Call to Action - Paving the Path to the Clinic

Join a panel discussion exploring the critical pathways from rare disease research to clinical implementation, featuring leading experts in biotechnology, genetics, and drug development. Discover how scalable technologies and biological advances are being translated into treatments and cures for patients with rare genetic diseases through insights from moderator David Meeker and distinguished panelists including Fiona Marshall, Wendy Chung, Yael Weiss, John Maraganore, and Anna Greka. Learn about the collaborative efforts between scientists, physicians, patients, patient-led foundations, and biopharma advisors working to accelerate progress in rare disease therapeutics. Gain understanding of the regulatory landscape and strategic approaches needed to successfully navigate the journey from laboratory discoveries to clinical applications, with particular focus on how the Broad Institute's expertise in scaling scientific efforts contributes to breakthrough treatments for underserved patient populations.