All ASGCT workshops are held on May 13, 2025, from 8 a.m. - Noon CT.
Annual Meeting Workshops are add-on deep dive sessions and require an additional purchase to attend.
ASGCT's 2025 Workshops will not be livestreamed. Workshop registrations purchased by virtual attendees of the 28th Annual Meeting will be available as on-demand recordins on May 17, 2025. Registration for the annual meeting is managed through your ASGCT account. Please sign in or create an account to begin your registration:
2025 Workshops
Current Advancements In The Development Of Cell Therapy For Cancer
In this workshop, we will discuss the latest advancements in the field of cell therapy/CAR T therapy for cancer. We will discuss the emerging role of T cell metabolic fitness, as well as linking the gut microbiome to efficacy of T cell therapies. Combinatorial approaches with immune checkpoint inhibitors as well as use of novel effector T cells such as gammas delta T cells will be discussed. Finally, innovative cell therapy approaches through use of in vivo delivery and nanotechnology will be reviewed.
Implementing Prime Editing For In Vivo Therapeutic Development And Towards Better Analysis Of High Throughput Functional Screens
This workshop explores two key areas: precision gene editing for in vivo therapeutic development and high-throughput screening for drug discovery. On the therapeutic side, discussions cover topics like optimizing guide sequences for targeting critical genes, improving prime editing through enhanced guide design, and overcoming DNA repair challenges using novel strategies such as protein fusions or small molecule binders.
For target discovery, the focus shifts to high-throughput screening technologies, including base editing toolboxes for saturation mutagenesis and variant effect, self-delivering CRISPR systems for co-delivery with other vectors, and optical pooled screens that enable mapping of multi-omic phenotypes in cells and tissues. These topics demonstrate how precision gene editing can accelerate progress in both preclinical and research settings.
Moving Lentiviral Vectors Through The Investigational New Drug (IND) Process
Gene and cell therapies for lentivirus-modified cell products have been approved by the FDA. The coming years will see more therapies reach this milestone as gene and cell therapy expands its presence within the healthcare market. As a result, academic and commercial investigators will need to navigate the expectations required for submission of an Investigational New Drug Application (IND) to the FDA, which includes guidance on chemistry, manufacturing and control (CMC) activities for a human gene therapy product. The IND-enabling studies require an interdisciplinary approach to safety assessment, efficacy, pathology and toxicology.
This workshop will focus on lentiviruses, with attention paid to:
- Integration, mechanisms and outcomes
- CMC activities
- Immune responses to the vector or therapeutic cargo
Each session will conclude with a panel discussion where the experts will answer questions from attendees.
Regulation Ready: Key Compliance Updates and What They Mean for CGT Development
This in-depth workshop will explore recent regulatory guidance issued by the Food and Drug Administration (FDA), focusing mostly on Chemistry, Manufacturing, and Controls (CMC) guidances from the last two years. Expert presenters with real-world experience will provide a comprehensive examination of key guidances and their practical applications. Key topics will include practical applications of the Advanced Manufacturing Technology Development Program (AMTDP) and platform technologies designation program.
The workshop will also delve into the challenges of comparability and potency for CGT drug products, featuring case studies from industry. Special attention will be given to the use of human- and animal-derived materials, with a detailed breakdown of the recent allogeneic guidance and its applicability across various product classes. The session will include opportunities for extended audience Q&A, and consideration of future regulatory guidance needs.
The Business of Advocates Advancing CGTs
There is a strong presence of patient advocacy organizations (PAOs) acting as the catalyst driving research to develop cell and gene therapies for many rare diseases. As a PAO, you may find yourself taking a larger role in the business aspect of translating therapeutics, which can be a daunting endeavor faced with both successes and setbacks. This workshop will explore real world business models that PAOs have pursued, ranging from ultra-rare disease nonprofits to established organizations pursuing venture philanthropy.
It will also review different funding mechanisms to advance an organization’s mission. This workshop intends to foster meaningful discussion to help guide organizations, whether in the startup phase or mature, as they face many decisions on the best business model to align with their goals and strategies to build their revenue and mission. This workshop is hosted in partnership with the National Organization for Rare Disorders.
Navigating the Nonclinical Pathway to Clinical Trials for AAV Based Therapeutics
This session will provide the current state of thinking on what a nonclinical package would look like to getting to a successful IND-filing for an AAV therapeutic. In the absence of an internationally aligned regulatory guidance, this session will address the nonclinical testing strategies for gene therapies drawing on the 2013 FDA and more recent regional guidances along with data driven and case study approaches that have provided success for academics and industry alike.
Topics addressed in this session will include core aspects to the IND including proof of direct benefit through nonclinical pharmacology, a pathway through your nonclinical safety package including managing unique routes of administration, biodistribution assessment, species selection and design of toxicology studies, how to translate this data into a clinical plan and best practice in pre-IND and IND regulatory writing and interactions. In addition, specialty areas will be discussed including genomic integration, considerations for pregnancy and children, vector quality and unique considerations for rare disease.