USP Workshop on CMC for Gene Therapy: Regulations, Standards and Quality

USP Workshop on CMC for Gene Therapy: Regulations, Standards and Quality

Register Agenda Program


This workshop will include presentations and case studies from subject matter experts in manufacturing, regulation, process development, and analysis of gene therapy products, intermediates, and raw materials. Panel discussions and networking events will allow audience members to engage the speakers and USP staff on challenges that they face and opportunities to develop best practices and standards to support this emerging therapeutic area.

Key Session Topics

  • Establishing or identifying Critical Quality Attributes (CQA), Critical Process Parameters (CPP), and Key Process Parameters (KPP) for gene therapy products and ancillary materials
  • Advances in manufacturing and purification technologies
  • Setting specifications for release of ancillary materials, drug substance and drug products
    • Identification and characterization of impurities in raw materials, drug substances, and drug products
    • Bioassay development for measurement of potency
    • Developing stability-indicating assays
  • Advanced technologies for identification and characterization of ancillary materials and gene therapy products
    • Next generation release methods to determine DNA size, aggregation, etc.
    • Alternative analytical methods to quantify and report impurities
    • Novel approaches to conserve drug substance/product given small batch sizes
    • Bridging between old and new analytical or manufacturing methods
  • Novel strategies for formulation and delivery
  • Raw and starting material sourcing, qualification, and characterization 
  • Development of documentary standards and reference standard materials
  • CGMP Requirements for manufacturing ancillary materials for gene therapy products

Why Attend?

  • To better understand USP’s standards in development for gene therapy products
  • To learn about new gene therapy targets and novel advances in manufacturing, purification technologies, analytical methods strategies and bioassay
  • To learn regulatory expectations for gene therapies
  • To provide feedback to USP, regulators, and other workshop participants on challenges and potential solutions to help advance this field
  • To network with other manufacturers, scientists, and regulators in this area

Who Should Participate?

  • QA/QC analysts
  • R&D scientists, managers
  • Team members in CMC development projects
  • Manufacturing and process development scientists, managers
  • Regulatory affairs specialists
  • Contract research organizations
  • Contract manufacturing organizations

Pricing Information

Participant Type Regular Fee Per Person
(1-2 individuals from the same organization
Early Bird Pricing
(If registered by January 20, 2020)
Multi-Registrant Pricing
(3 or more individuals from the same organization; all must register at the same time to receive the discount)
Academia $650 $500 $585
Association $650 $500 $585
Government $200 N/A N/A
Industry $1000 $750 $800
Student $200 N/A N/A
Virtual* $395 N/A N/A

*Connection information for Virtual Participants will be sent approximately three (3) business days prior to event.

Advance Registration Deadline:

Advance Registration will be available through Thursday, February 14, 2020; 12:00p.m. EST.

On-site registration will be available starting at 8 am, Tuesday, February 18, 2020 at the USP Meetings Center.

Cancellation and Substitution Policy

Cancellation must be received on or before February 7, 2020 via email to

Refunds will not be issued for cancellations received after this date. There is a $100 processing fee on all cancellations; however, it is permissible to send a substitute registrant from your same organization. Substitutions must be submitted in writing via email to

Invitation Letters for Visas

USP cannot provide an invitation letter to potential workshop attendees; however, once you are registered, USP will provide a registration confirmation that may be utilized to obtain a visa.

Registration Questions

Please contact or +1 301-816-8130.

Local Area Information

Visit our Travel & Transportation page for information on lodging, airports, and local transportation in and around USP’s Rockville, Maryland, campus.

Rockville, MD, USA