Agenda

NERDS 2025 Workshop Tentative Agenda

Day 1, Thursday, October 9th 2025

  • 7:45 AM - 9:15 AM: Registration / Light breakfast
  • 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
    • Discussant: Lee-Jen Wei, Harvard T.H. Chan School of Public Health; Moderator: Ran Duan
  • 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
    • FDA case study of Xenopzoyme: A Clinical Dose Escalation Strategy for a Rare Disease Drug Program — Qi Zhang
    • Efficient platform design for screening indications in rare disease — Ziqian Geng
    • Enhancing clinical trials through prognostic score covariate adjustment — Roland Brown
  • 12:10 PM - 1:10 PM: Lunch
  • 1:10 PM - 3:00 PM: Invited talks — Session 2: Gene and Cell Therapy
    • Moderator / Chair: Han Cong
    • Modifier-directed therapeutics in Cystic Fibrosis — Lisa Strug
    • Statistics in CDx Development for Gene Therapies — Shuguang Huang
    • A single-arm confirmatory trial design for gene therapies — Yang Song
    • Model-informed drug development for advanced therapeutics — Avery McIntosh
    • Discussant: Daniel Li
  • 3:00 PM - 3:30 PM: Coffee Break / Networking
  • 3:30 PM - 5:10 PM: Invited talks — Session 3: AI and Machine Learnings
    • Moderator / Chair: Susie Sinks
    • Deep Learning Foundation Models for Medical Image Analysis — Jake Gagnon
    • Artificial Intelligence in Rare Disease Research: Accelerating Relief for the Underserved — Tim Smith
    • AI Co-scientist in Protein-disease Reasoning — Bingxin Zhao
    • Navigating the Scientific Landscape: A Multi-Agent Approach to Research Synthesis — Christian Merrill
  • 5:10 PM - 6:00 PM: Poster Session
  • 6:00 PM - 7:00 PM: Reception

Day 2, Friday, October 10th 2025

  • 7:45 AM - 9:15 AM: Registration / Light breakfast
  • 8:15 AM - 8:20 AM: Day 2 Welcome Address — Ying Zhou
  • 8:30 AM - 10:00 AM: Keynote Sessions
    • Keynote 1 — "Target Learning with Application in Rare Disease Trials" — Mark van der Laan, Professor of Biostatistics, UC Berkeley
    • Keynote 2 — Edward Neilan, Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD)
    • Discussant: Li Wang, Head of Statistical Innovation Group, AbbVie; Moderator: Ying Zhou
  • 10:00 AM - 10:30 AM: Coffee Break / Networking
  • 10:30 AM - 12:10 PM: Invited talks — Session 1: Real-world Evidence and HTA
    • Moderator / Chair: Xiang Zhang
    • Population-adjusted indirect comparisons in rare disease: methods, challenges, and considerations — Yingyi Liu
    • Integrating natural history insights into clinical development planning for gene therapy in a rare neurodegenerative disorder — Alex Sverdlov
    • Beyond MMRM: The crucial role of targeted machine learning in RCTs with small sample size and loss to follow-up — Susan Gruber
    • Propensity score-based unequal matching for rare disease clinical trials with external controls — Yusuke Yamaguchi

Event Locations

  • Breakfast: Charles View Ballroom ~ 16th floor
  • Session Room: Charles View Ballroom ~ 16th floor
  • Lunch: Charles View Ballroom ~ 16th floor
  • Poster Session: Mt Auburn Ballroom ~ 14th floor
  • Reception: Mt Auburn Ballroom ~ 14th floor