Pre-Meeting Workshops
Register separately for one or more workshops to be held May 15

The Pre-Meeting Workshops will be held Sunday, May 15.

Registration for the Pre-Meeting Workshops is separate from registration for the meeting.

Register Here

Morning Workshops

8 a.m.- 12 p.m. (ET)

Genome Editing: The Ever-Expanding Editing Toolbox
Room 207

Gene editing has become an essential technology in our day-to-day research and it is a promising approach for application in the clinics. In this workshop we will focus on the most novel gene editing strategies designed and the development of new editing tools by protein engineering. A special attention will be given to HDR improvement by optimized donor templates. Altogether, this workshop will contribute to learning about the ever-expanding gene editing toolbox.

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Newborn Screening: Toward a System That Keeps Pace with Gene Therapy Advances
Room 206

This workshop will dive into the key policy issues affecting the U.S. newborn screening system, given the anticipated wave of innovative therapy approvals. Topics will include modernizing the NBS process; addressing the challenges of data collection via NBS pilot programs; and evaluating emerging screening and clinical programs.

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Emerging Clinical and Pre-Clinical Approaches to Oncolytics
Room 102 A/B

Oncolytic virus therapy has the potential to become a new arm of anti-cancer treatment, potentially augmenting existing immunotherapy approaches such as PD-1 inhibition and replacing others, such as chemotherapy, as combination partners for immunotherapy. In this workshop, the key leaders in the development of oncolytic virus therapy will present new approaches both in the lab and in the clinic in the development of oncolytic virus therapy.

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A Focus on Gene and Cell Therapy for Clinicians
Salon G

This workshop is designed for clinicians with experience caring for patients for which a gene or cell therapy is available. Speakers will review the underlying biological basis for gene therapy, the regulatory framework for oversight of clinical trials to develop safe and effective products for gene therapy, and the primary forms of toxicity encountered with various gene therapy vector types. Attendees will gain an appreciation for the nature of the process of clinical research and the challenges and opportunities presented to patients and families participating in studies.

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Issues and Trends in Clinical Development for Cell and Gene Therapies 
Room 204

This workshop will discuss strategies for optimizing gene and cell therapy clinical development. In particular, the program will explore the planning and execution of pediatric studies, best practices for managing unexpected adverse events, and opportunities to utilize innovative study designs. Speakers will share both conceptual ideas as well as case studies that illustrate how to address these areas of clinical development.

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Coming of Age: Stem Cell Therapies from Bench to Clinic
Room 201

Curative stem cell therapies have been a dream for many researchers in the field. In the past 20-30 years, the convergence of several technologies finally made this a reality in the clinic. This includes understanding stem cell populations, stem cell niches, ES, IPS technologies, and differentiation cocktails. The advent of editing technologies, such as CRISPR, further turbo-charged the field. We are no longer confined to the design of nature and are now able to build synthetic biology on top of nature's design and make synthetic and immune evasive stem cells. In this workshop, we would like to discuss the latest advances in basic stem cell science and also highlight the latest advances in stem cell therapeutics.

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Viral Vector Development
Room 202

This workshop will provide guidance on how to choose, design, and produce the appropriate viral vector for your purposes. Comprehensive reviews of lentiviral, adenoviral, and other vectors will lead us to discuss considerations of viral tropism, vector production in small-scale for laboratories, animal model choices, and vector dose. A final session will be dedicated to developing adeno-associated viral vectors for preclinical and clinical applications, including capsid choice, expression cassette design, and vector production from small to large scales. 

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Afternoon Workshops

1-5 p.m. (ET)

AAV Vector Integration
Room 202

While AAV vector genomes primarily persist as episomes, low rates of vector integration into the host genome do occur. This workshop will address various facets of this topic, including animal models, and genomic and bioinformatic platforms to assess clinical risk. The program will also cover the pathogenesis of hepatocellular carcinoma, clinical monitoring, and regulatory requirements. 

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Kick-Starting My Career in BioIndustry: From Entry to Thriving in the Matrix
Room 206

Bioindustry holds many opportunities for personal and professional growth, but how is it that I get STARTED in the industry and, just as importantly, how can I have my career thrive once IN the industry? These are important considerations, too important to be left to armchair speculation or hope that the right position/career move comes along. Thriving in bioindustry takes planning, circumspection, and active work!! During this workshop, we'll hear from the pros who have recently made the successful transition to bioindustry as well as those who have risen through the ranks to successful careers. All of our speakers are passionate about communicating their wisdom to colleagues seeking paths in bioindustry. If you want to get started on the right foot or continue running on the path of career development, this workshop is for you!

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Early-Stage Clinical Development: What You Need to Know
Room 201

In this workshop, experienced panelists will highlight how academic investigators may successfully transition from preclinical to clinical development. Topics will include developing a target product profile (TPP) and considering nonclinical, clinical, and CMC aspects of a development program. Additionally, the program will address breaking into the clinical trial space and partnering with biotechnology companies and CDMOs.

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Immune Effector Cell (IEC) Therapies
Room 204

The Immune Effector Cell (IEC) Therapies Workshop will feature discussions of the current state of IEC therapeutic approaches with updates on IEC-related toxicities , new directions in T cell engineering specificities and new generation CAR-T cell therapies.

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What to Expect as a Participant in a Gene Therapy Clinical Trial
Salon G

This workshop is intended for patients and caregivers, along with advocates and providers, to better understand what to expect when participating in a clinical trial for gene therapy. It is a complex process with many important considerations including eligibility, understanding the procedure, potential risks or outcomes, and logistical challenges an individual or family may face. The speakers will share their knowledge to address these topics and help manage expectations for those considering clinical trial participation. 

Patients, caregivers, volunteer advocates, and employees of non-profit patient advocacy organizations can receive FREE registration for this workshop. Read these instructions to learn more.

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Driving Standards to Accelerate Development: Convergence of FDA Perspectives and Industry Needs 
Room 207

The workshop will discuss the role of documentary standards and reference materials in the context of product development and meeting FDA regulatory expectations. Speakers will address national and international standards development efforts including consortia models for filling scientific gaps that can foster standardization and the development of reference materials. Developers will share their perspective on needed manufacturing standards. 

Download the Workshop PDF

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