ARM has spent nearly four years on a mission to answer the question “What is the biggest challenge to cell and gene CMC and how can ARM address it?”. Many of the hurdles to streamlined, cost effective manufacture of cell and gene therapy products derive from a lack of standardized methodologies and training around CMC programs. Borrowing from the ‘A-Mab’ model created for the monoclonal antibody sector, ARM members have produced a similar document titled A-Gene, a case study-based guide to integrating QbD principles in gene therapy CMC programs.
In conjunction with the A-Gene document, which will be released on Thursday, June 24, ARM will be hosting a five-part live webinar series highlighting specific A-Gene chapters. These webinars will occur monthly until culminating in a half-day virtual workshop in November.
Process Development Using Quality by Design (QbD) Principles
Thursday, June 24 | 4pm – 5pm ET
Jessie Sun, Ph.D., Director, Upstream Process Development, Pharmaceutical Development, Ultragenyx Gene Therapy
James Warren, Ph.D., Vice President, Pharmaceutical Development, Ultragenyx Gene Therapy
This webinar will discuss process development and how it aims to establish and characterize a manufacturing process that can be scaled up to manufacture-size batches while continuing to yield a consistent, quality product. Various risk-based approaches are utilized to ensure that the process stays within appropriate limits and meets all safety and quality benchmarks. Note this initial webinar is only available to ARM members.
Process Control Strategy
Wednesday, July 28 | 11am – 12pm ET
Althea Micklewright, Director, PSQA Business Excellence Lead, Pfizer
Iryna Sanders, Ph.D., Senior Principal Scientist, Pfizer
This webinar will discuss how a comprehensive control strategy for a pharmaceutical product (including gene therapy products) is the key to achieving process consistency, product quality, safety, and efficacy. The speakers will describe the approaches to developing a comprehensive control strategy and will provide an overview of the key elements of a control strategy for a gene therapy product.
Upcoming A-Gene Webinars
Upstream & Downstream Processing
August | Date and Time TBD
This webinar will discuss Chapter 5: Upstream & Downstream Processing.
September | Date and Time TBD
This webinar will discuss the vector manufacturing process and how it culminates with the formulation and vialing of the purified vector product. While this stage may come temporally at the end of the process, it is of the utmost importance to begin the gene therapy development process while considering the “end goal”. This webinar outlines the best practices for fill-finish, formulation, characterization, CCIT, and long-term storage and stability that can be used to develop a robust final gene therapy product.
Regulatory Considerations & Comparability
October | Date and Time TBD
This webinar will discuss the regulatory frameworks in the United States, European Union and Japan. It will also provide guidance to sponsors on the best practices and recommendations in managing manufacturing changes during early development by establishing basic concepts in comparability appropriate for gene therapy products.
A-Gene Case Study Virtual Workshop
November | Date and Time TBD
Join ARM for a half-day virtual workshop focusing on the best practices for the gene therapy sector put forth in the A-Gene document.