacturing facilities as the sole option for producing biologics. Smaller facilities became possible and single-use technology has added flexibility. These are the foundations to Biogen’s hybrid manufacturing network. Utilization of a manufacturing network is as big a driver of manufacturing economics as the productivity of the processes executed in these facilities. While the basic technology utilized in biologics manufacturing has been established decades ago, there is still great demand for innovation, which has to be directed towards optimizing network utilization. In this context, we will discuss Biogen’s Next Generation Manufacturing concept, which combines process innovation, facility design, and advanced process control strategies in a holistic approach to manufacturing innovation.

Krystyn J. Van Vliet view bio | back to agenda
Professor, Department of Materials Science and Engineering and Department of Biological Engineering; Director of Manufacturing Innovation, MIT Innovation Initiative, MIT

Manufacturing Innovation via Public-Private Partnerships: Challenges and Opportunities for Emerging Therapies

Promising emerging treatment modalities, including modified protein, gene, and cell therapies, face many challenges that require innovation in manufacturing technology and development of a well-trained workforce. In biopharmaceutical manufacturing as well as several other manufacturing sectors ranging from digital to additive manufacturing, public-private partnerships have the potential to speed that technical innovation, to enable helpful standards, and to educate a workforce for a range of rewarding careers in advanced production. Here we will discuss the US public-private partnerships called Manufacturing USA Institutes. We will then discuss how such partnerships could advance promising new technologies to manufacture and qualify cells as products, using examples from the US and Singapore, including industry-academia-clinic collaborations with regulatory agency engagement that can accelerate technology adoption by small and large companies.

J. Fraser Wright view bio | back to agenda
Co-founder and Chief Technology Officer, Spark Therapeutics

AAV – Based Gene Therapy: Scalable CMC to Support Clinical Program Advancement

AAV-based gene therapy vectors have been developed and are being evaluated in several clinical programs for serious genetic diseases. This presentation will provide an overview of challenges relating to the chemistry, manufacturing and controls for AAV investigational products, describe strategies to address key challenges such as product quality and manufacturing scalability, and summarize select clinical program results.