On November 20th, 2024, ARM, International Society for Cell & Gene Therapy, and Danaher Corporation convened key stakeholders to explore how to advance gene editing platforms. This Scientific Exchange with the FDA seeks to make progress on therapeutic development for rare conditions that are scientifically feasible but commercially non-viable.
Successful platforms hold promise to deliver faster and broader patient access to novel gene therapies while maintaining a favorable risk-benefit balance and appropriate regulatory rigor. The exchange highlighted case studies from academics and industry developers focused on gene editing, with the objective of defining repeatable platforms that streamline development and regulatory requirements across multiple therapies for rare populations.
ARM, ISCT, and Danaher co-authored a July 2025 publication in Cytotherapy and Nature Biotech exploring how platform-based approaches can be used to develop gene-editing therapies for rare conditions that are scientifically feasible but commercially non-viable. The publications highlight outcomes from our 2024 FDA Scientific Exchange on Gene Editing Platforms, showing that platform approaches can lower development costs and streamline regulatory pathways.
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