This session will focus on emerging new areas of gene therapy, in the clinic or advancing towards the clinic
This session will explore how the generation of stem cell models from patients with genetic diseases has increasingly improved to the extent that they can now predict disease phenotypes and even be used for drug discovery to correct the condition.
Understanding stem cell systems is challenging due to their cellular heterogeneity. Advanced single-cell technologies now allow for multi-layered deep molecular characterization and insights into individual cell behaviors. This session will discuss these recent methodological advancements and their impact on stem cell research.
Embryo models formed from stem cells open a scientifically and ethically conducive path for embryology. Here, speakers will focus on ‘Non-Integrated Embryo Models’ that mimic only certain tissues of human embryo development, often lack extra-embryonic membranes, and manifest the ability to form connected embryonic tissues and organs.
This session will examine how mechanical forces direct behaviors of stem cells and define physiological properties of their resident tissues. We will explore the molecular basis of force sensing and force generation, explore how forces are transduced into biochemical signals, and discuss how altered forces promote stem cell pathologies.
Organized by Novo Nordisk A/S There are many challenges in moving an academic discovery towards clinical application. A major hurdle to overcome is how to understand and characterise the composition and behaviour of the cellular drug product. Technologies, methodologies, and the level of detail required for academic publication do not always match those required for large scale manufacturing, or by regulators for clinical trial approval and, ultimately, the granting of marketing authorisation. In this Focus Session, we will address issues such as scale up, prevention of off-target differentiation, in vitro prediction of function, and in vivo proof of engraftment and viability. An expert academic and industry faculty will discuss these issues, the techniques that currently exist to address them, and the innovation challenges facing the field in order to close the technological gaps that exist in these areas.
Organized by BlueRock Therapeutics Despite their immense potential for treating a wide range of diseases characterized by irreversible cell loss and high unmet medical needs, the broad clinical application of PSC-based stem cell therapies faces major obstacles. These hurdles include technical limitations, high costs, as well as challenges related to poor cell survival and engraftment. The purpose of this session is to provide an update on the ongoing efforts within both industry and academia aimed at overcoming significant barriers hindering the widespread adoption of PSC-based stem cell therapies in the clinic.
The "Career Paths in Stem Cells and LEO" panel at the ISSCR Conference is designed to highlight the contributions and challenges faced by early-career researchers and postdocs in the field of stem cell research conducted in low Earth orbit (LEO). This 30-minute session will feature young scientists and postdoctoral researchers who are at the forefront of this exciting area of research. The panel aims to provide insights into their innovative work, discuss the unique challenges of conducting research in space, and explore opportunities for career development. Panel Objectives: 1. Highlight Contributions: Showcase the innovative research being conducted by students, postdoctoral researchers, and early-career scientists in stem cell research in LEO. 2. Discuss Challenges: Address the specific challenges faced by early-career researchers working in space-based science and potential strategies to overcome them. 3. Explore Opportunities: Discuss future opportunities for young researchers in stem cell research and space science, including potential collaborations and funding sources. 4. Provide Insights: Offer practical advice and insights on career development in this niche field.
This session will focus on the critical role of optical genome mapping (OGM) in assessing the genome integrity and off-target events in cells engineered for therapy development, specifically within the context of stem cell research. The presentation will begin with an introduction by Alicia Bertolotti from Bionano, who will highlight how OGM provides a high resolution, genome-wide, sensitive, and reproducible workflow that can be applied in cell and gene therapy applications. Following the introduction, Dr. Wanda Gerding from the Department of Human Genetics and Lucia Gallego Villarejo from the Department of Molecular Biochemistry at Ruhr-University of Bochum will present findings from their recent publication. Their study leverages the CRISPR/Cas9 gene-editing technology in human induced pluripotent stem cells (hiPSCs) to create cerebral organoids. They found that while the edited hiPSCs retained their pluripotency and could still form organoids without significant phenotypic changes, optical genome mapping revealed various genomic alterations like chromosomal copy number variations. Professor Emeritus Dr. Jeanne Loring from the Scripps Research Institute will then share her expertise on iPSC culture and genome engineering. Her talk will focus on the use of OGM to assess the genomic stability and safety of iPSCs—crucial for their use in disease modeling, drug testing, and potentially therapeutic applications. Overall, this session highlights the necessity of using high-resolution, unbiased genome-wide analysis workflow of OGM to assess the safety and efficacy of gene-edited models, propelling forward the field of cell and gene therapy research.
