Ladders to Cures Accelerator Symposium: Session 3 | Towards Clinical Applications

Session 3: Towards Clinical Applications
Chair: Magdalena Riedl Khursigara

In vivo prime editing rescues alternating hemiplegia of childhood in mice
Holt Sakai

Toward a Cure for Cerebral Cavernous Malformations: Genetic Insights and Perturb-seq in Action
Rajat Gupta

We are excited to invite you to the second annual Ladder to Cures Symposium | Reaching New Heights whose goal is to share progress in scalable technologies and advances in biology leading to treatments and cures for patients with rare genetic diseases. This hybrid all-day event will be held on May 16th, 2025 in the Ó³»­´«Ã½ Auditorium and streamed online. Anyone with an interest in rare disease research is invited to attend.

Building on Ó³»­´«Ã½â€™s strengths of scaling scientific efforts to accelerate progress, this symposium will convene scientists, physicians, patients/patient-led foundations and advisors from the biopharma and regulatory space to join the conversation.

For more information visit: /L2C

Ladders to Cures Accelerator Symposium: Reaching New Heights