8:15 AM - 8:20 AM: Day 1 Welcome Address — Ran Duan
8:20 AM - 8:30 AM: Remarks from Industry — Josh Chen
8:30 AM - 10:00 AM: Keynote Sessions
Keynote 1 — "The Science of Possibility: Discovery and
Development of CFTR modulators to treat Cystic Fibrosis" —
Carmen Bozic, Chief Medical Officer, Vertex Pharmaceuticals
Keynote 2 — "The Molecular Bases of Inherited Cardiomyopathies:
From genes to therapies" — Jonathan G. Seidman, Harvard Medical
School Slides
Discussant: Lee-Jen Wei, Harvard T.H. Chan School of Public
Health; Moderator: Ran Duan Slides
10:00 AM - 10:30 AM: Coffee Break / Networking
10:30 AM - 12:10 PM: Invited talks — Session 1:
Innovative Design and Methods in Rare Diseases
Moderator / Chair: Ziqian Geng
Bayesian Approaches for Information Borrowing and Adaptive
Decisions — Yuan Ji Slides
FDA case study of Xenopzoyme: A Clinical Dose Escalation
Strategy for a Rare Disease Drug Program — Qi Zhang Slides
Efficient platform design for screening indications in rare
disease — Ziqian Geng Slides
Enhancing clinical trials through prognostic score covariate
adjustment — Roland Brown Slides