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Ladders to Cures Symposium 2023 - Advances in Rare Disease Research and Therapies
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- 1 Anna Greka,
- 2 Todd Golub,
- 3 Introduced by Heidi Rehm,
- 4 Introduced by Anna Greka,
- 5 Q&A: Elizabeth Engle and Ted Love,
- 6 Chaired by Paul Blainey,
- 7 In silico assessment of druggability of target-disease pairs; Sumaiya Iqbal
- 8 Multiplexed imaging for subcellular interrogation of rare kidney disease; Matt Brown
- 9 Neurobiological insights from human cellular models; Ralda Nehme
- 10 Mechanism of Feature Learning in Neural Networks; Adit Radhakrishnan
- 11 Session 1 Speakers Q&A,
- 12 Introduced by Anahita Vieira
- 13 Q&A: Simon Frost
- 14 Chaired by JT Neal and Jillian Shaw,
- 15 Mitochondrial disorders; Vamsi Mootha
- 16 Two steps forward, one step back: Development of AAV-based gene therapies for congenital myopathies; Alan Beggs
- 17 Large scale CRISPRi-Perturb-seq in endothelial cells identifies shared disease mechanisms between rare and common vascular disorders; Rajat Gupta
- 18 From patients to clinical trials: translational research in neurodevelopmental disorders; Mustafa Sahin
- 19 Session 2 Speakers Q&A,
- 20 Chaired by Anne O’Donnell-Luria and Anthony Philippakis
- 21 Role of biomarkers in rare disease drug development; Eric Minikel
- 22 Building direct-to-patient portals; Clare Bernard and Mounica Yanamandala
- 23 Actionable genetic targets in the era of gene therapies; Tim Yu
- 24 Session 3 Speakers Q&A,
- 25 Introduced by Alex Burgin
- 26 Moderated by Eric Lander, with panelists Nancy Andrews, Daniel Fischer, Anna Greka, Kerry Jo Lee, David Meeker, Vamsi Mootha, and Heidi Rehm
- 27 Closing remarks: Anna Greka
