2018 Schedule of Events
November 6th, 2018
8:00am · Michael Chambers
President and CEO, Aldevron
Opening RemarksRead Bio
Michael Chambers has served as President and CEO of Aldevron since founding the company in 1998 as an undergraduate at North Dakota State University (NDSU) in Fargo. He and fellow student John Ballantyne started producing plasmid DNA in a small lab at NDSU for use by researchers in the biotech community.
Over the next 20 years, they expanded and grew the business in Fargo, North Dakota, to become one of the industry’s leading — and most respected — contract manufacturing organizations. Aldevron products and services are key components of the world’s most innovative gene and cell therapy commercial products.
This year, Michael was named one of the “100 Most Intriguing Entrepreneurs” by Goldman Sachs and honored at its “2018 Builders and Innovators Summit” in October.
Under his vision and leadership, Aldevron acquired Genovac Antibody Technology of Freiburg, Germany, in 2004. Five years later, the company expanded into protein production in Madison, Wisconsin. Recently, the company completed construction of a 70,000 square-foot building in Fargo, the largest plasmid DNA manufacturing facility in the world. Michael continues to lead the Aldevron team of more than 200 on the next phase of its growth trajectory.
Michael received Bachelor of Science degrees in biotechnology, chemistry and microbiology from North Dakota State University.
8:20am · Laurence J.N. Cooper, M.D., Ph.D.
Chief Executive Officer, ZIOPHARM
Genetic engineering: T-cells from bedside to bench to boardroomRead Bio
Prior to becoming the Chief Executive Officer of ZIOPHARM in May 2015, Dr. Laurence Cooper was a tenured Professor (early/exceptional promotion) at The University of Texas MD Anderson Cancer Center (MDACC), with joint appointments in the Division of Pediatrics and Department of Immunology. He also served as Section Chief of Cell Therapy at the Children’s Cancer Hospital at MDACC where, as a Visiting Scientist at MDACC, he will continue to lead scientific efforts to develop new treatment approaches which pair genetic engineering with immunotherapies.
287Dr. Cooper has coauthored dozens of peer-reviewed journal articles, abstracts, and book chapters. He has initiated multiple trials under INDs infusing T cells and NK cells. He is undertaking the first protocols using a new approach to gene therapy based upon the Sleeping Beauty transposon/transpoase system and has helped develop clinical-grade artificial antigen presenting cells for numerically expanding and activating lymphocytes. Dr. Cooper obtained his M.D. and Ph.D. degrees at Case Western Reserve University in Cleveland and then training in Pediatric Oncology and Bone Marrow Transplantation at the Fred Hutchinson Cancer Research Center in Seattle.
9:00am · Matthew Porteus, M.D., Ph.D.
Associate Professor, Department of Pediatrics, the Institute of Stem Cell Biology and Regenerative Medicine; Child Health Research Institute at Stanford
Translating Genome Editing of Somatic Stem Cells to the Clinic: Using the Best ToolsRead Bio
Matthew Porteus M.D., Ph.D. is an Associate Professor in the Department of Pediatrics, the Institute of Stem Cell Biology and Regenerative Medicine, and the Child Health Research Institute at Stanford. He received his B.A. from Harvard University in “History and Science” and his M.D. and Ph.D. degrees at Stanford University. He completed his post-doctoral medical training in Pediatrics and Pediatric Hematology/Oncology at Boston Children’s Hospital and the Dana Farber Cancer Institute, and his post-doctoral research training under the mentorship of Dr. David Baltimore.
His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood but also of other organ systems as well. His research program has made important discoveries in advancing the field of genome editing including the first use of genome editing using engineered nucleases in human cells and optimizing the use of the CRISPR/Cas9 system in primary human stem cells.
He also works as an attending physician on the Pediatric Hematopoietic Stem Cell Transplant service at Lucile Packard Children’s Hospital where he cares for children under going bone marrow transplantation for both malignant and non-malignant diseases. His goal is to combine his research and clinical interests to bring innovative curative therapies to patients. He served on the National Academy Study Committee of Human Genome Editing and as a History and Science major at Harvard he wrote his undergraduate thesis on the social interpretation of the recombinant DNA controversy in the early 1970s.
9:40am · Luk H. Vandenberghe, Ph.D.
