Pre-Conference Workshops

Workshop A | Tuesday, August 4 | 9:00am -11:00am EDT

Exploring the ‘What, Why & How’ of Re-Dosing for Gene Therapy 

The immunogenicity of viral vectors is perhaps the biggest challenge the gene therapy field faces. Whilst viral vectors have many favorable characteristics they are non-replicating and thus over time transgene expression is often expected to slowly reduce. Thus, the possibility of re-dosing a patient would be a desirable remedy to boost expression. Re-dosing is limited by a unique set of challenges itself, however. The biggest of which being the formation of Nab’s to the viral vector which facilitate vector clearance and thus inhibit transduction. Consequently, in cases where re-dosing is desirable or unavoidable, the immune response stands to be the biggest challenge that the field must work to overcome.

Attend this workshop to learn about:

• The main concerns regarding immunogenicity and re-dosing
• How likely re-dosing is by analyzing factors of current gene therapy efficacy and safety
• What are the implications for the safety and efficacy of gene therapies if re-dosing is to occur?
• What effect repeat exposure has on the immune response to gene therapies

Workshop Leaders: 

David Favre, Vice President Translational Medicine, Asklepios BioPharmaceutical

Takashi Kishimoto, Chief Scientific Officer, Selecta Biosciences Inc.

Workshop B | Tuesday, August 4 | 11:30am - 1:30pm EDT

Standardization of Assay Design, Validation & Application

Immune monitoring assays form a critical component of the analyses that decision-makers use to qualify or exclude patients from clinical trials. Despite this, the specific assays that are employed in the gene therapy space and the parameters by which patients are admitted to clinical trials (according to NAb presence) can vary greatly.

Attend this workshop to learn about:
• Nonclinical studies of pre-existing immunity to AAV vectors
• How studies of vector immunogenicity can be translated to the selection of patients for clinical trials
• How assays can be utilized alongside a companion diagnostic to better inform decision making regarding immunogenicity

Workshop Leaders: 

Andrew Melton, Senior Scientist II, BioMarin

Brian Long, Associate Director, BioMarin

Klaudia Kuranda, Immunology Leader, Spark Therapeutics

Workshop C | Tuesday, August 4 | 2:00pm - 4:00pm EDT

Outsourcing Immunogenicity Assays to CROs for Gene Therapy

As a growing contingent of the gene therapy field look towards the expertise of CROs for services related to assay development and validation, the importance of choosing the right CRO to work with has never been greater. At a time when there has never been a greater choice of CROs out there to work with, being able to effectively and efficiently vet these solution providers can have huge ramifications for your clinical progress.

Attend this workshop to learn about: 

• What challenges sponsors and CRO’s face with immunogenicity bioanalytical assays
• What factors are important to consider when selecting a CRO and how to vet them
• Do and should CROs vet sponsor relationships?
• How to establish quality and long-standing relationships between sponsors and CROs
• Regulatory aspects of immunogenicity assay for cell and gene therapy: Is the current guidance sufficient? What else is needed?

Workshop Leader: 

Liching Cao, Director Bioanalytical Operations, Sangamo