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Key Sessions

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

8:10am - 8:15am 5 mins
Clinical and Commercialization Developments
Chairperson’s Opening Remarks
  • Erika Trauzzi, Ph.D. - Director, Regenerative Medicine Applications, Sartorius Stedim Biotech
more
8:45am - 9:15am 30 mins
Clinical and Commercialization Developments
Pluripotent Stem Cells: Making the Case for Commercialization
  • Robert Deans - CTO, BlueRock Therapeutics
more
9:15am - 9:45am 30 mins
Clinical and Commercialization Developments
PANEL DISCUSSION: Leveraging key lessons learned from protein production to drive cell therapy innovation.
  • Moderator Anne Montgomery - Editor In Chief, BioProcess International Magazine
  • Panelist Philip Vanek, Ph.D. - General Manager, Cell Therapy Growth Strategy, GE Healthcare
  • Paenlist Jon Rowley, Ph.D. - Chief Executive & Technology Officer, RoosterBio Inc.
  • Panelist James Miskin - Chief Technical Officer, Oxford Biomedica
more

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.

Objectives:

  • To introduce and discuss what key lessons can be gleaned from the evolution of traditional bioprocessing to support the optimal future development of regenerative medicines
  • To review the rapid changes in bioprocessing materials, operator specialization, and the changing utilization of processing technologies shaping the current therapeutic development pathway
  • To propose expectations of what may happen over the next decade of its maturation.

Moderator: BPI Magazine

9:45am - 10:25am 40 mins
Main agenda
Networking Refreshment Break in the BWB Poster & Exhibit Hall
10:25am - 10:30am 5 mins
Bioprocessing
Chairperson’s Remarks
  • Christopher Bravery, Ph.D. - Director, Consulting on Advanced Biologicals Ltd, Advanced Biologicals Ltd.
more
10:25am - 10:30am 5 mins
Commercialization
Chairperson’s Remarks
  • Chris Gemmiti, Ph.D. - Head of Operations, Sentien Biotechnologies Inc.
more
10:25am - 10:30am 5 mins
Clinical
Chairperson’s Remarks
  • Amanda Micklus - Principal Analyst, Datamonitor Healthcare
more
10:30am - 11:00am 30 mins
Clinical
Crafting the Future Clinical Trial Design for Therapeutics Utilizing Gene and Cell Therapy Technologies
  • Dan Tierno - Strategic Implementation Manager, Bayer
more

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.

11:00am - 11:30am 30 mins
Clinical
Development of Multiplex Sensitive Anti-Drug-Antibody Assays for CRISPR/Cas9 Genomic Medicines
  • Junxia Wang, M.D., Ph.D. - Associate Director, Immunosafety and Bioanalytical Development, Editas Medicine
more

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

11:30am - 12:00pm 30 mins
Clinical
Strategies for Rapid Clinical Translation of hMSC-based Regenerative Medicine Products
  • Priya Baraniak - Scientist, RoosterBio
more

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.

10:30am - 11:00am 30 mins
Commercialization
21st Century Cures Act and Regenerative Medicine Advanced Therapy (RMAT) Designation
  • Joyce Frey-Vasconcells, Ph.D. - Regulatory Consultant, Frey-Vasconcells Consulting, LLC
more

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.

11:00am - 11:30am 30 mins
Commercialization
Experiences of Navigating the RMAT Designation Pathway
  • Kevin Healy, Ph.D. - Senior Director, Global Regulatory Affairs, Roivant Sciences Inc.
more

The 21st Century Cures Act included several provisions to accelerate and incentivize drug development, including creation of the Regenerative Medicines Advanced Therapy (RMAT) Designation. This designation confers several regulatory benefits to qualifying cell therapies and tissue products intended to treat life-threatening diseases. The presentation will give an overview of the application process and regulatory benefits associated with the RMAT designation.

11:30am - 12:00pm 30 mins
Commercialization
Navigating U.S. and EMA Regulatory Requirements for Autologous Cell Therapy Product
  • Kanti Thirumoorthy, Ph.D. - Senior Director, Quality, Kite Pharma
more

The unique nature of autologous cell therapy requires novel solutions to meet some of the regulatory requirements. Availability of small quantities of product and the need for a short time to release produce to patient necessitate establishing product and process monitoring programs to accommodate for the constraints of autologous therapy while satisfying the appropriate regulatory requirements.

10:30am - 11:00am 30 mins
Bioprocessing
Application of ‘Omics Tools for Better Understanding and Characterizing Cell-Based Allogeneic Products for Cardiac Repair
  • Patricia Gomes-Alves, Ph.D. - Senior Scientist, IBET - Instituto de Biologia Experimental e Tecnológica
more

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.

11:00am - 11:30am 30 mins
Bioprocessing
US Approval of Three Rapid Microbiological Methods for MACI Product Release
  • John Duguid - Senior Director, Research & Development, Vericel Corporation
  • Michael Brewer - Director, Head of Pharma Analytics, Thermo Fisher Scientific
more

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.

11:30am - 12:00pm 30 mins
Bioprocessing
Exosomes in Cell Manufacturing: From By-Product to Product
  • Kelvin Ng, Ph.D. - Scientist, Process & Product Development, RoosterBio, Inc
more

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.

12:00pm - 12:30pm 30 mins
Technology Workshop
Acoustic Cell Processing: A Disruptive Technology for Cell Clarification, Concentration, Washing and Perfusion in Therapeutic and Cell/Gene Therapy Application
  • John Rozembersky - Vice President, Marketing & Application Technologies, FloDesign Sonics
more

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.

