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Clinical Drug Development & Investigations
Symposia

Symposium 10: First-in-Patient Dose Prediction for Adeno-associated Virus-mediated Gene Therapy

Tuesday, September 12, 2023
8:00 AM - 11:30 AM PT
Location:Grand Ballroom E-H
CE: 3.0

    Co-Chair & Faculty(s)

  • Xiaofei Wang, PhD

    Clinical Pharmacologist, CBER/OTP/Office of Clinical Evaluation/Div of General Medicine
    US Food & Drug Administration

  • Peng Zou, PhD

    Director, Quantitative Clinical Pharmacology
    Daiichi Sankyo Co Ltd

    • Faculty(s)

  • Kefeng Sun, PhD

    Clinical Pharmacology
    Takeda Pharmaceutical Co Ltd

  • Tao Zhang, PhD (he/him/his)

    Assistant Professor, Pharmaceutical Sciences
    Binghampton Univ

The target audience for this Symposium is clinical pharmacologists involved in clinical development of gene therapy products.

Goals & Objectives:

Following the completion of this activity, the learner will be able to:
  • Identify major knowledge gaps in dose extrapolation from preclinical species to humans for adeno-associated, virus-mediated gene therapy;
  • Articulate the major mechanisms which cause poor human dose prediction from preclinical models;
  • Apply allometric scaling approaches and interspecies normalization approach to predict First-in-Patient dose for adeno-associated, virus-mediated gene therapy.

Presentations:

  • Clinical Pharmacology Considerations for Adeno-associated, Virus-mediated Gene Therapy: A Regulatory Perspective

    Co-Chair & Faculty: Xiaofei Wang, PhD – US Food & Drug Administration

  • First-in-Patient Dose Prediction for Adeno-associated, Virus-mediated Gene Therapy

    Co-Chair & Faculty: Peng Zou, PhD – Daiichi Sankyo Co Ltd

  • Interspecies Scaling of Transgene Products for Intramuscular & Intravesical Gene Therapies

    Faculty: Tao Zhang, PhD (he/him/his) – Binghampton Univ

  • Clinical Pharmacology Considerations for Recombinant Adeno-associated, Virus-based Gene Therapy: Focus on Biodistribution, Immune Response & Durability

    Faculty: Kefeng Sun, PhD – Takeda Pharmaceutical Co Ltd

  • Faculty Panel Discussion, Questions & Answers, Learner Feedback & Evaluation