2019 Sickle Cell Disease/Advancing Cures Awardees

Awarded: 9/1/2019

Approximately $5 Million

In 2019, six projects were awarded a combined total of over $5 million in research funding to address the underlying cause of sickle cell disease, attacking it at its core by restoring hemoglobin function through various approaches.

2019 SCD/AC Awardees

Daniel E. Bauer, M.D., Ph.D. and Alex Kentsis, M.D., Ph.D.
Dana-Farber/Boston Children's and Memorial Sloan Kettering Cancer Center
Project name: Enhanced Nuclease Delivery for Therapeutic Gene Editing of Hematopoietic Stem Cells in Sickle Cell Disease

James J. Bieker, Ph.D. and Jeffrey A. Glassberg, M.D.
Icahn School of Medicine at Mount Sinai
Project name: Quantitative Modulation of an Erythroid Regulator as a Novel Genetic Target for Sickle Cell Disease

Shuaiying Cui, Ph.D.
Boston Medical Center
Project name: Inhibition of LSD1 by Small Molecule Inhibitors Stimulates Fetal Hemoglobin Synthesis for Sickle Cell Disease

Punam Malik, M.D.
Cincinnati Children's Hospital Medical Center
Project name: Reducing Error-prone Repair for Therapeutic Correction of Sickle Cell Anemia

Stuart H. Orkin, M.D.
Dana-Farber/Boston Children's
Project name: Structure-based Small Molecules for HbF Reactivation

Matthew H. Porteus, M.D., Ph.D.
Stanford University School of Medicine
Project name: Optimization of Gene Correction using Genome Editing in Human Hematopoietic Stem Cells

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