5th Annual World Congress: Realizing the Promises of Cell and Gene Therapy

A former FDA reviewer, Lauren Black is now a Distinguished Scientist at Charles River with more than 25 years of experience in drug development, focusing on accelerated translation to Phase I/II clinical trials.

She works mainly at the first in human (FIH) stage - in high risk diseases using novel products. Before Charles River, Dr. Black was a reviewing pharmacologist in the FDA’s CDER and CBER, during which time she assessed preclinical data, worked on many FDA guidances, and represented FDA externally on oligonucleotides, safety pharmacology, choice of relevant species, and utilization of PAD as the response index for setting human safety margins. She was a founding co-author of FDA’s Human Start Dose guidance. As a specialist in biologic therapies and targeted agents, she was elected to serve in BIO’s BioSafe committee in leadership roles, resulting in a white paper on selective uses of large animals for human risk assessment. She sits on several government, academic, and industry advisory panels discussing in vitro toxicology, portfolio prioritization, technology transfer/capitalization, and regulatory strategy. Dr. Black received her BS from Carnegie Mellon University and PhD in pharmacology from the Virginia Commonwealth University School of Medicine, where she researched GPCRs.

James C. Foster is Chairman of the Board, President, and Chief Executive Officer of Charles River Laboratories, a leading provider of essential products and services that fully support the drug discovery and early-stage development process. Since beginning his career at Charles River in 1976, Mr. Foster has held a variety of positions including: Vice President of Administration and General Counsel, President of Charles River Biotechnical Services, and Executive Vice President, Charles River. Mr. Foster was named President in 1991, Chief Executive Officer (CEO) in 1992, and Chairman in 2000.

As CEO, Mr. Foster has built Charles River from a niche research models and services business to a full service, early-stage drug research partner to more than 100 of the world’s largest biopharmaceutical companies and academic institutions, as well as thousands of emerging and established biotechnology companies. Today, the Company offers a unique portfolio of products and services required to take a novel drug therapy from discovery through non-clinical development.

Mr. Foster is a Senior Advisor at Cell Signaling Technology. He is Chairman of the Board of Trustees of the Institute of Contemporary Art in Boston, and a member of the Presidential Advisory Council for Berklee College of Music. He is a guest lecturer at the Massachusetts Institute of Technology (MIT).

Mr. Foster holds a B.A. degree from Lake Forest College, an M.S. degree from MIT, and a J.D. degree from Boston University School of Law.

Richard Horgan is the Founder and President of Cure Rare Disease. He has a deep passion for reimagining how rare and ultra-rare diseases are treated. With a younger sibling impacted by a rare disease, Rich has a strong interest in accelerating promising treatments for rare diseases. He formed an interdisciplinary collaboration of world-class researchers and clinicians to pioneer the rapid development of customized therapies for rare, genetic diseases. Prior to making his foray into biotech, Rich had extensive experience working in new business development at Corning Incorporated where he led the successful launch of a new product line. He also launched a successful car washing business in New York. He holds a BS from Cornell University where he graduated summa cum laude and an MBA from Harvard Business School where he was awarded the Blavatnik Fellowship for Life Science Entrepreneurship. Rich was recognized by Business Insider as one of ‘30 leaders under 40 transforming healthcare in 2020’ and, most recently, was named on the 2021 Forbes 30 under 30 list.

Peter Marks, M.D., PhD, is the Director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies. Dr. Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University. Following this, he completed an Internal Medicine residency and Hematology/Medical Oncology fellowship at Brigham and Women's Hospital in Boston, where he subsequently joined the attending staff as a clinician-scientist and eventually served as Clinical Director of Hematology.

He then moved on to work for several years in the pharmaceutical industry on the clinical development of hematology and oncology products prior to returning to academic medicine at Yale University where he led the Adult Leukemia Service and served as Chief Clinical Officer of Smilow Cancer Hospital. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016. Dr. Marks is board certified in internal medicine, hematology and medical oncology, and is a Fellow of the American College of Physicians.

Chris is a clinician-scientist with more than 25 years of cell and gene therapy experience spanning R&D, clinical medicine, bioprocessing and commercialization. He works to train the next generation of world-class scientists as a professor of cell and gene therapy at University College London and reviews cutting-edge research from around the world as senior editor of the journals Cell and Gene Therapy Insights and Regenerative Medicine. Chris was instrumental in the founding of the Alliance for Regenerative Medicine (ARM), the UK-Israel Science Council and the London Regenerative Medicine Network. He serves on the board of directors for Krystal Biotech and OriBiotech, and is a member of the scientific advisory board for several public and private companies, the UK Cell & Gene Therapy Catapult, and the Canadian Centre for the Commercialization of Regenerative Medicine. 

