Sector Call Summaries: Gene Therapy (2020)
1) SCB Updates
Staff shared key SCB accomplishments from the past year
2) Landscape and Needed Standards Report feedback
Landscape Report feedback:
Add information on which standards are formally/informally FDA-recognized
Include more about framework of how SDOs are involved with SCB
Add more about implementation—how to do so, include links to relevant publications beyond the standards themselves
Add new sorting methods: types of products, processes, materials, equipment, quality/testing issues, supply chain
Capture search results for a pull-down menu of common terms
Include links to relevant documents, places standards can be purchased
Add list of standards related to starting materials for gene therapy products
Needed Standards Report feedback:
The gene therapy sector working group identified several ways to revise this report to make it more user-friendly and valuable, including adding standards needs related to:
Guidelines for vector quantification and validation
Reference standards for adeno-associated virus (AAV) antibodies
Validation of potency and purity assays
Comparability between assays for measuring immune response
3) SCB Project Updates
Shared project updates specific to the gene therapy sector
4) Communications Feedback
Invited feedback on ways to improve communication with stakeholders
5) Workshop/Courses
SCB is planning future workshop opportunities focused around standards for gene therapy. Possible topics include:
Measurement challenges and solutions for viral vector reference materials
Viral vector characterization, including needs, fit-for-purpose concerns, quality testing, and reporting
Data management needs
Impact of manufacturing standards on supply chain management
The new open ballots page was also shared.
6) Other Business
1) COVID-19 UPDATES
Staff provided an update on plans to reschedule in-person SCB-hosted events from summer/fall to later in winter/spring
Shared updates from major standards developing organizations (SDOs), including virtual events and free access to standards relevant to COVID-19
2) SCB UPDATES
Shared key SCB accomplishments from the past year
3) PROJECT UPDATES
Shared project updates specific to the gene therapy sector
4) IN-DEVELOPMENT STANDARDS
Provided updates on current in-development standards in need of expert input
5) VIRAL VECTOR STANDARDS WORKSHOP
Staff shared plans for a September viral vector workshop hosted by the National Institute of Standards and Technology (NIST), Food and Drug Administration (FDA), and SCB. In advance of the workshop, SCB is collecting feedback for NIST and FDA on what viral vector standards are a priority for the field.
Participant feedback on high-priority viral vector standards for the field:
Reference materials and documentary standards related to titer in adeno-associated virus (AAV) vectors
Assay method for genomic titer (e.g., quantitative polymerase chain reaction [qPCR] and Digital Droplet PCR [ddPCR])
Standards addressing how viral vectors are classified and how quality is controlled in manufacturing processes where they are not the main component of therapy (e.g., CAR‑T therapy)
Definition of ancillary material versus raw material for viral vectors
A lexicon for newcomers to the field that captures definitions that are agreed on by experts but not written down
1) SCB News
Staff shared news about upcoming offerings, including standards implementation courses, planned feasibility reports, planned updates to the Regenerative Medicine Standards Landscape report, and a rapid microbial testing article that will be published in Cytotherapy journal.
2) Gene Therapy Standards Updates
Shared project updates specific to the gene therapy sector
3) Identification and Prioritization of Needed Standards
Participants looked at Impact/Urgency ratings of standards from the Community Perspectives: Needed Standards in Regenerative Medicine report and provided suggested changes.
Participants also suggested additional standards needs to add to the report.
SCB will distribute a survey to gather additional feedback on these topics.
4) Upcoming Annual Meeting and SDO Forum
SCB is planning an annual meeting for January 2021 to inform the public about published and in-development standards, increase engagement with standards, and solicit input on standards needs.
There will be a meeting among SDO representatives in November to discuss current standards efforts under way and harmonize efforts, as well as focus on next steps for addressing community-identified needs.
1) SCB UPdates
Staff shared recent updates, including:
Upcoming projects including feasibility assessments, training for regenerative medicine standards implementation, and updating the Regenerative Medicine Standards Landscape report to be an interactive, searchable portal
Plans to hire additional staff to help with upcoming initiatives
2) Cell Therapy Standards Updates
Shared project updates specific to the gene therapy sector
3) Identification and Prioritization of Needed Standards
Participants provided feedback on:
The functional areas with the greatest impact and urgency in the field
The standards areas with the greatest impact and urgency in the field
Which standards areas have the greatest community support
Which standards areas have the greatest scientific consensus
Participants also suggested additional standards needs to add to the report.
SCB will distribute a survey to gather additional feedback on these topics.
4) Upcoming Annual Meeting and SDO Forum
SCB is planning an annual meeting for January 2021 to:
Inform the public about published and in-development standards
Identify and prioritize needed standards
Increase engagement with standards
Answer questions on standards implementation