Add a specialized, deep dive session to your Annual Meeting experience.

All workshops are held on May 7, 2024, 8 a.m. - Noon ET.

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Revolutionizing Care: Gene Therapy Unveiled for Clinicians

New in 2024: Eligible for CME! (In-person and Livestream Only)

Gene and cell therapies are increasingly a part of modern medical care, yet formal training programs for clinicians are lacking. This workshop seeks to establish baseline education as well as highlight emerging trends in the biology, toxicity, and administration of G/C Therapy products for healthcare professionals from diverse backgrounds.

This activity has been approved for AMA PRA Category 1 credit(s)

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education through the joint providership of the UMass Chan Medical School and ASGCT. The UMass Chan Medical School is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

The UMass Chan Medical School designates this live activity for a maximum of 4 AMA PRA Category 1 Credit(s)™. Physicians should claim only credit commensurate with the extent of their participation in the activity.

Jointly provided by the University of Massachusetts Chan Medical School and ASGCT.




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AAV Manufacturing for CMC Success: Translating Preclinical Gene Therapy Products to Clinical Success

This workshop is especially geared to those who are early-career or who want to learn the basics of CMC considerations for AAV gene therapies. The intended audience for this workshop includes those working in early-stage companies (pre-Phase I) developing AAV gene therapy products.  

The AAV manufacturing workshop will provide an overview of how early process development decisions for an AAV drug product impacts IND enabling GLP studies that will ultimately inform success in the GMP manufacturing environment for clinical trials, and eventually a BLA. Importantly, the drug product is comprised of: i) the AAV capsid to ensure delivery to a specific tissue/cellular location, while de-targeting others, and ii) the genetic payload constituting the therapy of interest. Early decisions during the research and development phase around choice of capsid and design of the genetic payload can influence design of the CMC process and the biological outcome associated with the drug product. The first half of the workshop will review how early decisions influence CMC manufacturing from an academic and industry perspective, the importance of appropriate analytics to analyze the drug product and designing IND enabling toxicology studies that most closely emulate human biological outcomes. The second half of the workshop will provide a regulatory perspective on CMC, review the practicalities for moving CMC to novel AAV production platforms, and provide insights around the CMC impacts of applying AI to early design of the drug product. 

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Advancements in Immune Effector Cell Therapies: Innovations and Future Prospects

This workshop will focus on the latest developments in immune effector cell therapies, mainly for cancer but also other diseases, highlighting cutting-edge research, delivery technologies, and ongoing clinical trials in the field. Attendees will have the opportunity to discuss the challenges and opportunities in this rapidly evolving area. 

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Creating an Analytical Roadmap for Gene & Cell Therapies

This workshop is intended for those with an existing CMC knowledge base who want to take a deeper dive into analytical issues. The workshop will focus on multiple modalities including both cell and gene therapies. Discussion will focus more on development at later phases – Phase II/III through BLA. 

It is generally accepted today that there is not one specific analytical roadmap that works for ​​every cell and gene therapy product. Rather, sponsors adopt a holistic approach, taking into consideration existing regulatory guidelines, their product characteristics, and how their therapy will be used. In this workshop, attendees will learn about the tools and general ​​frameworks that can be tailored for their specific drug product as they progress through the drug development life cycle. Presenters will ​first ​share insights on the current state of CMC analytics, followed by an overview of the trends likely to shape the future of the CGT field. Time will be reserved for extended Q&A. 

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Delivery and Development of Precision Genome Editing Technologies

This workshop will focus on the intersection between the engineering, delivery, and in vivo application of genome editing tools. We will discuss how novel delivery research drives innovation and need for new genome editing strategies, and vice versa. Attendees of this workshop can hope to learn about the most recent applications of a variety of genome editors across a broad range of target cell and organ systems. 

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Why do Rare Disease Clinical Trials Often Miss the Mark? Best Practices and Approaches to Optimize Design of Rare Disease Clinical Trials

While clinical trials are a required part of the drug research and development process, interventional clinical trials for rare diseases often have suboptimal designs due to recruitment limitations, insufficient representation of patient populations, and inadequate endpoints. Moreover, funding considerations can impact the clinical development of various investigational therapies. However, many groups and organizations have developed innovative strategies for patient recruitment by making organized efforts to work directly with the various communities, cultures, and stakeholders a new treatment may affect. This workshop will share proven practices of optimizing CT enrollment, trial designs, and endpoints, as well as funding management when trials may unexpectedly end. This workshop is co-hosted with NORD.

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