Advancing rare disease therapies: Breakthroughs and barriers in rare disease drug development

Join Dr. Harriet Holme, Drug Development Clinician at Weatherden and Chair/Founder of PCD Research, and Charlotte Guzzo, Co-Founder and COO of Sano Genetics, as they discuss the critical need for innovation in rare disease research and treatment.

In this webinar, Harriet and Charlotte share insights into the challenges of rare disease drug development, from overcoming diagnostic delays to addressing regulatory hurdles and economic barriers. Drawing on their personal and professional experiences, they talk about the power of collaboration, cutting-edge tools like CRISPR, and advancements in newborn genetic screening to accelerate the development of life-changing therapies.