On November 20th, ARM, ISCT, and Danaher will convene 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. The Exchange will review 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. Successful platforms hold promise to deliver faster and broader patient access to novel gene therapies while maintaining favorable risk-benefit balance and appropriate regulatory rigor. The attached background materials provide additional context.
The Meeting will be live streamed for public viewing. Zoom links will be provided on Monday, November 18th, alongside additional comprehensive pre-meeting materials.
The following brief reflects the motivation and framework behind the facilitated discussion in the all-day scientific exchange on November 20, 2024. The full summary of the meeting will be published in H1 2025.
Please contact Josephine Lembong at jlembong@alliancerm.org.
