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Explore CRISPR gene therapy breakthroughs in this keynote presentation from the Ladders to Cures Accelerator Symposium. Discover the journey from Casgevy, the first FDA-approved CRISPR gene therapy, to ambitious future applications through Jimi Olaghere's compelling narrative that connects cutting-edge genetic medicine to personal triumph. Learn about the revolutionary potential of CRISPR technology in treating rare genetic diseases, understand the clinical development pathway that led to Casgevy's approval for sickle cell disease and beta-thalassemia, and gain insights into how gene editing is transforming the landscape of precision medicine. Examine the intersection of scientific innovation and human resilience as the presentation draws parallels between conquering genetic disorders and scaling metaphorical mountains like Kilimanjaro. Understand the current state and future directions of gene therapy research, including scalable technologies that could accelerate treatments and cures for patients with rare genetic diseases. This presentation is part of a symposium designed to convene scientists, physicians, patients, patient-led foundations, and industry advisors to advance rare disease research and therapeutic development.
