Teach Me in 10: Accelerating Cell and Gene Therapy Development

Dr. Saba Ghassemi is an Assistant Professor in the Department of Pathology and Laboratory Medicine and serves as a Principal Investigator at the Center for Cellular Immunotherapies at the University of Pennsylvania. Her research focuses on developing novel CAR T cells with enhanced potency through a multidisciplinary approach that integrates engineering with CAR T cell immunology. Dr. Ghassemi pioneered the development of an abbreviated culture paradigm, resulting in less differentiated progeny with improved potency. This breakthrough led to a successful clinical trial at UPenn utilizing a 3-day manufacturing process. Additionally, she has devised a novel method for delivering CAR transgenes into quiescent T cells, enabling the generation of highly potent CAR T cells within 24 hours. Dr. Ghassemi is currently spearheading efforts to transition this innovative platform into an upcoming clinical trial at Penn. Her groundbreaking work has garnered recognition through several research grants and patent applications aimed at enhancing CAR T cells' efficacy, expansion, and fitness for adoptive immunotherapy. Dr. Ghassemi's ongoing research endeavors focus on optimizing, streamlining, and automating the manufacturing process of CAR T cells, with the overarching goal of enhancing the translational applicability and accessibility of these novel therapies to a broader range of geographical locations and patient populations.

Dr. Romero received her Ph.D. from the University Granada (Spain) in 2009 and completed her postdoctoral fellowship work with Dr. Donald Kohn in 2015 at UCLA. During her postdoctoral fellowship she trained in experimental hematology, viral vectors, gene editing methods, and GMP cell processing. She is currently an Assistant Adjunct Professor in the Department of Microbiology, Immunology and Molecular Genetics at UCLA. Currently her central research work aims enhancing gene editing by investigating efficacy and toxicity from the editing protocols; studying the relationship between HSC cell cycle status and gene editing outcomes and engraftment of the long-term HSC in different immunodeficient mouse models; and translating the research-scale methods of editing to GMP-compatible, clinical scale manufacturing. While the field of Gene Editing of HSC is constantly advancing, it is her interest to study new gene editing method, such as Base Editing of DNA to correct monogenic disorders; and RNA Base editing to transiently inhibit the transcripts of genes implicated the DNA damage response pathway triggered after gene editing to improve long-term engraftment of the edited HSPC.

Dr. Tianfu Zhang graduated from Nanjing University with his PhD in biology. After working in over 10 years in CGT-related research, production, and quality management, he has an extensive background in a wide variety of cell therapies, including DC, CAR-T, TCR-T, iPSC, and gene therapies. He has published over 7 SCI-level papers with over 20+ patents. Dr. Zhang is currently responsible for the R&D of CGT Reagents for Cell Therapy Manufacturing, focusing on process and quality control challenges.

Tia Harmon is a Cell and Gene Therapy, Technical Product Sales Specialist for PHC Corporation of North America (PHCNA). She is committed to providing solutions for cell metabolism research that aid in the development of disease treatments. Tia has worked in the industry for 20 years in the government, academic, and biotechnology sectors. She holds a PhD in Biomedical Sciences from Morehouse School of Medicine. Tia is passionate about expanding knowledge surrounding Cell and Gene Therapy. In her spare time, she enjoys taking in the beautiful sights that Virginia has to offer. If you would like to learn more about the Cell and Gene Therapy portfolio offered by PHCNA, please feel free to email her at Tia.Harmon@us.phchd.com.

Matt is a Senior Applications Scientist in the Market Development group at Refeyn. He has over 10 years of industry experience in small-molecule drug discovery and developing gene editing technologies. As a protein scientist, he has worked with various classes of proteins including GPCRs, enzymes, multimeric complexes and ribonucleoprotein complexes. Matt holds a PhD in Biochemistry from the University of Connecticut, where he studied the application of photoactive proteins in biophotonic devices.

Dr. Sabrina Solt is a naturopathic doctor, entrepreneur, and content creator, specializing in anti-aging, biohacking, and regenerative medicine. With over a decade of experience, she is the founder of Stem Cell Therapy Professionals and has worked with thousands of patients, including Olympic medalists, CEOs, and actors. A member of the International Stem Cell Clinician Association, Dr. Solt has been featured in numerous podcasts, documentaries, and health summits. She is a sought-after speaker, having addressed audiences at the International Cell Surgical Conference on topics like stem cell therapy and hormone optimization. Through her practice and educational outreach, Dr. Solt continues to empower people to unlock their health potential and lead vibrant, long-lasting lives.

