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Investigational New Drug, Device and Therapeutics Symposium

Friday, June 16, 2023
4:00 PM – 5:30 PM East Coast USA Time
Location: Grand Salons A-E

Live Stream
Disclosure(s):
Ahmar U. Zaidi, MD: No relevant disclosure to display

Learning Objectives:

  • To understand the development of new therapies in the pipeline.
  • To recognize differences among therapies.
  • To understand how new therapies may improve lives of patients with SCD.

Presentations:

  • 4:00 PM – 4:05 PM East Coast USA Time
    Introduction

    Speaker: Ahmar U. Zaidi, MD (he/him/his) – Agios Pharmaceuticals

  • 4:05 PM – 4:15 PM East Coast USA Time
    A phase 2a, randomized, open-label study to evaluate multiple dosing regimens of subcutaneous ALXN1820 in adult patients with sickle cell disease

    Speaker: Yang Dai, MD, PhD (he/him/his) – Alexion, AstraZeneca Rare Disease

  • 4:15 PM – 4:25 PM East Coast USA Time
    Novo Nordisk in Sickle Cell Disease

    Speaker: Adam Wufsus, PhD (he/him/his) – Novo Nordisk, Inc.

  • 4:25 PM – 4:35 PM East Coast USA Time
    Autologous Cell Therapy Involving Zinc Finger Nuclease-mediated Gene Editing

    Speaker: Ben Hsu, MD, PhD (he/him/his) – Sangamo Therapeutics

  • 4:35 PM – 4:45 PM East Coast USA Time
    Agios sickle cell disease clinical program

    Speaker: Sarah Gheuens, MD, PhD (she/her/hers) – Agios Pharmaceuticals

  • 4:45 PM – 4:55 PM East Coast USA Time
    A phase I/IIA Open-label Dose-Finding Study with Subsequent Double-blind, Placebo-controlled, Randomized Study of L-citrulline in Sickle Cell Disease Presenting to the Emergency Department in Vaso-occlusive Crisis (VOC) in Children, Adolescents and Young Adults

    Speaker: Suvankar Majumdar, MD (he/him/his) – Asklepion Pharmaceuticals

  • 4:55 PM – 5:05 PM East Coast USA Time
    Pfizer Sickle Cell Disease Pipeline

    Speaker: Valerie Cachat, MSN, APRN, CPNP (she/her/hers) – Pfizer

  • 5:05 PM – 5:15 PM East Coast USA Time
    CSL889 (Hemopexin) and Vamifeport as Potential Therapeutics in Sickle Cell Disease

    Speaker: Paul Shore, MD, MS – CSL Behring

  • 5:15 PM – 5:30 PM East Coast USA Time
    Q&A

    Chair: Ahmar U. Zaidi, MD (he/him/his) – Agios Pharmaceuticals

    Speaker: Yang Dai, MD, PhD (he/him/his) – Alexion, AstraZeneca Rare Disease

    Speaker: Adam Wufsus, PhD (he/him/his) – Novo Nordisk, Inc.

    Speaker: Ben Hsu, MD, PhD (he/him/his) – Sangamo Therapeutics

    Speaker: Sarah Gheuens, MD, PhD (she/her/hers) – Agios Pharmaceuticals

    Speaker: Suvankar Majumdar, MD (he/him/his) – Asklepion Pharmaceuticals

    Speaker: Valerie Cachat, MSN, APRN, CPNP (she/her/hers) – Pfizer

    Speaker: Paul Shore, MD, MS – CSL Behring