As the landscape of organoid research continues to evolve, we explore real-world applications in disease modeling and developmental biology. Robert Opitz, Institute of Experimental Endocrinology, will discuss using hiPSC-derived cerebral organoids as a model to study the regulatory role of thyroid hormones (TH) during early human cortex development. His team applied single-cell RNA sequencing and RNAscope™ in situ hybridization to identify TH-induced changes in specific neuronal cell types and spatial expression patterns of genes regulating cortical neurogenesis, capturing these changes with high spatial resolution. Kai Kretzschmar, Julius-Maximilians University, will present on using organoid technology to analyze the cellular and molecular biology of oral epithelia in both steady and disease states. The complex stem cell niches and structural heterogeneity of the oral cavity have left it underexplored compared to skin and gut epithelia. Generating robust organoid cultures relies on creating consistent and reproducible conditions. Through the provision of high-quality reagents and innovative technologies for spatial biology and automated protein analysis, Bio-Techne enables researchers to increase their culture consistency and get the most out of their precious samples. Join this Innovation Showcase to learn more about these advancements in organoid research and get recommendations for achieving reproducible culture conditions.
Pluripotent stem cells (PSCs) offer promising potential for life-saving therapies, but their clinical adoption faces challenges due to inadequate scalable production processes. Key process hurdles include understanding bioreactor mixing, medium exchange, gas transfer, and cell harvest. Developing reliable methods to scale up manufacturing processes is crucial for robust and reproducible production of clinically relevant lot sizes. Our systematic process development has identified the following key requirements to overcome these challenges: versatile bioreactors with well-characterized hydrodynamics, efficient and scalable MX methods, well characterized gas transfer in large-scale bioreactors, and scalable, low-shear methods for cell harvest, wash, and concentration. The Vertical-Wheel bioreactor-based platform enables scalable production of shear-sensitive PSCs and PSC derived products through planar seed train expansion and scalable 3D expansion/differentiation phases. We successfully scale production from 0.1L to 15L, generating clinically relevant quantities of various cell types (PSCs, PSC derivatives, MSCs, and other primary cells) and growth modalities (microcarriers, aggregates, and single cells). By proactively addressing scale-dependent challenges, we have established reliable large-scale processes for shear-sensitive cell therapy production, facilitating their transition from research to clinical use and enabling widespread access for the population.
Organoids and organotypic cultures are revolutionizing our capability to model tissues in vitro by leveraging the biological capacity of tissue-specific stem cells to generate models that closely mimic the intracellular and intercellular phenomena observed in vivo. These cultures have consequently proven valuable in diverse applications from developmental biology to evaluating antiviral effects and the toxicity of novel drugs. In addition to biological fidelity, technical characteristics such as scalability, access to both apical and basolateral surfaces, automatability, and the ability to incorporate cells from different functional lineages can be critical for being able to use stem cell-based cell cultures in impactful applications. This presentation describes novel protocols and tools, including suspension culture, culture and co-culture at the air-liquid interface, apical-out and other matrix-free methods, and variability-reducing cultureware, and reviews the benefits these adaptations have for automation, culture scale-up, increasing reproducibility and better enabling experimental measurements, including imaging.
Human induced pluripotent stem cells (hiPSCs) and genome engineering technologies have become powerful in vitro tools to model and study various genetic conditions. In this session, Prof. Bill Skarnes, a recognized leader in stem cell genome engineering and Director of Cellular Engineering at the Jackson Laboratory for Genomic Medicine, will present on 1) the efforts undertaken that led to the identification of KOLF2.1J as a suitable reference hiPSC line, 2) provide updates on the generation of hundreds of variants in KOLF2.1J using high-throughput precision editing as part of the NIH-funded iPSC Neurodegenerative Disease Initiative (iNDI), and 3) introduce the MorPhiC project that aims to develop a catalog of molecular and cellular phenotypes for null alleles for every human gene. In the second half of this session, Dr. Camille Januel, from the Chan Zuckerberg Biohub San Francisco, will discuss her efforts using CRISPR technology to tag organelles with the aim of studying organelle dynamics in various hiPSC-derived cell types. This platform will enable the detailed examination of organelles via live cell imaging and proteomics to better understand cellular impacts of disease-associated mutations.