Kathrin C. Meyer, PhD, studied Cellular and Molecular Biology at the Institute of Cell Biology in Berne, Switzerland. Her post-doctoral research was performed in Brian Kaspar’s laboratory at the Center for Gene Therapy in Columbus Ohio. During that time, Dr. Meyer established a new and fast reprogramming method for in vitro modeling of neurodegenerative diseases using patient skin cells. Moreover, Dr. Meyer developed intrathecal gene therapy programs for several neurodegenerative diseases including Spinal Muscular Atrophy and Batten Disease. Multiple clinical trials that are based on this work are currently ongoing at Nationwide Children's Hospital. In 2017, Dr. Meyer became a Principal Investigator at Nationwide Children’s Hospital. She is also an Assistant Professor in the Department of Pediatrics at The Ohio State University, Columbus, Ohio.

Dr. Niss has served as Chief Technology Officer since March 2018. Dr. Niss joined Mustang in March 2017 as Vice President of Operations, where he initiated and oversees the establishment of Mustang’s cell therapy manufacturing facility. Prior to Mustang, Dr. Niss was Cell Therapy Asset Leader at Biogen, where he oversaw CMC-related activities for gene-edited hematopoietic stem cell and lentiviral gene therapy programs for sickle cell disease and hemophilia, respectively. Earlier in his career, Dr. Niss was Senior Technical Project Leader at Novartis’ cell therapy manufacturing facility in Morris Plains, New Jersey, where he directed the transfer and implementation of the CTL019 process from Penn to Novartis. He also served as Senior R&D Program Manager at EMD Millipore, where he established processes for the large-scale expansion of adult and pluripotent stem cells. Dr. Niss began his career in senior research positions in Pfizer’s Regenerative Medicine and Immunology groups. He holds a Ph.D. in molecular biology from Humboldt University of Berlin, and an M.S. in microbiology from the University of Göttingen in Germany. Dr. Niss completed his postdoctoral research at Boston Children’s Hospital and the Dana-Farber Cancer Institute.

Kenneth J. Olivier Jr., PhD, has been developing drugs for over 18 years and is currently serving as vice president of development at TScan Therapeutics, Inc. Dr. Olivier’s work experience includes biotechnology and pharmaceutical companies developing small molecule drugs, biologics, diagnostics, antibody drug conjugates, antibody directed nanotherapies, gene (AAV, LV vectors) and cell based therapies (TAA and TCR) for indications in cardiology, neurology, metabolic disease, neuromuscular disease, immunology, rare disease, infectious disease, fibrosis and oncology. Though his primary duties have been safety evaluations, Dr. Olivier has held the roles of project leader, head of project management, head of regulatory, head of pharmacokinetics, head of bioanalytical development, validation and sample analysis and head of nonclinical supporting multiple INDs, IMPDs, CTAs, NDAs, BLAs, PLEs and IDEs. Dr. Olivier obtained a BS in Toxicology from the University of Louisiana at Monroe and a PhD in Veterinary Biomedical Sciences from Oklahoma State University.

With a drive for continuous improvement and learning, Dr. Olivier is an adjunct faculty member at Oklahoma State University and has taught drug development courses at Harvard, MIT and through the American College of Toxicology, for which he is currently serving on the Editorial Board (International Journal of Toxicology) and formerly as an elected Council Member. Dr. Olivier has spent the past 15 years training CSOs, CEOs, CMOs, Project Team Leaders, Functional Heads and Team members on how to transition from discovery to development to market approval (within private and public companies), covering all topics related to drug development.

Glenn Pierce, M.D., Ph.D., has been interim chief scientific officer since May 2021 and a member of the board of directors since January 2017. Glenn currently serves as entrepreneur-in-residence at Third Rock Ventures. At Third Rock, he co-founded Ambys Medicines and serves as interim chief medical officer. Glenn previously served in a number of roles at Biogen, most recently as chief medical officer leading the hematology, cell and gene therapies division. Prior to Biogen, he served in small, large, public and private biotech/biopharma firms, including Bayer, Inspiration, Avigen, Selective Genetics and Amgen in the areas of tissue regeneration and hematology. Glenn is the co-author of more than 150 scientific papers and received more than 15 patents. He served on the medical and scientific advisory council, the board of directors and was president of the board of the National Hemophilia Foundation. Glenn also served on the Blood Products Advisory Committee at the FDA and the Committee on Blood Safety and Availability at the U.S. Department of Health and Human Services. He currently serves on the boards of directors of the World Federation of Hemophilia as the Vice President Medical and Global Blood Therapeutics. Dr. Pierce received a Doctor of Medicine and a Doctor of Philosophy in Immunology, both from Case Western Reserve University in Cleveland, and completed his postgraduate training in pathology and hematology research at Washington University in St. Louis.

Luk H. Vandenberghe, PhD, is an Associate Professor at Harvard Medical School and Associate Member of the Broad Institute of Harvard and MIT in Boston, MA, USA. 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 aims to gain a deeper understanding of mechanisms of gene transfer, to develop technologies to overcome hurdles to gene therapy clinical applications, and to translate specific gene therapy programs in vision, hearing, and other fields. Recent studies leverage structural and evolutionary information on AAV as a starting point for the rational 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 publication and more than a dozen licensed patents, mostly related to gene therapy methods, technologies, and applications.

