Immune Cell Engineering and iPSC
Immune cell engineering is the forefront modality in translational medicine today. Immunotherapies are rapidly moving to iPSC as a source for engineered therapeutics based on the ability to derive hematopoietic cell types from a replenishable seed stock and to efficiently genetically engineer a master cell bank. With the development of powerful synthetic biology tools it’s now feasible to build boolean logic loops in target selection for tumor recognition, or to incorporate sense and respond circuits which regulate payload delivery, control immune polarity, or provide smart safety switches. Integration of these attributes into next generation regulatory T cells and macrophages will frame discussion of these new horizons in immunotherapeutics.
Practical Considerations and Solutions in the Preclinical Development of Investigational Stem Cell Products
Human stem cell products have the potential to treat and prevent many diseases and there are growing research and development efforts underway to make next-generation stem cell therapies a reality. However, there are numerous scientific and regulatory considerations and challenges to address in the development of these investigational products, beginning in the preclinical phase prior to testing in humans. This Focus Session will present practical insights into key scientific and regulatory aspects of preclinical development for an investigational stem-cell product to justify it as safe and efficacious– critical elements of a regulatory submission to initiate human clinical studies.
The Science and Ethics of Human Brain Organoid Research
Rapid developments in human brain organoid research are likely to produce tractable new bioengineered tools for understanding functional interconnectivity of the human brain and dysfunction involved in many neurodegenerative diseases. Despite the excitement surrounding this subfield of stem cell research and its considerable scientific promise, advances could also raise novel ethical concerns. As researchers generate evermore realistic organoids that resemble human brains in vitro, it is critically important to understand what ethical boundaries may exist and where researchers and regulators should draw the line for research. In this focus session, panelists will discuss scientific, ethical, and policy issues surrounding the use of human pluripotent stem cells to generate self-organizing brain organoids and assembloids for research.
Tools for Basic and Applied Stem Cell Technology
Presented by COREdinate Group (ThermoFisher and Stem Cell Technology)
Stem Cell COREdinates (www.COREdinates.org) is a consortium of human pluripotent stem cell-focused cores that share expertise with protocols, reagents, and technological advancements to establish “best practices". The first part of our Focus session will have selected presentations from Stem Cell COREdinate members. These presentations will cover a number of different areas of expertise including reprogramming, gene editing, disease modeling, and biobanking. The second part of the session will focus on advances in gene editing and cell therapies.
Translating Your Discovery: From the Laboratory to Industry
Moving a discovery from the academic laboratory towards commercialization, or the so-called “translational process,” requires many steps and multiple areas of expertise that collectively advance the project toward a clinical or commercial endpoint. These include building your core team, protecting your intellectual property, financing the process, working with pharma, among other areas. This program brings together experts from industry, technology transfer, venture capital and academics who have gone through the process to help those that are currently translating discoveries or just want to learn more about the process.