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Ed Boyden, Ph.D.
Tools for Mapping and Repairing Disease States: Can We Make Therapeutic Invention into a Mature Design Science?
Massachusetts Institute of Technology
Tyler Jacks, Ph.D.
The Convergence Model of Cancer Research: Solving the Problems of Cancer through Interdisciplinary Approaches
Massachusetts Institute of Technology
Clinical and Commercialization Developments
After many trials, errors, and milestones, regenerative medicine has become a mainstream part of the biologics industry, supported by at least 670 companies and clinics of all sizes. However, there are many lessons experienced by the protein-based industry that can be applied to further improve commercialization success.
Moderator: BPI Magazine
Gene Editing and CRISPR – Entering the Clinic
Genomics paired with gene and cell therapy are quickly becoming a force in the development of therapeutics. Many of these therapeutics are targeted toward rare disease populations. As a result, clinical trial design is key for the success of these therapies. This session will focus upon gene and cell therapies, genomics, and leveraging of new technologies to design robust clinical trials.
The design and development of robust ADA assays for CRISPR/Cas9-based therapies present a unique challenge for the advancement of genomic medicines. To enable the screening of ADAs in response to a viral vector and a transgene protein product Cas9 in preclinical samples and human populations, we developed a sensitive immunogenicity assay which has great potential to detect low levels of ADAs, and allows for multiplexing of ADA measurements in a small sample
With an increasing percentage of Regenerative Medicine clinical trials stemming from hMSC-based technologies, the field is ripe for standardization that would reduce redundancy across the industry. This talk will outline bottlenecks in RegenMed product development and highlight strategies that companies can implement to simplify their development programs and greatly shorten the time needed for clinical trial initiation.
North American Regulatory, Policy and Healthcare Landscape Change
The regulatory environment changes and it is important to keep up with the potential FDA processes that can aid the development of a product. This talk will focus on changes made in the 21st Century Cures Act focused on regenerative medicine advances therapies. It will define the criteria and discuss the pros and cons of RMAT designation.
Analysis and Characterization
Establishing an allogeneic therapeutic approach requires besides a robust production platform, for creation of “off-the-shelf” cell banks, a comprehensive characterization of the cell-based product itself. This work describes the development of a GMP-compatible system for hCPC production to support allogeneic cell therapy for cardiac repair and proposes an analytical toolbox, based on ‘omics technologies, to guarantee cell-based product’s quality and potency.
Rapid detection of contaminants, especially those affecting critical quality attributes, is essential for cell therapy products with short shelf lives. Integrating quality into the process through lot segregation, raw material qualification, environmental control, personnel training, and detailed procedures is critical because final results for conventional microbiological tests may not be available prior to product release or patient administration.US FDA approval of the MACI BLA in 2016 included three RMM product release assays for sterility, endotoxin, and mycoplasma.
Cultured cells, particularly mesenchymal stem cells, release exosomes and other extracellular vesicles (EVs) which can harbor significant biological functions. Conditioned media, rich in EVs but often discarded, are therefore a valuable resource. This talk will examine how EVs can be harvested from conditioned media and characterized, and will also address potential scale up and regulatory challenges.
FloDesign Sonics has developed a novel acoustic cell processing platform based on multi-dimensional standing waves. The platform has broad applications in biopharmaceutical, e.g., cell clarification, continuous manufacturing, and cell processing within cellular therapy applications, e.g., cell concentration and wash, cell culturing, and microcarrier/cell separation. This technology is single use, continuous,and scaleable for processing volumes from 250 ml to 2000L and with closed, integrated and automated process capability.
Adeno-associated viral (AAV) vectors have demonstrated great promise to safely deliver genes in vivo. One major challenge facing the industry is how to manufacture sufficient AAV quantities for clinical and commercial use. Here we’ll describe how Corning® CellSTACK® and HYPERStack® technologies can be used to scale-up AAV production and how Penn Vector Core has optimized processes for early-phase clinical manufacturing.
Design of Late Stage Clinical Trials and Clinical Centers
More than 5 million people worldwide are living with Parkinson's Disease (PD). Restoring dopamine neurons with stem cell-derived neurons is an attractive treatment strategy. With an award from NY State (NYSTEM), we developed hESC-derived mDA neurons as a cell therapy for PD. Together with BlueRock Therapeutics, we will seek FDA approval for a clinical trial in 2018.
To date, it has not been possible to identify patient or disease-specific factors that predict why some B-cell chronic lymphocytic leukemia patients and not others have such dramatic responses to CAR T cell treatment. We explored the mechanisms associated with clinical response, providing evidence for intrinsic T cell fitness in mediating durable anti-tumor responses and long-term complete remissions.
International Regulatory Considerations
In late 2014, the Japanese PMDA, revolutionized the way cell therapies are regulated making Japan arguably the fastest regulated jurisdiction in which to commercialize cell therapies. This paradigm shift created a significant commercial opportunity in Japan. From observing what has transpired in the industry in the 36 months since the reforms, what can be learned of the opportunity and how can it be leveraged?
The registration process for Advanced Therapy Medicinal Products (ATMPs) in Europe spans several legislative domains; Tissues and Cells, Gene Modified Organisms, Medicinal Product. This can add complexity to the registration process for these products. This talk will cover practical aspects of registering ATMP for clinical trial and marketing Authorization in Europe including a discussion of the accelerated pathways available to developers.
Relieving Bottlenecks In Gene Therapy Bioprocessing
We have developed platforms to manufacture T lymphocytes genetically modified with chimeric antigen receptors (CARs) using gamma retroviral vectors for Phase I clinical trials. These bioprocesses allow the generation of clinical doses in approximately 10 days. Yet, multiple challenges pertaining to manufacturing standardization, logistics and product characterization remain to be overcome to achieve broad usage and eventual commercialization of this therapeutic modality
Cell-based drug products manufactured with lentiviral vectors (LVVs) require advances in analytical methods to characterize their safety and efficacy. This talk will demonstrate that unique analytical challenges posed by gene therapy drug products can be addressed with new analytical methods involving CyTOF, single cell PCR, and in-vitro disease modeling.
Biotech Week Boston Keynote Presentations
MIT’s Koch Institute for Integrative Cancer Research was created to take advantage of the strengths at MIT in cancer science and cancer-oriented engineering. The Institute fosters interdisciplinary thinking and collaboration to tackle the most challenging problems in cancer biology, diagnosis, early detection, and treatment. Tyler Jacks serves as the Director of the Koch Institute and also runs a cancer genetics and cancer immunology laboratory there. He will illustrate examples of progress against cancer through his own work as well as from collaborative efforts with other scientists and engineers.
Increasing Gender Diversity in BioPharma: What Works and What Is Working?
Moderator: Cissy Young, Executive Director, Russell Reynolds Associates
This panel on increasing gender diversity in the biopharma industry will focus on actionable strategies and programs that both men and women can work on together to help improve gender diversity in their companies. The emphasis will be on what everyone in our industry can do, whether at small, medium or large organizations, and at every level of their company to make a difference and deliver the benefits of gender diversity for corporate performance.
Actionable takeaways will include how to build programs for:
—Male Allies and Champions for Gender Diversity
—Flexible Work Policies
—Mentorship and Sponsorship Programs
Increasing gender diversity will be critical for continuing to build our innovative biopharma industry in the Boston area and to continuing to deliver economic growth to Massachusetts, so come hear what you can do to contribute in your own organization today.