12:00pm - 12:30pm 30 mins
Technology Workshop
A Platform Approach for Scaling AAV Production for Gene Therapy Process Development and Manufacturing
  • John Huynh - Director, The University of Pennsylvania
more

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.

12:35pm - 1:55pm 80 mins
Main agenda
Networking Luncheon in the BWB Poster & Exhibit Hall
1:55pm - 2:00pm 5 mins
Bioprocessing
Chairperson’s Remarks
  • Kim Warren, Ph.D. - Head of Operations, AVROBIO
more
1:55pm - 2:00pm 5 mins
Commercialization
Chairperson’s Remarks
  • Antonio Lee, Ph.D. - CEO & Managing Director, Medipost America Inc.
more
1:55pm - 2:00pm 5 mins
Clinical
Chairperson’s Remarks
  • Shon Green - Research Scientist, Eureka Therapeutics, USA
more
2:00pm - 2:30pm 30 mins
Clinical
A Cell Therapy for Parkinson’s Disease
  • Stefan Irion - Program Manager, Memorial Sloan Kettering Cancer Center, USA
more

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.

2:30pm - 3:00pm 30 mins
Clinical
Predictors of Response to CD19-Specific CAR T Therapy
  • Jun Xu - Product Development Specialist, Center for Cellular Immunotherapies, University of Pennsylvania
more

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.

3:00pm - 3:30pm 30 mins
Clinical
Designing Late Stage Clinical Trials for Success
  • Dalip Sethi, Ph.D. - Research & Development Senior Director, Cesca Therapeutics Inc
more
  • How is best to design the trial?
  • Do companies go for conditional or full approvals?
2:00pm - 2:30pm 30 mins
Commercialization
Strategies for Leveraging the Japanese Cell Therapy Opportunity: A Foreign Cell Therapy Company Perspective
  • R. Lee Buckler, B.Ed, J.D. - President and CEO, RepliCel Life Sciences, Inc.
more

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?

2:30pm - 3:00pm 30 mins
Commercialization
Practical Experiences of Registering Cell and Gene Therapy Products In Europe and Across Member States
  • Jacqueline Barry, Ph.D. - Chief Clinical Officer, Cell and Gene Therapy Catapult
more

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.

3:00pm - 3:30pm 30 mins
Commercialization
Please join other streams
2:00pm - 2:30pm 30 mins
Bioprocessing
Advances in CAR-T Cell Manufacturing
  • Isabelle Riviere, Ph.D. - Director, Michael G. Harris Cell Therapy and Cell Engineering Facility, Molecular Pharmacology, Memorial Sloan Kettering Cancer Center
more

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

2:30pm - 3:00pm 30 mins
Bioprocessing
Advanced Cellular Analytics of CD34 Drug Products
  • Ilya Shestopalov - Senior Scientist, Cell Analytics, bluebird bio, inc.
more

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.

3:00pm - 3:30pm 30 mins
Bioprocessing
Comparison of Adherent Cell Technologies for the Amplification of Virus Stocks and Viral Vectors
  • Steve Pincus, Ph.D. - Associate Vice President, Virology and Analytical Methods Development, Fujifilm Diosynth Biotechnologies
more

The implementation of adherent technologies can help overcome some of the challenges encountered during scale up and subsequent production of large scale high titer viral vectors. Achieving consistency and minimizing contamination risks will allow for increased productions which in turn will help de-bottleneck manufacturing operations.

2:45pm - 3:30pm 45 mins
Main agenda
Tools for Mapping and Repairing Disease States: Can We Make Therapeutic Invention into a Mature Design Science?
  • Ed Boyden, Ph.D. - Associate Professor, Media Lab and McGovern Institute Departments of Biological Engineering and Brain and Cognitive Sciences, Massachusetts Institute of Technology
more
4:00pm - 4:05pm 5 mins
Main agenda
Chairperson's Remarks
  • Travis McCready - President and CEO, Massachusetts Life Sciences Center
more
4:05pm - 4:45pm 40 mins
Main agenda
Genomics, Advanced Imaging and the Future of Medicine
  • J. Craig Venter, Ph.D. - Co-Founder, Executive Chairman, Head of Scientific Strategy, Human Longevity, Inc.
more
4:45pm - 5:15pm 30 mins
Main agenda
The Convergence Model of Cancer Research: Solving the Problems of Cancer through Interdisciplinary Approaches
  • Tyler Jacks, Ph.D. - Director, Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology
more

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.

5:15pm - 6:00pm 45 mins
Main agenda
BioPharma Executive Council Panel: Increasing Gender Diversity in BioPharma: What Works
more

Increasing Gender Diversity in BioPharma: What Works and What Is Working?

Moderator: Cissy Young, Executive Director, Russell Reynolds Associates

Panelists:

  • Susan Windham-Bannister, CEO, Biomedical Growth Strategies
  • Amri Johnson, Global Head of Diversity and Inclusion, Novartis Biomedical Research Institutes

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.

3:30pm - 4:15pm 45 mins
Main agenda
Refreshment Break in the BWB Poster & Exhibit Hall
6:00pm - 7:00pm 60 mins
Main agenda
Women in Bio/Women's Executive Leadership Dinner
7:00pm - 9:00pm 120 mins
Main agenda
BWB Social Event