Gene Editing for Immune Cell Therapies

ABOUT THE WEBINAR

Gene editing has emerged as a transformative tool for developing immune cell therapies, particularly in the context of cancer treatment and autoimmune diseases. By harnessing precise techniques such as CRISPR-Cas9, TALENs, and zinc-finger nucleases, it is possible to engineer immune cells with enhanced specificity and functionality. The modification of T cells (e.g., CAR-T cells) and NK cells through targeted gene editing can lead to improved targeting of tumor antigens, resistance to immunosuppressive tumor microenvironments, and decreased risk of graft-versus-host disease.

Additionally, gene editing facilitates the generation of universal, off-the-shelf cell therapies by eliminating endogenous T-cell receptors and human leukocyte antigens (HLAs), thereby expanding accessibility to a broader patient population. These advancements have enabled the creation of next-generation therapies that integrate multiple edits to improve efficacy and safety, such as the simultaneous knock-in of chimeric antigen receptors and knockout of inhibitory checkpoint receptors.

As research and clinical trials progress, gene-edited immune cell therapies hold the promise of revolutionizing personalized medicine, offering novel treatments for hematologic malignancies, solid tumors, and chronic infectious diseases. The field continues to evolve rapidly, with ongoing efforts to enhance precision, minimize off-target effects, and scale manufacturing processes for broader clinical application. 

KEY LEARNING OBJECTIVES

  • Examine the role of gene editing technologies, such as CRISPR-Cas9, TALENs, and zinc-finger nucleases, in enhancing immune cell therapies.
  • Explore how gene modifications in T cells (CAR-T) and NK cells improve tumor targeting, immune resistance, and reduce the risk of graft-versus-host disease.
  • Compare the advantages of gene-edited universal, off-the-shelf cell therapies with traditional patient-derived approaches in terms of accessibility and efficacy.
  • Discuss the challenges and future directions in optimizing gene-edited immune cell therapies, including precision improvements, safety, and large-scale manufacturing. 
 WEBINAR CO-CHAIRS

Tatiana Maron, PhD
Senior Researcher at the Immunopharmacology Laboratory Oswaldo Cruz Institut, Oswaldo Cruz Foundation (IOC/Fiocruz)
Brazil

Tatiana Maron holds a degree in Medical Biology from the Federal University of Rio de Janeiro (UFRJ, 2007) and earned her master’s and Ph.D. in Biological Sciences (with a focus on Physiology) from the Carlos Chagas Filho Institute of Biophysics (IBCCF), UFRJ (2009, 2013). During her Ph.D., she completed a research internship at the University of Toronto in Toronto, Canada (2010-2011). She is an affiliated member of the Brazilian Academy of Sciences, and she is a FAPERJ “Young Scientist of Our State” grantee. In 2019, she received the II Young Researcher Award in Sepsis from the Institut Mérieux and the Latin American Sepsis Institute.

She coordinates the Neuroinfections Network at the Oswaldo Cruz Institute and is the Chair of the Animal Ethics Committee at the same institution. Additionally, she is a permanent faculty member of the Graduate Program in Cellular and Molecular Biology at IOC, vice-coordinator of the IOC Summer Courses, Chair of the South and Central America (SCA) Regional Early-Stage Professionals (ESP) Subcommittee, and SCA Regional Secretary of the International Society for Cell and Gene Therapy. She is also a member of the Research Integrity Committee at Fiocruz.

She serves as a reviewer for international journals and has experience in teaching and research on cell therapy, with a focus on inflammation and stem cell therapy for infectious diseases affecting the central nervous system. Her main research interests include mesenchymal stem cells, extracellular vesicles, neuroinflammation, cognition, sepsis, and malaria. 

Martin Bonamino, PhD
Research Coordination at the National Cancer Institute (INCA)/ Oswaldo Cruz Foundation (Fiocruz)
Brazil

Martin Bonamino holds a degree in Medical Sciences (Biological Modality) from the Federal University of Rio de Janeiro (2000) and a Ph.D. in Biological Chemistry from the Federal University of Rio de Janeiro (2004). Currently, he is a researcher at the National Cancer Institute and a Specialist at the Oswaldo Cruz Foundation, where he co-coordinates the FioCâncer Network.

He is a Cientista do Nosso Estado (CNE) fellow from FAPERJ, a member of the ANVISA technical working group on advanced therapies, the Global Outreach Committee of the American Society of Gene and Cell Therapy, and the South and Central America Legal and Regulatory Affairs Committee of the International Society for Cell and Gene Therapy. He is also the President of the Brazilian Association for Cell and Gene Therapy (2023–2025). 

 WEBINAR SPEAKERS

Bruno Solano, MD, PhD
Scientist at Fiocruz and at the D'Or Institute for Research and Education (IDOR)
Brazil

Bruno Solano holds a Medical degree from the Federal University of Bahia (2009), a Master’s in Biotechnology in Health and Investigative Medicine from Fiocruz (2012), and a Ph.D. in Human Pathology from the Federal University of Bahia (2016). Additionally, he completed an MBA in Health Management at Fundação Getúlio Vargas (2017). As part of his academic trajectory, he served as a visiting researcher at the Neuroimmunology Laboratory of the San Raffaele Scientific Institute in Milan, Italy (2010–2011), and at the Innovative Genomics Institute at the University of California, Berkeley (2023).

Currently, he is a Public Health researcher at the Gonçalo Moniz Institute (Fiocruz-BA) and the D'Or Institute for Research and Education (IDOR). He also serves as the medical coordinator and technical director of the Center for Biotechnology and Cell Therapy at São Rafael Hospital. His professional contributions include serving in leadership roles within the Brazilian Association of Cell Therapy, acting as First Secretary (2018–2021) and Treasurer (2022–2023). His research focuses on clinical and translational projects in cell therapy, regenerative medicine, gene editing, CRISPR/Cas9, and biomarker development, contributing to advancements in these cutting-edge fields. 

Marcelo Pereira, MSc, PhD
Scientist at the Nationwide Children's Hospital
United States

As a São Paulo Research Foundation (FAPESP) scholar, Marcelo conducted his undergrad and master research under the supervision of Dr. João Santana da Silva and Dr. Ana Paula Campanelli at the School of Medicine of Ribeirão Preto, FMRP-USP – São Paulo/Brazil. His focus in undergrad was understanding the modulation of immune and granulomatous inflammatory response during P. brasiliensis infection. He obtained his Masters degree in Basic and Applied Immunology there and studied the role of CD4+ and CD8+ cells in immune protection to P. brasiliensis.

Marcelo obtained his Ph.D. degree in cellular and molecular biology in Dr. Dario Simōes Zamboni’s Lab at the same academic institution. At this time, his research was focused on the determination of immune response mechanisms that contribute to the control of Legionella pneumophila in a murine model.

As an immunologist and now part of Dr. Lee’s Lab at Nationwide Children’s Hospital, his focus is to study how immunotherapy mediated by NK cells can tackle pediatric cancer, specially by using gene-modified NK cells by CRISPR/Cas9.

Experience in areas of: Immunology, Microbiology and Tumor Immunology, acting on the following topics: NK, CRISPR/Cas9, PDX models, CARs, CAR-T-CD19, TAMs, M1 and M2 macrophages, Flavonoids, Apigenin, NLRs, TLRs and pathologies related to pulmonary system as Legionella pneumophila, Legionaire's disease, protective immunity, granuloma, paracoccidioidomycosis, cellular immune response.