Speaker Profile

MD, PhD, Staff Physician, Division of Genetics and Genomics, Boston Children's Hospital

Biography
Timothy Yu, MD, PhD, is recognized as a leader in the field of gene therapy, particularly in the development of personalized gene therapies for individuals with rare genetic disorders. A graduate of Harvard College, he completed MD-PhD training at UCSF, neurology residency at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurogenetics at Boston Children’s Hospital. He joined the faculty of Boston Children’s Hospital in the Division of Genetics and Genomics in 2013. He is also an Associate Member at the Broad Institute Dr. Yu’s research group works at the intersection of genomics, informatics, and neurobiology with a focus on understanding and treating disorders of brain development. He was an early pioneer in methodology for genome sequencing and interpretation, which he applied to discover genes responsible for brain development and autism. He is also recognized for having developed the world’s first individualized genomic medicine: milasen, an antisense oligonucleotide targeting a unique genetic mutation causing a child’s fatal neurodegenerative condition – in under one year. Milasen was the first in a pilot series of individualized therapeutics for severe neurogenetic conditions that his team has since created. It also inspired efforts to advance individualized therapeutics for dozens of neglected genetic conditions worldwide, including the N-of-1 Collaborative, an international coalition founded by Dr. Yu to advance scientific, regulatory, and ethical best practices for this budding field. Dr. Yu has authored more than 80 scientific publications and has been recognized with numerous awards and honors, including the NORD Rare Impact Award, the Global Genes Rare Champion award, and the ANA Denny-Brown Young Neurologic Scholar award. He is dedicated to advancing precision medicine and improving patient outcomes through his research and clinical practice.


Talk
Maturing technologies for precision therapeutics offer hope to patients in the long tail of rare genetic diseases, but often require action at the individual mutation level, stressing an ecosystem organized around tackling one disease at a time. Through clinical examples I will discuss how scientific and regulatory pioneering will be required to enable this promise.


 Session Abstract – PMWC 2024 Silicon Valley

Track 1 - January 24 9.00 A.M.-4.45 P.M.


Track Co-Chairs:
Yael Weiss, Mahzi Tx
Peter Marks, FDA

  • PMWC 2024 Award Ceremony
    Pioneer Honoree: Katherine A. High, AskBio
    Pioneer Honoree: Timothy Yu, Boston Children's Hospital
  • Piloting Interventional Genomics for Orphan Diseases
    Keynote: Timothy Yu, Boston Children's Hospital
  • Accelerated Approval for Small Populations: Biomarkers & Endpoints (PANEL)
    Chair: Peter Marks, FDA
    - Jennifer Puck, UCSF
    - Maria Grazia Roncarolo, Tr1X
    - Katherine A. High, Rockefeller University
    - Jennifer Panagoulias, FAST (Angelman Syndrome Foundation)
    - Timothy Yu, Boston Children's Hospital
  • Novel Clinical Design Approaches in Rare Diseases (PANEL)
    Chair: Adam Shaywitz, Bridge Bio
    - Salvador Rico, Encoded Therapeutics
    - Eric Crombez, Ultragenyx
    - Elizabeth Berri Kravis, Rush University
  • Empowering Patient Advocacy in Rare Disease Therapies (PANEL)
    Chair: Yael Weiss, Mahzi Tx
    - Justin West, KCNT1 Foundation
    - Michael Graglia, Syngap Research Fund
    - Wendy Erler, Alexion Pharmaceuticals
  • Advancing the Frontier: Gene Editing for Rare Diseases (PANEL)
    Chair: Matthew Porteus, Stanford
    - James Chung, Kyverna Therapeutics
    - Lucas Harrington, Mammoth Biosciences
    - Aron Stein, Intellia Therapeutics
  • Advancing the Frontier: Gene and Cell Therapies for Rare Diseases (PANEL)
    Chair: Morten Sogaard, Astellas Gene Therapies
    - Pooja Agarwal, BioMarin Pharmaceutical
    - David Kirn, 4dmt
    - Adrian Veres, Dyno Therapeutics
    - Thomas Wechsler, J&J
  • PMWC Showcase
    - Dominic Borie, Kyverna Therapeutics

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