Emerging embryonic and induced pluripotent stem cell-derived cell therapies for a range of indications will be showcased in this session.
Stem cells can be derived from patients with many diseases and used as platforms for understanding molecular mechanisms underlying disease with a view to developing therapies.
This session on new technologies is being harnessed to precisely engineer the identity of clinically valuable human cells. The cross-disciplinary selection of speakers spans the fields of cell fate engineering and emerging technologies aiming to manipulate cells with high efficiency and fidelity.
Capturing stem cells in a particular state that reflects a lineage and stage is a major endeavor at the base of stem cell biology. In this session, speakers will focus on recent advances in the derivation of totipotent stem cells and recapitulation of germ cell development in the dish.
This session will explore the dynamic interplay between stem cells, their progeny, and niche cells in maintaining and repairing tissue architecture. Healthy organs require continual upkeep of tissue composition and form during maturity and robust regeneration after injury. We will delve into new and emerging mechanisms that underpin the physiological maintenance and repair of tissue architecture throughout our lifetimes.
Organized by Berlin Institute of Health (BIH) at Charité Berlin In this focus session, we will explore the opportunities and challenges associated with the development of stem cell-based therapies within the broader context of gene and cell-based therapies. The session will kick off with an introductory talk by Giulio Cossu, who will highlight the obstacles encountered in the translational process and the complexities of establishing a sustainable framework for delivering these therapies to patients, particularly those with rare diseases. Participants will be introduced to two distinct cases demonstrating how Advanced Therapy Medicinal Products (ATMPs) can reach patients, despite commercialization hurdles. Moreover, the session will delve into various strategies adopted across Europe to support the advancement of gene and cell therapy. We will examine the German national strategy for gene and cell therapy, the initiative by French INSERM PEPR Biotherapies and Bioproduction for innovative therapies, and the efforts of the UK Catapult for Cell and Gene Therapy. A panel discussion will provide insights into how these approaches address the promising opportunities and challenges in the field. This focus session aims to provide a comprehensive overview of the current landscape and future directions in stem cell-based therapy development, emphasizing collaborative and innovative strategies to navigate the existing hurdles.
Co-organized by Joint Research Centre (JRC), European Commission
Stem cell-based models are of special interest for pre-clinical studies and chemical safety assessment in the regulatory arena. However, experiments involving stem cells are associated with challenges such as variability and reproducibility. Standards can be a valuable tool to find the right balance between the intrinsic biological variability of stem cells and the acceptable level of reproducibility of test results. Several documents discussing standards and best practices for working with stem cells have already been published. The aim of this session, organised by the Joint Research Centre of the European Commission, is to discuss about the current level of adoption of standards and best practices in non-clinical research and on how to facilitate their implementation. The view of experts from stem cell research, industry, and regulatory agencies will be gathered in a panel discussion.
Join us for these events in the Poster and Exhibit Hall:
3:45 PM – 4:45 PM POSTER SESSION III: ODD
4:45 PM – 5:45 PM POSTER SESSION III: EVEN4:00 PM - 4:35 PM EXHIBIT HALL THEATER TALKSBite-sized, 15-minute sessions on specialized topics. Pop in and out of the Exhibit Hall Theater to catch these brief presentations from industry leaders.4:40 PM - 5:35 PM CAREER EXPLORATION PRESENTATIONS View these career focused, 5 or 10-minute presentations in the Exhibit Hall Theater. Talks will be given by BlueRock Therapeutics / Bayer AG and STEMCELL Technologies, Inc.MEET-UP HUBS: Connect with your colleagues and engage in conversation and networking at these common interest discussion forums.
Organized by the European Research Council
A scientific officer from the ERC Executive Agency will answer your questions about the ERC funding opportunities and update you about the recent changes.
Organized by The Stem Cell Podcast
Come and meet the hosts of The Stem Cell Podcast, Drs. Daylon James and Arun Sharma, and learn how your research could be featured on a future episode.
Organized by Steve Oh, Independent Cell Therapy Advisor, Singapore
The cell and gene therapy community has spent over 20 years investigating the front end of the manufacturing processes; however, the final cell or secretion products will likely be fresh or frozen in content. This Meet-up will discuss the challenges in delivering frozen cell products. There are seven important topics open for discussion: Primary Containers, Cryopreservation, Storage and Shipping, Stability, Thawing, and Post thaw Analytics.