Assistant Professor at Harvard Medical School; Associate Member of the Broad Institute of Harvard and MIT
Broadening the reach of gene therapy by discovery and collaboration in a competitive spaceRead Bio
Luk H. Vandenberghe, Ph.D., is an Assistant Professor at Harvard Medical School and and Associate Member of the Broad Institute of Harvard and MIT in Boston, MA. He directs the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear Infirmary in Boston, USA, a part of the Ocular Genomics Institute, a bench to bedside research program to study, diagnose, and develop treatments for diseases of the eye. He received a degree in cellular and genetic engineering from the University of Leuven, Belgium. His previous work led to the discovery of novel AAV serotypes such as AAV9, novel insights into AAV structure-function, and vector immunobiology.
His laboratory at Harvard addresses mechanistic questions on AAV virology, develops technologies aiming to overcome hurdles to gene therapy clinical applications, and actively translates gene therapy programs in hearing and vision. His research focuses on delivery questions, specifically on the adeno-associated virus (AAV) for therapeutic gene delivery. Recent studies leverage structural and evolutionary information on AAV as a starting point for the design of synthetic viral vector systems, a first generation of which is referred to as AncAAVs which are now progressing to the clinic for a number of indications.
Dr. Vandenberghe previously co-founded GenSight Biologics and Akouos. He also is a founder, board member, and advisor to Odylia Therapeutics, a non-profit catalyzing translation for gene therapies within the challenging field of ultra-rare disorders. Dr. Vandenberghe has over 50 peer reviewed publications and more than a dozen licensed patents, mostly related to gene therapy methods, technologies, and applications.
10:40am · Krishanu Saha, Ph.D.
Assistant Professor, Department of Biomedical Engineering, University of Wisconsin-Madison
Precise gene editing of mutant alleles using CRISPR-Cas9 ribonucleoprotein complexes
Krishanu Saha is an Assistant Professor in the Department of Biomedical Engineering at the University of Wisconsin-Madison. He is also a member of the Wisconsin Institute for Discovery, Carbone Cancer Center, and Stem Cell and Regenerative Medicine Center as well as the National Academies’ Forum on Regenerative Medicine.
Prior to his arrival in Madison, Dr. Saha studied Chemical Engineering at Cornell University and the University of California in Berkeley. He was a Society in Science: Branco-Weiss fellow at the Whitehead Institute for Biomedical Research at MIT, and in the Science and Technology Studies program at Harvard University. Major thrusts of his lab involve gene editing and cell engineering of human cells found in the retina, central nervous system and blood.
11:20am · Christopher Vakulskas, Ph.D.
Senior Staff Scientist, Integrated DNA Technologies
Discovery and characterization of novel SpCas9 and AsCas12a mutants isolated by protein engineering and directed evolution
Dr. Christopher Vakulskas, Ph.D. is a senior staff scientist in the molecular genetics research division of Integrated DNA Technologies. He has a Ph.D. in microbiology from the University of Iowa, where he studied genetic regulatory circuits in pathogenic bacterial species. He was an NIH Postdoctoral Fellow at the University of Florida where he studied RNA binding proteins and post-transcriptional gene regulation.
At IDT, Dr. Vakulskas has been involved in managing contract research services, leading process development for CRISPR protein purification and evolving novel CRISPR proteins with useful functions for commercialization. He conceived and executed a screen that led to the creation of a high-fidelity Cas9 mutant that was recently used to perform therapeutic gene editing in a recent issue of Nature Medicine.
12:00pm · Robert Whalen, D.Sc.
Chief Scientific Officer, Altravax; Maxygen
Directed Evolution by Molecular Breeding
Robert Whalen is Chief Scientific Officer at Altravax (since 2010) and more recently at Maxygen (since 2017), both in Sunnyvale, California. He oversees multiple programs applying molecular breeding to the creation of novel products in many areas of biotechnology from enzymology to therapeutics and vaccines.
Previously, Whalen was Director of Infectious Diseases at the original Maxygen (1999-2009), where he began to apply the molecular-breeding directed evolution approach to vaccine discovery. Prior to entering the biotechnology industry in 1999, he spent 27 years in France as a staff scientist and group leader at the Pasteur Institute in Paris. Much of his early work was centered on studies of muscle development. He was an early contributor to the development of DNA vaccines and has published a number of articles in that field.
In 1995, he created The DNA Vaccine Web site (now DNAvaccine.com) and served as a consultant to several biotechnology companies. From 1986-1996 he was a member of the Scientific Advisory Board of the French Muscular Dystrophy Group. He is the author of some 120 scientific papers, reviews, and book chapters in the fields of biochemistry, gene expression, developmental biology, molecular physiology, gene transfer, vaccinology, immunology, and directed evolution.
Robert Whalen obtained degrees from the Pennsylvania State University (B.S., 1970), Harvard University (A.M., 1974), and the French National Center for Scientific Research (D.Sc., 1991).
1:40pm · David Courtman, Ph.D.
