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eBriefing

Towards Transformative Therapies for Sickle Cell Disease

Towards Transformative Therapies for Sickle Cell Disease
Reported by
Elyssa Bernfeld

Posted March 06, 2018

Presented By

Biochemical Pharmacology Discussion Group

The New York Academy of Sciences

Overview

Every year, more than 300,000 people are born with sickle cell disease (SCD), a blood disorder marked by frequent vaso-occlusive crises (VOC) in which patients experience fatigue and extreme pain. These crises can lead to organ damage and in the long term, early mortality. To date, the FDA has only approved two treatments for the disease. But improved insight into the complex pathophysiology of SCD has led to the recent development of diverse approaches for treatment, including inhibition of hemoglobin polymerization, prevention and treatment of vaso-occlusive events, and disease corrective gene therapy approaches.

On October 24, 2017, the New York Academy of Sciences and the Biochemical Pharmacology Discussion Group presented Towards Transformative Therapies for Sickle Cell Disease. The event brought together leading researchers and scientists from across the field to discuss key biological mechanisms of the disease and innovative new approaches for treatment, while underscoring the importance of the patient experience.

Speakers

Kenneth I. Ataga (University of North Carolina at Chapel Hill)
Kenneth I. Ataga (University of North Carolina at Chapel Hill)
Marie Ojiambo (Sickle Strong Initiative)
Marie Ojiambo (Sickle Strong Initiative)
Dana Levasseur (Bioverativ)
Dana Levasseur (Bioverativ)
Wally Smith (Virginia Commonwealth University Medical Center)
Wally Smith (Virginia Commonwealth University Medical Center)
Deepa Manwani (The Children's Hospital at Montefiore, Albert Einstein College Of Medicine)
Deepa Manwani (The Children's Hospital at Montefiore, Albert Einstein College Of Medicine)
Philip D. Gregory (bluebirdbio)
Philip D. Gregory (bluebirdbio)
Debra Pittman (Pfizer)
Debra Pittman (Pfizer)
Brahm Goldstein (Global Blood Therapeutics)
Brahm Goldstein (Global Blood Therapeutics)
Vijay G. Sankaran (Boston Children's Hospital, Harvard Medical School)
Vijay G. Sankaran (Boston Children's Hospital, Harvard Medical School)
Tolulope O. Rosanwo (Boston Children's Hospital)
Tolulope O. Rosanwo (Boston Children's Hospital)
Patrick Hines (Functional Fluidics & Children's Hospital of Michigan)
Patrick Hines (Functional Fluidics & Children's Hospital of Michigan)
Session 1: The Patient Perspective and Patient Reported Outcomes
Session 2: Vascular Pathology of Sickle Cell Disease
Session 3: Hemoglobin Reprogramming
Session 4: Novel Therapeutic Approaches