Project: Evaluating Pre-existing Immunity to Adeno-Associated Viruses

TYPE: standards advancement project

Partners: ISO / TC 276 and the SCB Gene Therapy Sector Working Group

Description:

While gene therapies have significant potential to treat a variety of diseases and health conditions, patients with a pre-existing immunity to adeno-associated viruses (AAVs)—used as the vector for a variety of gene therapies—may face gene therapy treatment complications or sub-optimal outcomes. Despite the impact of this challenge on treatment safety and efficacy, the gene therapy community lacks common language and standard processes for evaluating pre-existing AAV immunity. As gene therapy companies expand and develop therapies requiring larger AAV doses, the gene therapy field will benefit from standards in this area.

SCB is coordinating the development of a consensus-based documentary standard that defines common methods for evaluating pre-existing immunity to AAVs.

Anticipated Availability: Late 2023

Progress:

(learn more about the standards advancement process)

Assembled a Pre-existing Immunity Standards Working Group: SCB has established a working group for this effort and initiated biweekly working group meetings to coordinate the standard advancement effort.

Coordinating White Paper Drafting: The working group has drafted a white paper on the topic and is currently pursuing publication.

Conducted Feasibility Assessment: In August 2019, SCB organized a meeting of 38 experts from multiple stakeholder groups to discuss barriers and opportunities for potential AAV standard topics. SCB developed a feasibility report on the assessment outcomes and planned next steps.

Begin Drafting Standard: SCB presented the standard to ISO and it was accepted as a work item in July 2021. SCB will coordinate the working group as it drafts the standard.

Get Engaged:

To join the working group or learn more about this initiative, please contact SCB.