Scientist and Director of Biotherapeutics at the Ottawa Hospital Research Institute; Assistant Professor at the University of Ottawa; Chief Scientific Officer of Northern Therapeutics
Translation of Cell Based Gene Therapies for CARDIO-Pulmonary Disorders
Dr. Courtman is trained in cellular and molecular pathology and biophysics with, extensive expertise in the development of novel therapies for cardiovascular disease. He has a long standing interest in the development of novel cardiovascular biomaterials and the regulation of biological responses within the unique spatially-oriented matricellular environment of blood vessels.
Dr.Courtman has participated in the development of a number of cellular-based gene therapies for the treatment of cardiopulmonary disorders taking them from initial concept, through appropriate animal disease models, and into first-in-human clinical trials. He is currently directing the manufacturing of these cell products for trials treating patients suffering from pulmonary arterial hypertension, myocardial infarction, and sepsis.
He is a Scientist and Director of Biotherapeutics at the Ottawa Hospital Research Institute, Assistant Professor at the University of Ottawa, and Chief Scientific Officer of Northern Therapeutics.
2:20pm · Palani Palaniappan, Ph.D.
Head of Technical Operations, Sarepta Therapeutics
Microdystrophin gene therapy for musculoskeletal diseases
Palani Palaniappan, Ph.D., has more than 25 years of pharmaceutical experience and continues to contribute to commercial registration and clinical development of numerous candidates in small molecule, biologics, ADCs and cell therapy. He serves as the head of Technical Operations at Sarepta Therapeutics, and focuses on small molecule and antisense based RNA medicines. He leads Sarepta’s gene therapy CMC and Manufacturing team and is responsible for all stages of development from research through commercial.
Prior to Sarepta, Palaniappan held increasing leadership positions and recently lead biologics and new modality development at Takeda Pharmaceuticals with responsibility for global development of drug candidates in variety of modalities including cell and gene therapy, microbiomes and antibody drug conjugates. Before Takeda, he spent a number of years each at Millennium Pharmaceuticals, Biogen, Gilead Sciences and Par Pharmaceuticals. He completed executive leadership education at Oxford University and Insead, post-doctoral work athe t University of California Riverside and Virginia Commonwealth University Richmond. He received his Ph.D. from the Indian Institute of Technology Kanpur.
3:00pm · Mark Osborn, Ph.D.
Assistant Professor, University of Minnesota
Translational Gene and Cellular Engineering
Dr. Mark Osborn is an Assistant Professor at the University of Minnesota in the Department of Pediatrics and the Division of Blood and Marrow Transplantation. He is a member of the Masonic Cancer Center, the Stem Cell Institute, The Institute for Engineering in Medicine, and the Center for Genome Engineering. His laboratory focuses on gene and cell therapy for inherited and acquired lethal pediatric disorders and applies the latest methodologies in gene therapy and genome engineering.
November 7th, 2018
7:00am · Breakfast and Networking
8:20am · Andrew Moreo
Operations Manager, GMP and Viral Vector Core Facilities, Nationwide Children’s Hospital (NCH)
Challenges in Manufacturing AAV in a Phase I Environment: A practical approach to meeting demand and managing expectation in an evolving field.
Andrew Moreo serves as Operations Manager for the GMP and Viral Vector Core Facilities at Nationwide Children’s Hospital (NCH) in Columbus, Ohio.
Mr. Moreo has a B.S. in Biology from Purdue University and nearly two decades of experience in genetic and molecular research, with the last 11 years focused on AAV production. During his time at NCH, Andrew has led the development of an adherent AAV production platform that is robust, adaptable and high-yielding across a wide array of serotypes.
This platform has delivered over 1.5×1017 viral particles in support of 40+ Phase 1 INDs. He also led in the expansion of the GMP facility from a single product legacy space to a new multi-product facility capable of handling up to four independent products simultaneously.
In his role, Moreo continues to lend his expertise in the development of new vector production platforms as well as the transfer of existing technologies to clients wishing to pursue advanced phase production.
9:00am · Anne Kantardjieff, Ph.D.
Director of Plasmids and Small Molecules Manufacturing, bluebird bio
Manufacturing considerations in cell therapy development
Anne Kantardjieff serves as Director of Plasmids and Small Molecules Manufacturing at bluebird bio. Prior to that, she was the Director of Early Stage Process Development at Alexion Pharmaceuticals, where her team was responsible for cell line development as well as upstream and downstream process development for Alexion’s early clinical biologics portfolio.
