Ladders to Cures Accelerator Symposium: Session 4 | Next Generation Solutions for Rare Disease Patients

Session 4: Next Generation Solutions for Rare Disease Patients
Chair: Yael Weiss

Ó³»­´«Ã½ Clinical Labs: Sequencing, Registries, and Patient Partners
Niall Lennon

Harnessing Cell-Type Specific Gene Regulation for Targeted Gene Delivery
Arya Rao

Dystrophin for all
Michelle Rengarajan

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