Cell and Gene Therapy 2023

Matthew Porteus MD, PhD is the Sutardja Chuk Professor of Definitive and Curative Medicine and a Professor in the Department of Pediatrics, Institute of Stem Cell Biology and Regenerative Medicine and Maternal-Child Health Research Institute at Stanford. He is the Director of the Stanford Center for Definitive and Curative Medicine and the co-Executive Director of the Stanford Cell and Gene Therapy GMP Facility called the Laboratory for Cell and Gene Medicine. His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood (most notably sickle cell disease) but also of other organ systems as well. He received his undergraduate degree at Harvard in History and Science where his honors thesis studied the recombinant DNA controversy of the 1970s. He then completed his MD and PhD training at Stanford, clinical training in Pediatric Hematology/Oncology at Boston Children’s Hospital, and post-doctoral research training with Noble Laureate David Baltimore at CalTech. He 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 develop innovative curative therapies. He served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NexTRAC advisory committee evaluating the emergence of new technologies. He has been a scientific founder of CRISPR Tx and a founder of Graphite Bio and serves on several SAB’s. He is a strong advocate for assuring that the next generation of transformative medicines reaches the global community in partnership with those communities

Stephanie Cherqui is Professor in the Department of Pediatrics, Division of Genetics at the University of California San Diego. The main focus of her laboratory is the development of stem cell and gene therapy-based therapeutic approaches for genetic disorders, and the understanding of the mechanism of action for hematopoietic stem cell-mediated tissue repair. She established that hematopoietic stem and progenitor cell (HSPC) transplantation could reverse multi-organ degenerative disorders, even in the context of an intracellular membrane protein such as cystinosis, a lysosomal storage disorder. Her work led to the first in human HSPC gene therapy clinical trial for cystinosis. She is now applying this strategy to other disorders including the neuro-muscular degenerative disease, Friedreich’s ataxia. Dr. Cherqui received her Ph.D in 2002 at Necker hospital (Paris, France) and was appointed Assistant Professor at The Scripps Research Institute in 2009. She joined UC San Diego in 2012. She is the Chair of the Cystinosis Stem Cell and Gene Therapy Consortium, and a member of the American Society of Gene and Cell Therapy (ASCGT) Gene and Cell Therapy of Genetic and Metabolic Diseases committee. She is also a member of the Scientific Review Board of the Cystinosis Research Foundation and a Scientific Council member for the Cure Cystinosis International Registry (CCIR). Her research is mainly funded by grants from the National Institute of Health (NIH), California Institute of Regenerative Medicine (CIRM), the Cystinosis Research Foundation, and Friedreich’s ataxia Research Alliance (FARA).

Tracy Zhao is the West Coast Field Application Scientist at ACROBiosystems. She worked as a staff scientist in PerkinElmer for 3+ years, with rich experience in the development of IVD Immunoassay diagnostic kits. At ACROBiosystems, Tracy is working with leaders in both industry and academia to support their research needs. Tracy is part of a team that helps to guide the use and development of recombinant proteins that can be used for COVID-19, CAR T-cell therapy, as well as many other immune and auto-immune conditions. As a member of ACRO, she is highly interested in leveraging the company’s resources and state-of-the-art technology to help our partners and clients develop next-generation therapies.

Martin Michaelis received his Pharmacy Degree (Approbation, 1998) and his PhD (2001) from the Goethe-University, Frankfurt am Main, Germany. He then worked as a postdoc and later a deputy group leader in the research group of Professor Jindrich Cinatl at the Institute of Medical Virology (Goethe-University, Frankfurt am Main, Germany) and the Dr Petra Joh-Haus, a private research institute run by the Frankfurter Stiftung für krebskranke Kinder in Frankfurt am Main. In 2011, Martin joined the School of Biosciences at the University of Kent (Canterbury, UK) as Professor of Molecular Medicine). He runs a joint wet/ dry laboratory research group together with Mark Wass (Professor of Computational Biology at Kent).

Dr. Xiaowen Bai's research interests are centered around the application of stem cells for disease modeling and tissue regeneration. She has authored more than 80 research articles, reviews, and book chapters, all of which focus on stem cell-based tissue regeneration, disease modeling, and drug testing. Currently, her laboratory is primarily focused on investigating the molecular mechanisms underlying the roles of non-coding RNAs, mitochondria, and genetic factors in heart and brain injury in mice. To translate these findings to humans, Dr. Bai's lab utilizes human-induced pluripotent stem cell-derived tissue-specific cell culture and organoid models.