Kantardjieff is the recipient of the 2018 ACS BIOT Van Lanen award, recognizing her service to the Biotechnology Division of the American Chemical Society division, where she also serves as secretary. She obtained her Ph.D. in Chemical Engineering from the University of Minnesota, and her B.Eng. in Chemical Engineering from McGill University in Montreal, Quebec, Canada.
9:40am · Maritza C. McIntyre, Ph.D.
President, Advanced Therapies Partners, LLC
Standards development in gene and cell therapy
Dr. McIntyre has 20 years of experience in the development, evaluation and regulation of biological and small molecule products within startup biotech firms, the Food and Drug Administration (FDA), and as a consultant. Dr. McIntyre was a product reviewer and ultimately Branch Chief in the Division of Cellular and Gene Therapies at FDA/CBER, where she was actively involved policy development and liaison activities to stakeholder groups. She has since worked in regulatory affairs and product development at Bavarian Nordic, REGENXBIO, Inc. and NanoCor Therapeutics. She served as Executive Vice President of Regulatory Affairs and Product Development at Bamboo Therapeutics where, as part of the senior management team, she participated in portfolio selection, product development and fundraising that resulted in an initial $50 million finance round and ultimate the sale of the company to Pfizer.
As president of Advanced Therapies Partners LLC, Dr. McIntyre provides strategic regulatory and product development advice to biotech companies, academics, and venture capital firms. She has proven success in defining development strategies for products with complex regulatory challenges including special designations (orphan, RMAT, pediatric orphan drug designation), endpoint selection, accelerated approval, complete response letters and dispute resolution. She has also been involved in the preparation of some of the first BLA and MAA submissions for gene therapy products to FDA and EMA. She has multidisciplinary experience, including chemistry manufacturing and control (CMC), preclinical, and clinical with a wide range of product types, including novel gene and cell therapy products, vaccines, biological products and small molecules at varied stages of product development.
Through her participation in industry associations, including ASGCT and the Standards Coordinating Body she has continued to contribute to gene therapy regulatory policy development.
Dr. McIntyre received a Ph.D. in virology from the University of Chicago and graduated magna cum laude with an Honors B.S. in biology from Wayne State University.
10:40am · Ted Tisch
Chief Operating Officer, Synthego
Ted Tisch serves as COO of Synthego. He joined Synthego when it had 20 people and where he is now responsible for product development, operations ands commercial functions, as the company develops full-stack engineering platforms delivering innovative genome engineering solutions.
Before joining Synthego, Ted spent 25 years in at Bio-Rad Laboratories developing leading reagent, instruments and software products in Research & Development, running Reagent Operations and serving six years as Vice President and General Manager at Bio-Rad Laboratories of the Protein Function Division (>$200M revenue).
Ted holds a Master of Science in Organometallic Chemistry from the University of Nevada, Reno and a Bachelor of Science in Inorganic Chemistry from Fort Lewis College.
11:20am · Theresa M. Reineke, Ph.D.
Distinguished McKnight University Professor, Department of Chemistry, University of Minnesota
Nonviral Synthetic Polymers Promote Cellular and In Vivo Delivery of Therapeutic Genes and Editing SystemsRead Bio
Theresa M. Reineke is a Distinguished McKnight University Professor in the Department of Chemistry at the University of Minnesota. She also holds graduate faculty appointments in the Departments of Chemical Engineering/Materials Science and Pharmaceutics. Her educational background includes receiving a Bachelor of Science from the University of Wisconsin-Eau Claire, a Master of Science from Arizona State University, and a Ph.D. from The University of Michigan. She then received a National Institutes of Health Postdoctoral Fellowship to further her research background at the California Institute of Technology prior to beginning her independent faculty career.
Her research group is currently focused on enabling fundamental and applied technology advancements of polymers in the fields of gene and cell therapy, oral drug delivery, and sustainability for which she has published more than 110 peer-reviewed manuscripts and received several awards such as the American Society of Gene and Cell Therapy Outstanding Investigator Award, the National Institutes of Health Director’s New Innovator Award, and the American Chemical Society Division of Polymer Chemistry Carl S. Marvel Creative Polymer Chemistry Award.
Reineke is also a founding Associate Editor of ACS MacroLetters and currently on the Editorial Advisory Boards of the ACS journals Biomacromolecules and Bioconjugate Chemistry.
12:00pm · James Brown, Ph.D.
Vice President of Corporate Development, Aldevron
Plasmid DNA in Cell and Gene Therapy: From Bench to BedsideRead Bio
James Brown, Ph.D., joined Aldevron in 2015 and serves as Vice President of Corporate Development. His responsibilities include developing and implementing strategies for expanding Aldevron’s DNA, mRNA, antibody, and protein products and production services. In this capacity Dr. Brown heads the product management team, which expands existing product features and develops new products, including pALD-X80, an off-the-shelf rAAV helper plasmid.
