MENU

Accelerating cures for rare childhood diseases using iPSC models

Speaker
  • CEO/Founder, RARE Science
    Biography
      Dr. Christina Waters' broad range of experience in leading medical research in both biotech/large pharmaceutical companies to non-profits converge to specialize in new approaches to personalized medicine and implementation of new innovative research initiatives to accelerate treatments to patients.
      She serves as SVP and GM of the Global Rare Disease Program at WuXi NextCODE and Founder/CEO of RARE Science, a non-profit research organization that accelerates discovery of therapeutic solutions for kids with rare disease. She serves as a Scientific Advisory board member, for Global Genes, which focuses on rare disease advocacy. Dr. Waters received her Ph.D. in Genetics from UC Davis and was a Postdoctoral Scholar and Associate of the Howard Hughes Medical Institute, California Institute of Technology. Dr. Waters completed an NIH Postdoctoral fellowship at University of California, Berkeley, and received a BS degree from San Diego State University. Dr. Waters received her MBA from UCLA.

    Abstract

    Creating inducible Pluripotent Stem Cell (iPSC) collections of rare patients scattered world-wide can lower the barrier of biological discovery of rare disease, provide a platform for potential drug repurposing and seed new drug development programs where there is a great unmet medical need. Currently approximately 8,000 rare diseases have been identified that affect close to half billion people world-wide of which only 5% have available therapies.   More than half, hundreds of thousands, of children have rare disease of which 30% die before their 5th birthday.  Two main challenges are 1) access to genetic sequencing and 2) if sequencing is available only 30-50% of the time the etiology is identified that leads to clinical intervention. Even with identification of a causal gene, understanding how the gene, gene variants and variants in concert with other genomic mutations change biology and lead to a clinical phenotype is needed.  In the first step, there is a need to find a critical number of families with the same rare disease which necessitates searching for them around the globe.  Secondly, the creation and use of iPSC lines once families have been identified enable validation of variance of significance through techniques such as gene editing in addition to revealing causal biological pathways. This methodology can accelerate identification of therapeutic options in the immediate addressing the urgency needed in addition to seeding longer term new targeted drug development programs.                                                           


    Show Resources
    You May Also Like
    MAY 11, 2021 10:00 AM PDT
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    C.E. CREDITS
    MAY 11, 2021 10:00 AM PDT
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    Date: May 11, 2021 Time: 10:00zm PDT Your samples are some of the most valuable assets in the laboratory. After spending countless hours on extraction and preparation, your conclusions could...
    JUN 09, 2021 7:00 AM PDT
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    C.E. CREDITS
    JUN 09, 2021 7:00 AM PDT
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    Date: June 9, 2021 Time: 09 June 2021, 7am PDT, 10am EDT, 4pm CEST cells with dramatic implications on the validity of past cell culture related research. The fact that at least 509 cell lin...
    DEC 02, 2020 8:00 AM PST
    C.E. CREDITS
    DEC 02, 2020 8:00 AM PST
    DATE: December 2nd, 2020 TIME: 08:00am PDT, 11:00pm EDT Bioreactors and shakers are used to cultivate microorganisms, plant, insect, and mammalian cells in different volumes. Upscaling of pr...
    NOV 16, 2020 8:00 AM PST
    C.E. CREDITS
    NOV 16, 2020 8:00 AM PST
    Date: November 16, 2020 Time: 8:00am (PST), 11:00am (EST) CRISPR screening has become the prime discovery tool in modern biomedical research and drug discovery. At the same time, most screen...
    MAR 18, 2021 8:00 AM PDT
    C.E. CREDITS
    MAR 18, 2021 8:00 AM PDT
    DATE: March 18, 2021 TIME: 8:00am PDT Sequencing of bulk cells, single cells, and nuclei is opening doors in the understanding of complex biological processes....
    JUN 03, 2021 12:00 PM CST
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    JUN 03, 2021 12:00 PM CST
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    DATE: June 3, 2021 TIME: 12:00pm SGT This webinar is a virtual event that focuses on utilizing the Gibco CTS Rotea System for Cell and Gene Therapy...

    Accelerating cures for rare childhood diseases using iPSC models


    Specialty

    Research And Development

    Gene Expression

    Dna

    Big Data

    Cancer Research

    Tumor

    Biomarkers

    Cancer

    Earth Science

    Oncology

    University

    Gene Sequencing

    Drug Discovery

    Mass Cytometry

    Cell Culture

    Geography

    Asia50%

    Europe50%

    Registration Source

    Website Visitors100%

    Job Title

    Student50%

    Medical Laboratory Technician50%

    Organization

    Manufacturer - Other50%

    Academic Institution50%


    Show Resources
    Loading Comments...
    Show Resources
    Attendees
    • See more