To access the livestream on May 16th, go to broad.io/watchL2C2025.
To view the Poster Session details, go to broad.io/L2C2025poster.
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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. View the detailed agenda below.
We look forward to seeing you on May 16th!
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L2C Symposium | Reaching New Heights
Agenda
8:30am - 9:00am Registration
9:00am - 9:10am Welcome Remarks
Speaker: Anna Greka
9:10am - 9:35am KEYNOTE 1 | Brainwide Silencing of Prion Protein by AAV-Mediated Delivery of an Engineered Compact Epigenetic Editor
Speaker: Jonathan Weissman
9:35am - 9:55am KEYNOTE 2 | From Casgevy to Kilimanjaro: CRISPR Gene Therapy
Speaker: Jimi Olaghere
9:55am - 11:05am SESSION 1 | Next Generation Scalable Technology
Chair: John Doench
9:55am - 10:15am Harnessing Functional Genomics and Machine Learning for Rare Cancers
Speaker: Gabe Griffin
10:15am - 10:35am Rapidly Identifying Drugs for Rare Disorders by Pooled Image-Based Profiling
Speaker: JT Neal
10:35am - 10:55am Multiplexed Optical Pooled Screening for Proteinopathy Therapeutic Discovery
Speaker: Matt Brown
10:55am - 11:05am Q&A for Session 1
11:05am - 11:15am Coffee Break
11:15am - 12:25pm SESSION 2 | Actionable Mechanisms driving Rare Disease
Chair: Jillian Shaw
11:15am - 11:35am Somatic Repeat Expansion as a Shared Therapeutic Target for Repeat Expansion Disorders
Speaker: Ricardo Mouro Pinto
11:35am - 11:55am Characterization of the functional and clinical impacts of CACNA1A de novo missense variants
Speaker: Jen Pan
11:55am - 12:15pm Broadening the Role of Survival of Motor Neuron Protein
Speaker: Lee Rubin
12:15pm - 12:25pm Q&A for Session 2
12:25pm - 1:15pm Lunch
1:15pm - 2:00pm SESSION 3 | Towards Clinical Applications
Chair: Magdalena Riedl Khursigara
1:15pm - 1:35pm In vivo prime editing rescues alternating hemiplegia of childhood in mice
Speaker: Holt Sakai
1:35pm - 1:55pm Toward a Cure for Cerebral Cavernous Malformations: Genetic Insights and Perturb-seq in Action
Speaker: Rajat Gupta
1:55pm - 2:00pm Q&A for Session 3
2:00pm - 3:10pm SESSION 4 | Next Generation Solutions for Rare Disease Patients
Chair: Yael Weiss
2:00pm - 2:20pm Broad Clinical Labs: Sequencing, Registries, and Patient Partners
Speaker: Niall Lennon
2:20pm - 2:40pm Harnessing Cell-Type Specific Gene Regulation for Targeted Gene Delivery
Speaker: Arya Rao
2:40pm - 3:00pm Dystrophin for all
Speaker: Michelle Rengarajan
3:00pm - 3:10pm Q&A for Session 4
3:10pm - 3:20pm Coffee Break
3:20pm - 3:45pm KEYNOTE 3 | Engineering new modalities for the treatment of rare disease
Speaker: Fiona Marshall
3:45pm - 4:15pm PANEL DISCUSSION | Call to Action: Paving the Path to the Clinic
Moderator: David Meeker
Panelists: Fiona Marshall, Wendy Chung, Yael Weiss, John Maraganore, Anna Greka
4:15pm - 4:25pm Q&A for Panelists
4:25pm - 4:30pm Concluding Remarks
4:30pm - 6:00pm Poster Session and Networking
