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The Food and Drug Administration (FDA) Center for Biologics Evaluation and Research is announcing a public workshop entitled “Quantitation of AAV-Based Gene Therapy Products.” 

Purpose of the workshop:

The purpose of the public workshop is to discuss best practices when measuring the concentration of AAV vectors and to provide FDA perspective on qualifying such assays. The public workshop will bring together academia, industry, and other stakeholders involved in research, development, and regulation of AAV-Based gene therapy products.

Background:

AAV vectors are remarkably promising for treating a variety of genetic diseases. The development of safe, high quality AAV vectors requires robust assay methods. The strength of AAV products is most commonly measured in terms of viral genomes. For many manufacturers, qPCR is the assay of choice to measure AAV vector concentration. More recently, manufacturers are also developing innovative methods to quantitate the titer of AAV vectors based on chromatography, capsid detection, light absorption or other methods.

Topics for Discussion at the Public Workshop

• Overview of AAV concentration-determining assays
• Critical elements for assay optimization and qualification
• FDA perceptive on qualification and validation of AAV concentration assays
• Development of AAV concentration assays: Case Studies

Entrance for the public workshop participants (non-FDA employees) is through Bldg. 1, where routine security check procedures will be performed.  For parking and security information, please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

REGISTRATION:

To register for the public workshop, please visit the following website:  https://www.eventbrite.com/e/quantitation-of-aav-based-gene-therapy-products-public-workshop-tickets-49175474167 .  Please provide complete contact information for each attendee, including name, title, affiliation, address, email, and telephone.  Registration is free and based on space availability, with priority given to early registrants.  Persons interested in attending this public workshop must register by November 23, 2018.  Early registration is recommended because seating is limited; therefore, FDA may limit the number of participants from each organization.  There will be no onsite registration, however, you will need to check-in upon arrival.  If you need special accommodations due to a disability, please contact Loni Warren Henderson or Sherri Revell no later than November 30, 2018.

FOR FURTHER INFORMATION CONTACT:

Loni Warren Henderson or Sherri Revell, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 1118, Silver Spring, MD 20993, 240-402-8010, [email protected] (subject line: AAV-Based Gene Therapy Products Workshop).

• Recording, part 1 - Quantitation of AAV-Based Gene Therapy Products Workshop
• Recording, part 2 - Quantitation of AAV-Based Gene Therapy Products Workshop
• Recording, part 3 - Quantitation of AAV-Based Gene Therapy Products Workshop
• Recording, part 4 - Quantitation of AAV-Based Gene Therapy Products Workshop