Uploaded June 2025 | Updated September 2026, 2 weeks ago

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Ladders to Cures Accelerator Symposium: Reaching New Heights
Friday, May 16, 2025
Broad Institute

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

Broad Clinical Labs: Sequencing, Registries, and Patient Partners
00:00 Speaker: Niall Lennon

Harnessing Cell-Type Specific Gene Regulation for Targeted Gene Delivery
25:39 Speaker: Arya Rao

Dystrophin for all
38:20 Speaker: 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 Broad Auditorium and streamed online. Anyone with an interest in rare disease research is invited to attend.

Building on Broad’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: broadinstitute.org/L2C


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