Extracellular Vesicles (EVs) can shuttle information between cells. While the significance of natural EV cargo delivery is debated, bioengineering techniques enable efficient loading of EVs, including with components of gene editing machineries.

This session will highlight various strategies for loading EVs with gene editing components and discuss concepts for increasing their effective delivery for functional purposes.

Growing evidence suggests that MSCs can undergo rapid apoptosis following systemic administration and that it is the host response promoting MSC death that drives many of the beneficial responses seen in a range of disease models.

This may be a paradigm shift in how MSC-based cell therapies may be best utilized. Leading investigators in this area will explore the data and potential ramifications.

MSC-based cell therapies are heavily weighted towards intravenous administration or towards direct joint administration for orthopedic diseases.

Are these the only ways to go?

Leading investigators in MSC therapeutics will explore the pros and cons of other routes of administration.

Artificial intelligence, machine learning, and agent-based modeling are proving to be powerful tools not just for cell product manufacturing but for understanding and predicting biologic and potential therapeutic actions of cell-based products.

Leading experts in these areas will provide a broad and provocative overview including the generation of living cell-based robots.

Rapid advances in cell biology and tissue engineering techniques have strong implications for use in cell and gene therapies. This session will explore recent exciting advances in organ-specific cell engraftment, synthetic gene circuits, and lessons learned from thymus biology.

The leap from bench to bedside for iPSC-based therapies continues to be one of the most exciting and challenging in clinical research. This concurrent session focuses on the current progress of several iPSC-based therapies to make clinical entry for patients with hematologic disorder, Parkinson’s or corneal endothelial dysfunction.

Join us in this session as we review innovative translational approaches to establish desired cell features for various therapeutic applications. We will address gene editing characterization requirements and how they are met with corresponding QC and nonclinical safety strategy as well as evaluate the potential of both autologous and allogeneic approaches to promote a holistic iPSC therapeutic strategy.

As first generation MSC products navigate through late-stage trials and early commercialization in multiple geographies, the GMP supply chain and scalable MSC manufacturing technology have progressed to the point that MSCs can be starting materials or sub-components within next generation therapeutic products, with product development and clinical translation timelines happening at an accelerated pace.

This session will highlight leading technology where MSCs are manufacturing starting materials, but the final product is not a simple cell suspension for infusion.

Yes, “live MSCs” are still good for something.

How the process development stage of a CGT product goes is critical to both the speed and success of getting your product to market and into patients. Process development approaches and considerations are constantly changing based on the state of technology and regulation. In this session we describe recent success stories in process development and outline how successful companies have approached this stage of product development.

We will also tackle the future of process development from both a regulatory perspective and considerations for applying machine based learning and AI to your process development. Attendees will come out of this session with insights on what to think about in their next stages of process development for their CGT product.

MSC exosomes are currently the most extensively researched and most advanced in clinical development. While exosomes derived from other sources, such as bacteria, milk, and immune cells, receive comparatively less attention in research and clinical applications, they harbor significant clinical potential as vaccines, drug delivery vehicles, or therapeutic agents.

Several of these exosomes are either undergoing clinical testing or are on the verge of entering such trials.

Gene editing is not just for T cells. Join us for an exciting, cutting edge session to learn and discuss how precision engineering can be used to engineer different cell types and microorganisms.

Learn about recent advances in developing macrophage therapies; understand how CAR-engineered Tregs can induce immune tolerance with implications for autoimmune diseases, organ transplants and stem cell transplantation. Explore the role of gut microbiome in intestinal diseases and how microbiome engineering might aid in abetting them.

This session addresses the use of surrogate endpoints in the US accelerated approval process, requirements for acceptance of surrogate biomarkers as primary endpoints in clinical trials and how such surrogate biomarkers of clinical efficacy can be qualified.

In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). At the time, this was a dream to be able to successfully genetically correct an enzymatic defect to treat disease. In 2016, a version of this therapy using a different viral vector was approved in Europe.

Several gene modified hematopoietic stem cell products are now approved to treat rare diseases Such as ADA-SCID, cerebral adrenal leukodystrophy, metachromatic leukodystrophy and hemoglobinopathies including sickle cell disease and beta thalassemia. One of these products is the first to use CRISPR gene editing. What were the scientific, technical, and clinical hurdles overcome to bring these therapies to regulatory approval? What are the next therapies on the horizon and how should we think about developing therapies and models for access to more patients?

The current early clinical trials landscape for Advanced Therapy Medicinal Products is growing, but progression into later clinical stages is stumbling across several challenges, with investment being one of them.