Dr. Appel is former chair of the Stanley H. Appel Department of Neurology at Houston Methodist Hospital in Houston, Texas. He is Director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics and the Peggy and Gary Edwards Distinguished Endowed Chair for the Treatment and Research of ALS at Houston Methodist Research Institute, and Professor of Neurology at Weill Cornell Medical College. He was previously Chair of the Department of Neurology at Baylor College of Medicine as well as Chief of the Neurology division and the James B. Duke Professor of Medicine at Duke University Medical Center, North Carolina. Dr. Appel serves as Chair of the Scientific Advisory Board for Coya Therapeutics, Inc. An internationally renowned researcher and neurologist, Dr. Appel is dedicated to the treatment of patients with ALS and is one of the nation's foremost experts on the disease. He has spent more than 40 years devoted to understanding the human brain. Dr. Appel is creator and director of what

Xi Lu is a senior scientist for cell and gene therapy at Bio-Techne. He oversees development for multiple stem cell and immune therapy products including media and matrices. Xi’s post-doctoral research works at Uppsala University and also UTH Health Science Center at Houston focused on biomaterials for tissue regeneration including bone and nerve. Xi received his Ph.D. in Biomedical Engineering from Washington University in St Louis where he developed microfluidic devices for investigating axonal degeneration. He holds a B.S. in Biomedical engineering from Georgia Tech.

Gabriella Perell is a Product Manager for Cell and Gene Therapy at Bio-Techne focused on immune and stem cell therapy tools and reagents. Gabriella previously developed scaled viral vector purification processes and viral clearance solutions within Process Development at Amgen. Gabriella obtained her doctorate from the University of Minnesota investigating protein-protein interactions through cross-linked peptides and fluorinated proteins, and holds a bachelors in Chemistry from the University of St. Thomas.

Dr. Yama A. Abassi received his undergraduate degree in biochemistry with Honors from SUNY/Stony Brook in May of 1992 and a PhD degree in Molecular, Cell and Developmental Biology from University of California at Santa Barbara in 1999. Dr. Abassi joined the Burnham Institute for Cancer Research as an NIH Post-Doctoral fellow in 1999 and performed his research on understanding the molecular and genetic basis of cancer. His research was published in several peer-reviewed and internationally recognized journals. Subsequent to post-doctoral training, Dr. Abassi joined the Biopharma industry to develop targeted therapies for cancer and work on personalized medicine. Dr. Abassi has also developed unique technologies for screening of novel drugs against cancer and other diseases. He has over 10 issued patent applications and has authored over 30 research publications. Dr. Abassi is currently working on developing novel technologies and methods for in process measurement and monitoring of cell therapies quality attributes and how such solutions can be used for process development and optimization as well as for manufacturing of donor samples.

Habib Malik is a Field Application Scientist at Bio-Techne supporting innovative analytical tools. He has held various positions over the past 10 years within Pharma/Biotech ranging from Quality Control Bio and Pharmaceutical Analysis to Field Service Engineering and Application Science, focusing on molecular biology and immunochemistry automated solutions. He holds a BSc (Honors) in Biomedical Science at the University of Bradford and an MSc in Cancer Pharmacology at the Institute of Cancer Therapeutics, Bradford.

Dr. Tamara Laskowski is the Head of Clinical & Process Development at Lonza-Personalized Medicine Business Unit. In her role, she supports the development of novel cell therapies targeting solid and haematological malignancies and the transition of these cell therapy processes from pre-clinical stage into clinical manufacturing. Moreover, Dr. Laskowski’s team oversees the implementation of enhanced analytics into process development, with the goal of identifying and monitoring critical attributes associated with product fitness and function. Dr. Laskowski received a dual doctorate degree in the fields of Human Molecular Genetics and Immunology from MD Anderson Cancer Center UTHealth where her work focused on targeted genome editing of patient stem cells to correct genetic mutations linked to immune disorders. Tamara has worked with leaders in CAR engineering, T and NK cell therapy development, and immunotherapy. Prior to joining Lonza, Tamara held a team leader role in the Immunotherapy Platform led by Dr. James Allison where her work focused on strategic industry partnerships and implementation of innovative immune-monitoring platforms to support clinical trials. Subsequently, Tamara joined the NK cell therapy team led by Dr. Katy Rezvani as a Scientific Director where she managed various pre-clinical and translational programs and led the integration of novel analytical platforms to support therapeutic target discovery and therapy development.

Nikolai Petrovsky is Research Director of Vaxine, an Adelaide-based biotechnology company focused on vaccine development and Professor of Medicine at Flinders University. He is a clinical endocrinologist and has a PhD in immunology. His work in vaccines and artificial intelligence has been awarded over 50 million dollars in grants from the US National Institutes of Health. He has authored over 200 peer-reviewed research papers and is an inventor on multiple vaccine patents. He developed SpikoGen®, a vaccine against COVID-19 that in October 2021 received emergency use authorization in the Middle east, making it the first recombinant spike protein vaccine against Covid-19 in the world to achieve regulatory approval and the first novel approved human vaccine developed in Australia since the Q fever vaccine 40 years ago.

Kevin Holden, PhD is Head of Science at Synthego in Redwood City, California. A senior member of a team responsible for developing platforms that deliver high-quality, scalable CRISPR genome engineering solutions, he helps to define Synthego’s scientific strategy and innovation through collaborative engagement with key opinion leaders in the genome engineering community. He has over 15 years of biotechnology experience with a focus in synthetic biology and genome engineering.