Towards Transformative Therapies for Sickle Cell Disease

Towards Transformative Therapies for Sickle Cell Disease

Tuesday, October 24, 2017

The New York Academy of Sciences, 7 World Trade Center, 250 Greenwich St Fl 40, New York, USA

Presented By

Biochemical Pharmacology Discussion Group

The New York Academy of Sciences

 

Sickle Cell Disease (SCD) is a debilitating blood disorder with high mortality rates, yet to date only a single therapy for the disease has been approved by the FDA. Markedly, 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. This symposium will discuss these recent developments highlighting key biological mechanisms of disease and reviewing relevant clinical science around SCD, while underscoring the importance of the patient experience.

Call for Abstracts

Abstract submissions are invited for a poster session, and two abstracts will be selected for short talks. For complete submission instructions, please visit our online portal. The deadline for abstract submission is August 25, 2017.

Registration

Member
$60
Nonmember Academia, Faculty, etc.
$105
Nonmember Corporate, Other
$160
Nonmember Not for Profit
$105
Nonmember Student, Undergrad, Grad, Fellow
$70
Member Student, Post-Doc, Fellow
$25
Deadline:
5
days
left

Sponsors

The Biochemical Pharmacology Discussion Group is proudly supported by:

      

Regeneron


ACS New York Section

Premiere Supporter

Promotional Partners

Sickle Cell Advisory Consortium of New York

The William E. Proudford Sickle Cell Fund

The Sickle Cell Cure Foundation

Sickle Cell Community Consortium

National Organization for Rare Disorders

Tuesday

October 24, 2017

8:30 AM

Breakfast and Registration

9:00 AM

Introduction and Welcome Remarks

Speakers

Sonya Dougal, PhD
The New York Academy of Sciences
Patrick Hines, MD, PhD
Functional Fluidics & Children's Hospital of Michigan

Session 1: The Patient Perspective and Patient Reported Outcomes

9:15 AM

Patient's Perspective

Speaker

Marie Ojiambo
Sickle Strong Initiative
9:45 AM

Transformative Pain Therapy in Sickle Cell Disease

Speaker

Wally Smith, MD
Virginia Commonwealth University Medical Center
10:15 AM

A Natural History Study to Evaluate Longitudinal Changes in Sickle Cell Disease

Speaker

Debra Pittman, PhD
Pfizer
10:45 AM

Networking Coffee Break

Session 2: Vascular Pathology of SCD

11:15 AM

Therapeutics in development to treat SCD Vasco-Occlusive Events

Speaker

Deepa Manwani, MD
The Children's Hospital at Montefiore, Albert Einstein College Of Medicine
11:45 AM

A Phase II, Randomized, Placebo-Controlled Trial of Crizanlizumab in Sickle Cell Disease

Speaker

Kenneth I. Ataga, MD
University of North Carolina at Chapel Hill
12:15 PM

Data Blitz session

Speaker

Early Career Investigator Selected from Abstracts
12:20 PM

Data Blitz session

Speaker

Early Career Investigator Selected from Abstracts
12:25 PM

Data Blitz session

Speaker

Early Career Investigator Selected from Abstracts
12:30 PM

Networking Lunch and Poster Viewing

Session 3: Hemoglobin Reprogramming

2:00 PM

New Therapeutic Avenues for Fetal Hemoglobin Induction in Sickle Cell Disease

Speaker

Vijay G. Sankaran MD, PhD
Boston Children's Hospital, Harvard Medical School
2:30 PM

Disrupting a BCL11A Enhancer with Zinc Finger Nucleases for the Treatment of Sickle Cell Disease and β-Thalassemia

Speaker

Dana Levasseur, PhD
Bioverativ
3:00 PM

Networking Coffee Break

Session 4: Novel Therapeutic Approaches

3:30 PM

GBT440, a Novel Sickle Hemoglobin Polymerization Inhibitor

Speaker

Josh Lehrer, MD
Global Blood Therapeutics
4:00 PM

Lentiviral Vector Gene Therapy for Sickle Cell Disease

Speaker

Philip D. Gregory, D. Phil
bluebirdbio
4:30 PM

F1000 Poster Prize Presentation

Speaker

Sunita Hett, PhD
Pfizer
4:40 PM

Closing Remarks

Speaker

Ellis Neufeld, MD, PhD
Boston Children’s Hospital
5:00 PM

Networking Reception and Poster Viewing

6:00 PM

Adjourn