Prior to joining Aldevron, Dr. Brown was Vice President, Technical Operations, at REGENXBIO Inc., a gene therapy company. In this role he was responsible for contract manufacturing, vendor management, operations, quality assurance and biological reagent sales. Prior to REGENXBIO Dr. Brown served in roles of increasing responsibility in operations and quality assurance at MedImmune, Meso Scale Discovery and IGEN International, Inc. Dr. Brown holds a Ph.D. in chemistry from Stanford University and a B.S. in chemistry from Butler University.
2:10pm · Michelle Berg
Vice President, Patient Affairs & Community Engagement, Abeona Therapeutics
Coming of Age Together: Rare Disease Advocacy and Gene TherapyRead Bio
Michelle Berg joined Abeona in June of 2015, serving initially as Vice President, Patient Advocacy and transitioning in late 2017 to Vice President, Patient Affairs & Community Engagement to better reflect departmental focus. As lead for the Patient Affairs department, Michelle’s role encompasses development and provision of education and information on rare disease, drug development, gene therapy, and advocating for change and awareness for the impact on communities served specifically through Abeona’s pipeline programs. Berg is a contributing author and speaker on patient focused programming and rare disease advocacy.
Previously, she was the first hire with Aldevron, LLC, now a leading global contract manufacturing organization specializing in biological reagents for R&D through clinical trial use. During her time there, she held multiple positions with increasing responsibility, contributing to the growth and reach of the organization, focused on developing strong relationships, collaborations, and services. Additionally, she performed research on behalf of the Department of Plant Sciences, North Dakota State University. Her B.S. in Biotechnology was attained from North Dakota State University in 1997. She brings over 17 years of experience in the gene and cell therapy, vaccine, gene editing and molecular diagnostic fields.
2:50pm · Robert (Bob) W. Levis
A patient's perspective
An early experimental trial participant at Penn Medicine for Dr. Carl June’s CAR-T therapy.Read Bio
After receiving a Bachelor of Science degree in metallurgical engineering from Grove City College and a Master of Science degree in materials science and physics from the University of Illinois, Bob joined Air Products & Chemicals, Inc., in 1975. He held various positions as an applications development engineer at the Allentown, Pennsylvania, corporate headquarters.
In 1979 he was assigned to Brazil as manager of Applied R&D, with Air Products Gases Industriais Ltd. The four-year experience in Brazil led to other overseas business assignments including five years each in Japan and Taiwan, where he managed Air Products’ gases and equipment business through joint venture companies in these countries.
In June 1999, Bob was named Vice President & General Manager for Air Products Asia where he oversaw the company’s gases and equipment business in Southeast Asia. This executive responsibility was expanded in 2002 as V.P. & General Manager for the company’s largest division in Asia, based in Singapore for a total of eight years.
Bob is currently consulting through Asia & Brazil Connections, LLC with small and medium sized U.S. enterprises on Asia and Brazil market entry opportunities including ABEC, Inc (biopharmaceutical engineering and equipment manufacturer) where he was employed during 2011/2012 as Director, Asia Business Development.
Bob was diagnosed with Chronic Lymphocytic Leukemia (CLL) in 2002. He continued to “watch & wait” until 2008 when he moved back to the U.S. and underwent FCR (fludarabine, cytoxan, rituxan) treatment for his CLL. He maintained a complete remission for 3+ years when his CLL relapsed to the most aggressive 17p del form. Bob enrolled in a Pharmacyclics trial (ibrutinib vs. ofatumumab) at Penn Medicine in Philadelphia, and was randomized to the ofatumumab arm. The ofatumumab treatment did not stop the progression of his CLL and he was denied crossover access to ibrutinib.
Facing death while alive on hemoglobin and platelet transfusions, he enrolled as one of the early experimental trial participants at Penn Medicine for Dr. Carl June’s CAR-T therapy. He received his genetically modified T-cells on March 12, 2013 and was in complete remission for another 3+ years. A second CAR-T therapy during 2017 at Penn Medicine reduced the CLL in his bone marrow to < 5.0 %. Now Bob maintains normal blood labs while on the oral kinase inhibitor, ibrutinib.
Bob and Carl June have become friends helping others to overcome their blood cancers. Bob remains an avid sportsman and competitor while consulting on patient advocacy initiatives. Google “Bob Levis Leukemia” for various media coverage of his CAR-T experience.
3:00pm · Tom Whitehead
Co-Founder, Emily Whitehead Foundation
Emily Whitehead’s journey to CAR-TRead Bio