Organized by Pierre Schembri-Wismayer, DeNovo Cell Ltd/University of Malta
In this meet-up, we welcome individuals who group isolating iPSCs, particularly those derived from blood - to learn about optimise methods and quality testing for Clinical level iPSCs.
Organized by Olivia Majhi, Cytogenetics Lab, Department of Zoology, Banaras Hindu University, India
This Meet-up will facilitate networking among students and postdocs while discussing the essential skill sets required for success in academia and industry. This discussion can help the participants understand the soft skills, communication strategies, leadership qualities, collaboration dynamics, time management tactics, and networking strategies crucial for thriving in both academic and industrial environments. From honing soft skills to mastering effective strategies, attendees will gain valuable insights to thrive in diverse professional environments.
Organized by Ran Jing, Harvard University Medical School, USA
This hub provides a networking opportunity for scientists working on using pluripotent stem cells to study immunology and develop PSC-derived immune cells (T, NK, macrophages, etc.) for cellular immunotherapies.
Human neurons programmed through transcription factor (TF) overexpression model neuronal differentiation and neurological diseases. However, programming specific neuron types remains challenging. Here, we modulate developmental signaling pathways combined with TF overexpression to explore the spectrum of neuron subtypes generated from pluripotent stem cells. We screened 480 morphogen signaling modulations coupled with NGN2 or ASCL1/DLX2 induction using a multiplexed single-cell transcriptomic readout. Analysis of 700,000 cells identified diverse excitatory and inhibitory neurons patterned along the anterior-posterior and dorsal-ventral axes of neural tube development. We inferred signaling and TF interaction networks guiding differentiation of forebrain, midbrain, hindbrain, spinal cord, peripheral sympathetic and sensory neurons. Our approach provides a strategy for cell subtype programming and to investigate how cooperative signaling drives neuronal fate.
We’ve all seen the graph of people that start a science degree and how many stay within academia. While the data may seem disheartening, what the graph doesn’t show us are the myriad of career stories about the people that left academia and used their scientific superpowers to continue to make an impact on science and society beyond academia. In this talk, we’ll share some science career stories, and showcase how Scismic’s machine-learning powered platform can help uncover the range of alternative research careers that are out there, waiting for you to unleash your creativity and know how on overcoming a range of challenges that we face as a society.
NUVISAN Innovation Campus Berlin (ICB) GmbH is an Australian-owned, European-based CRO and your go-to partner for all your therapeutic development needs. Our expertise and capabilities span the entire pharmaceutical discovery pipeline from target identification and validation through to phase 3 clinical trials. Experts from our four main functions serve your project to streamline drug development. With flexible entry points, we thrive by working collaboratively with you to achieve both scientific rigour and yield the best possible outcome. In our presentation, we will give you a brief introduction to the company, an overview of our extensive capabilities (including our unique access to compound libraries not available elsewhere) and examples of how we can work with you to leverage human iPSCs in your drug discovery journey. We also look forward to meeting and sharing more details with you at our booth.
CytoTronics' Pixel introduces a revolutionary method for monitoring cell activity, utilizing label-free, non-invasive live cell electrical imaging, thereby surpassing traditional optical imaging techniques. The platform uses a high-density electrode array with single cell resolution (12.5 µm) to capture over 20 functional and morphological parameters, including tissue barrier integrity, cell-surface attachment, cell flatness, and motility through unique field-based impedance measurements. To complement, electrophysiological recordings, combined with the ability to electrically stimulate cells, enables functional assessments of electrogenic cells, such as cardiomyocytes and neurons. Measurements can be taken at intervals ranging from minutes to hours, creating "electrical heat maps" and time-lapsed videos revealing the end-to-end journey of an experiment. Cell features can be assessed at the well level generating a population average, or spatial data can be used to evaluate single-cell behavior and assess heterogeneity in cells and their responses. Further insights into kinetics are derived from time-domain analyses. Assay development, initially conducted in a single plate, can seamlessly scale up to a multi-plate format without compromising time resolution or readout quality. The Pixel represents a groundbreaking tool for characterizing in vitro disease models and streamlining phenotypic screening for therapeutic interventions.
Join the Stem Cell Podcast for a live show, where hosts Drs. Daylon James and Arun Sharma will discuss research taking place at the International Space Station (ISS). Experts from the ISS will discuss the opportunities and challenges of working with stem cells in a microgravity environment, and what this could mean for the future of stem cell-based therapies.
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