Dr. Lore Gruenbaum joined LLS in 2020 as the Vice President for the Therapy Acceleration Program (TAP), the LLS venture philanthropy program aiming to accelerate high-risk, innovative blood cancer therapeutics. She brings more than 20 years of drug discovery and clinical development experience to LLS working on small molecules, biologics, RNA-directed and cell therapies in multiple therapeutic areas including oncology, immunology, virology and neuroscience. Prior to joining LLS, Dr. Gruenbaum was employed as the Vice President, Biology at Gotham Therapeutics pioneering drug discovery in Epitranscriptomics. She was part of the executive leadership team at Applied Biomath where she led collaborative mechanistic modeling projects to accelerate and de-risk drug development for biotech and pharma and acted as the principal investigator on a NIH small business grant. Dr. Gruenbaum held roles of increasing responsibility at Boehringer Ingelheim and Roche Pharmaceuticals driving technical innovation, predictive toxicology, novel mechanistic and biomarker strategies for drug discovery and clinical projects. She completed her Ph.D. at Free University of Berlin with ‘summa cum laude’ and conducted postdoctoral studies at Yale University.

Dr. Philipp Nold began his journey in the world of biology at Ruhr-University Bochum, Germany, and furthered his education with a Diploma degree focusing on hematopoietic stem cell niches in microbioreactors. His groundbreaking work during his PhD at the University Hospital Giessen and Marburg on standardizing the cultivation of mesenchymal stem cells (MSCs) under Good Manufacturing Practice (GMP) conditions led to a successful application for a manufacturing authorization for these cells as therapeutic agents. Dr. Philipp Nold has contributed several pivotal scientific articles to the field, including a 2013 publication on GMP-compliant stem cell expansion. His role at the life science company Eppendorf SE as an Infield Application Specialist involved extensive customer support through trainings, sharing his expertise in stem cell bioprocessing, and conducting webinars and presentations. This role was crucial in facilitating the practical application of stem cell research in various settings. Since October 2023, as the Business Development Manager CGT (Cell and Gene Therapy) Europe, Dr. Philipp Nold has been responsible for identifying market opportunities, developing strategies to enhance the company's position in the CGT sector, and collaborating with various stakeholders to promote and implement innovative cell and gene therapy solutions. His work involves bridging the gap between scientific innovation and market needs, ensuring the advancement of cell and gene therapies across Europe.

Brian Cosgrove is a Principal Scientist at Tune Therapeutics and the research lead for the Hepatitis B program. Prior to his time at Tune Therapeutics, Brian was a postdoctoral fellow in the laboratory of Dr. Charles Gersbach at Duke University, where he pioneered the use of novel epigenetic editing approaches toward gene regulation and genomic screening for cellularengineering. Brian received his Ph.D. in bioengineering in 2017 from the University of Pennsylvania in the lab of Dr. Robert Mauck, and his B.S. in bioengineering in 2011 from Syracuse University. In his Ph.D. work, Brian developed novel biomaterial approaches to study and control stem cell differentiation.

Dr. Ryan Yi H Nai is a Senior Scientist in Bioanalytical Sciences at CSL, where he leads the development and implementation of capillary electrophoresis-based assays for the characterization and quality control of recombinant monoclonal antibodies, fusion proteins, and mRNA vaccines. With over a decade of combined experience in the biopharmaceutical industry and academia, Dr Nai has a strong track record and drive in delivering high-quality results in interdisciplinary research fields spanning analytical chemistry, molecular biology, and microbiology. Dr. Nai earned his Ph.D. in Analytical Chemistry from the University of Tasmania under the supervision of Professor Michael C. Breadmore. His doctoral research focused on the capillary electrophoresis of ribosomal RNA for microbial community characterization. At CSL, Dr. Nai provides technical leadership in CE techniques, including the SCIEX PA 800 Plus system, iCIEF, and microchip-based platforms and a pivotal role in facilitating cross-functional collaboration and knowledge transfer within CSL, advocating for advanced CE methodologies. His expertise extends to recombinant therapeutic product development, and RNA related technologies, and drug development.

As CEO of ERS Genomics, John has over 25 years’ experience as an executive, board member, venture capital investor, and investment banker focused on the life sciences and MedTech sectors. As CEO of Quanta Dialysis Technologies for nearly a decade, John led the development and commercial launch of an award-winning portable hemodialysis system designed to transform the delivery of kidney care to patients. He drove market entry into the UK and US, while raising a record-breaking $245 million private funding round and clinching the prestigious MacRobert Award for engineering excellence. Prior to this, he served as CFO of Nitec Pharma, securing a pivotal funding round and key partnerships that transitioned the company from development to commercial stage. John is also an experienced venture investor, with previous roles as Partner and co-Head of Healthcare Ventures at Downing LLP and Investment Director at NBGI Ventures. Before that, he worked at Atlas Venture and Kirkland Investors. Successful exited investments include Symetis, ACT/Epix, BoneSupport, and WebMD. John is a graduate of the University of Chicago and previously served on the i4i selection panel for translational research at the National Institute for Health and Care Research (NIHR). He currently serves as an independent Board member at Otivio AS and is a mentor at the Enterprise Hub of the Royal Academy of Engineering. John was named in the Sunday Times Maserati List as one of the UK’s “Top 100 Game Changing Innovators and Entrepreneurs.”