In such a novel and complex field, where product benefit-risk balance is still uncertain, being able to generate compelling data to persuade and assure public and private investors is vital. In this session, we will focus on investment challenges and opportunities and discuss ways to increase the value of clinical studies while maximising funding odds and successful progression towards patient access.

Join us for an insightful exploration into the use of CAR-T and regulatory T cells (Tregs) in treating autoimmune diseases. The session will highlight recent clinical progress with CAR-T cells, specifically targeting and modulating B-cells. Additionally, we'll delve into the role of Treg cells as a strategy for modulating immune responses.

As we navigate these exciting clinical advancements, our discussions will also consider the evolving landscape of commercialization in autoimmune disease therapies, exploring pathways to bring these innovations to market and provide tangible solutions for individuals facing autoimmune challenges.

Genome editing technologies has enabled modification of DNA sequences with unprecedented precision and efficiency. Together with efficient platforms for ex vivo and in vivo delivery into cell types of interest, it offers an exciting possibility of tailored medicine to effect lasting curative therapies. Recent advances in the technology landscape have paved the way for rapid development of new medicine for any genetic disease.

This session brings together pioneers in the field who will share important perspectives on developing and delivering these novel approaches that navigate current manufacturing, clinical and regulatory challenges to achieve affordable therapies.

Cell therapies, from cancer to monogenetic diseases, continue to demonstrate clinical benefit. Recent regulatory approvals demonstrate the changing paradigm across the therapeutic landscape of future ‘curative’ across many unmet medical needs. With continuing success comes increased responsibilities of (1) driving industrialization of manufacturing, (2) reducing cost of goods while maintaining consistent product quality, (3) demonstrating long-term durability of responses, in permitting democratized access to these therapies.

This session will showcase the current state of the industry and will highlight how the leaders in the field ate thinking of innovation in terms of NextGen Manufacturing, engineering processes to optimize fixed and operating cost considerations, reducing time in manufacturing and/or QC release, with advances in process & analytical technologies leading to accessibility / availability to such ‘curative’ therapies to patients.

Are you considering your strategy and approach early enough? Do you know what challenges and opportunities lie ahead to ensure CGT innovations actually get to those who can benefit in routine clinical practice?

The reimbursement landscape globally is complex, with every geography approaching it subtly, if not significantly, differently. In some areas the value proposition translates into pure negotiations, in other countries it can require a full in-depth health economics analysis to enable funding. Add in reference pricing, insurance company rules and requirements, and accountancy limitations around pricing agreements, and it gets even more complicated to secure reimbursement.

This session will unpick some of these topics in more depth and share insights from market access experts with specific experience in the cell and gene arena, with the aspiration that new companies start planning their access strategy in parallel to their phase II/III trials.

What impact will cutting-edge supply chain solutions, distributed networks, and emerging manufacturing technologies have on both centralized and decentralized manufacturing models? How should we adapt to accommodate the needs of evolving and expanding patient populations? Are there valuable lessons from past experiences that can guide us in mitigating potential risks in the future?

• Planning for growth and scalability today and reviewing lessons learned from the cell and gene therapy developers' perspective
• Impact of new patient populations on cell collections, manufacturing, and clinical settings
• Logistics and supply chain approaches

Cell therapies, from cancer to monogenetic diseases, continue to demonstrate clinical benefit. However, recent regulatory approvals notwithstanding, with for example six approved CAR-T products in US, only a small portion of eligible patients are able to avail of approved CAR-T products due to multiple confounding factors and no ability to impact the needle-to-needle time for providing therapies to patients with rapidly advancing disease.

Similar challenges also exist in delivery of engineered stem cell products for monogenetic diseases where patients are subjected to mobilization, toxic chemotherapy regimens, and concomitant stem cell transplant with its associated co-morbidity and co-mortality. Therefore, there is an urgent need to develop novel manufacturing and quality paradigms that permit same day patient treatment, leading to development of engineered cell therapies in vivo. This session will focus on advances in transfusion medicine, novel modalities for cell engineering, novel delivery technologies that permit in vivo cell engineering and challenge us to debate and define the future roadmap for In Vivo Cell Therapies.

The development of cell and gene therapies (CGT) for rare diseases has seen promise and efficacy of treatments, but also disappointment in realizing viable commercialization, reimbursement, and broad patient access.

This session will discuss challenges around development of CGT for rare diseases and patient access, the potential for life-altering treatments, and the mission that drives those committed to furthering these goals.