A BioPhorum member survey aimed to gather initial industry benchmarking data and interest in defining an industry position on visible particulate control strategy for cell and gene therapies.
BioPhorum CGT Regulatory Strategy workstream response to revised chapter 5.14 leading to EDQM Pharmeuropa monograph 34.3 gene therapy products for human use, chapter 5.34 and chapter 5.2.12. This content is available to BioPhorum members only.
A cross sub-team team of 18 members from 14 companies submitted feedback on WHO Approach towards the development of a global regulatory framework for cell and gene therapy products (document WHO/BS/2022.2424)
BioPhorum CGT Regulatory Strategy workstream members provide feedback on the FDA draft guidance on considerations for the development of CAR-T cell products .
BioPhorum CGT Regulatory Strategy workstream provided feedback on FDA draft guidance on human gene therapy products incorporating human genome editing .
This member document summarizes consolidated feedback and recommendations from a collaboration of BioPhorum members on the FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early phase clinical trial consultation.
BioPhorum members provide feedback on FDA draft guidance for industry studying multiple versions of a cellular or gene therapy product in an early-phase clinical trial.
Summary BioPhorum member only document detailing feedback and suggested recommendations on EMA draft toolbox guidance for PRIME marketing authorisation applications.
BioPhorum member only summary document providing feedback and recommendations to FDA consultation Human gene therapy for neurodegenerative diseases: draft guidance for industry
This paper highlights some of the challenges to develop potency assays for gene therapies and promotes a potential solution. It seeks to establish Industry alignment on the benefits of a matrix approach and provides high level guidance on how to adopt this strategy towards potency assay development and validation with examples for in vivo and ex vivo GT processes.