Grant Award Details

Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia
Grant Number: 
DISC2-13405
Investigator: 
Type: 
PI
Award Value: 
$1,177,739
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • Hematopoietic Stem Cell Gene Therapy for Alpha Thalassemia
Public Abstract: 

Research Objective

The objective of this research is to define the final therapeutic candidate for effective hematopoietic stem cell gene therapy to treat severe alpha thalassemia that requires life-ling transfusions

Impact

Severe alpha thalassemia may lead to fetal demise or a life-long need for chronic transfusions with multiple medical complications, especially iron overload from transfusions.

Major Proposed Activities

  • Develop lentiviral vectors carrying human alpha-globin gene for gene therapy of alpha thalassemia (AT) and perform initial tests in a cell line.
  • Test the activities of the vectors in hematopoietic stem cells from healthy donor in culture and by growing in immune deficient mice.
  • Test the activities of the vectors in hematopoietic stem cells from patients with severe AT in culture that produces red blood cells.
  • Test the activities of the vectors in a mouse model of alpha-thalassemia.
  • Determine final therapeutic candidate, complete draft target product profile, and develop assays of purity, activity and identity
  • Request INTERACT meeting.
Statement of Benefit to California: 

AT mutations are most commonly seen in patients of Asian ancestry, particularly SouthEast Asian and Southern Chinese. The prevalence of AT is rapidly growing the United States due to changing immigration patterns. In California, the proportion of the population that is either Asian is 15%, the second highest in the Unites States (behind Hawaii). Thus, patients carrying AT mutations now represent a significant (and ever-growing) public health problem in California.