Nate started his career in pharma working on computer systems validation for Amgen and quickly transitioned from validation into engineering change management, regulatory, data integrity, and other cGMP consulting. He later became involved in engineering capital project management overseeing large-scale capital investments.

Dr. Premsrirut is a co-founder and CEO of Mirimus, Inc., a company at the forefront of developing RNA interference and CRISPR gene editing technologies to further their potential for therapeutic applications. At the start of the COVID-19 pandemic, Dr. Premsrirut led her team in a call to action to address the lack of efficient testing methods that would be required to reopen our schools and economy and was named a winner in the XPRIZE Rapid COVID-19 Testing Competition for Mirimus’ innovative SalivaClear strategy. Dr. Premsrirut earned a B.A. in Molecular and Cell Biology and Biochemistry from UC Berkeley and joint M.D./Ph.D. from SUNY Stony Brook School of Medicine and Cold Spring Harbor Laboratory. She is a graduate of the Goldman Sachs Entrepreneurship program through Babson College. In addition, she has played an active role as a Board Member of NY BIO, a life science trade organization, and a member of the Board of Directors for BioBAT, a biotechnology incubator joint venture between SUNY Downstate and NYC Economic Development Center. She has been a vital force within the life science ecosystem in NYC, working to accelerate growth of this sector over the past decade.

Ananya Zutshi is a Blavatnik Fellow at Harvard Business School and Entrepreneur-in-Residence at Cellino, an autologous regenerative medicine company developing an AI-guided single-cell laser editing platform for cell-based therapies. Cellino has developed a scalable platform that automates and standardizes autologous stem cell production, accelerating the development of life-saving medicines for patients. Ananya is particularly focused on immuno-oncology, leveraging the Cellino platform to create best-in-class stem cell-derived immune cells specialized in eradicating cancer.

Prior to Cellino, she was a project manager at a leading contract development and manufacturing organization in the life sciences industry, managing project plans for clients focused on biologics. Ananya's experience in GMP Operations, Process Engineering, Business Development, and early-stage biology and technology investing has given her a holistic view of a complex industry. She has a background in biomedical engineering from Duke University, an MS in Engineering Sciences from the Harvard John A. Paulson School of Engineering and Applied Sciences, and an MBA from Harvard Business School, where she was a recipient of the Kaplan Life Sciences Fellowship and the MS/MBA Dupré Nunnelly Innovation Leader Award.

CureNFwithJack (“CNFWJ”) is a non-profit organization whose mission is to fund research to find an effective treatment or cure for neurofibromatosis (“NF”), a genetic disorder that causes tumors to grow anywhere along the nerves of the body. The inspiration and motivation behind CNFWJ is Jack Burke, a dynamic, inspiring boy who lives with NF. With Jack’s help, CNFWJ has encouraged thousands of people previously unfamiliar with NF to learn, donate and spread awareness about NF. It is CNFWJ’s hope that creating awareness, bringing people together, and supporting the medical community in its research endeavors will lead to the day that NF no longer exists.

Dr. Chuang works as the Director of Scientific Advisory Services providing scientific and regulatory expertise covering a variety of drug classes (small to large molecule), therapeutic areas, routes of administration, and animal species. He also manages multidisciplinary research teams for multiple drug development programs from target identification and drug discovery to preclinical safety programs.

Sam has a PhD in microbiology and immunology from the University of Tennessee, Health Science Center, and a Bachelor’s degree in biology from The University of Western Ontario. He is also a member of the American Society of Toxicology.

Matthew Hewitt, PhD, currently serves as Senior Director, Scientific Services, Cell and Gene Therapy (CGT) at Charles River Laboratories. Matt plays a critical role in driving CGT strategic vision as well as leading one of Charles River’s CGT sites.

Before joining the company, he was Head of R&D and Clinical Development for Lonza’s Personalized Medicine Business Unit where he led development of the Cocoon manufacturing technology, a closed, automated, scalable cell therapy manufacturing solution. In addition, he developed and executed numerous collaborations across academia and industry that leveraged the Cocoon technology.

Prior to Lonza, Matt led the Tumor Immunology and Microenvironment program at Bellicum Pharmaceuticals, which focused on improving cell therapy efficacy in solid tumors. He also led the Immunology group at the University of Pennsylvania’s Gene Therapy Program, leading and contributing to numerous AAV gene therapy programs.

Matt received his PhD in Biophysics and Physiology from the University of Alabama at Birmingham and completed his postdoctoral fellowship at Johns Hopkins University.

With over 20 years of pharmaceutical industry experience Dan has been associated with the commercialization of countless brand name pharmaceutical products spanning multiple therapeutic areas working primarily in marketing and sales functions. His greatest area of experience and expertise is in diseases of the Central Nervous System. With a diverse and well-anchored background in mass communications, his core passion revolves around innovation, strategic development and communications. Dan holds a Journalism degree from Carleton University, Ottawa, Canada and spent time working as a journalist for The Canadian Press prior to making the transition into the pharmaceutical industry via medical publishing. He is Past President of the Pharmaceutical Marketing Club of Québec (2010-2012) and was inducted into the Canadian Healthcare Marketing Hall of Fame in 2011.