Superior forward-oriented b-globin vector for treating Sickle Cell Disease

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Grant Award Details

Grant Number:
Human Stem Cell Use:
Cell Line Generation:
Award Value:

Grant Application Details

Application Title:

Superior forward-oriented b-globin vector for treating Sickle Cell Disease

Public Abstract:
Therapeutic Candidate or Device

forward-oriented globin-expressing vector CD34+ HSCs


Severe Sickle Cell Disease (SCD)

Therapeutic Mechanism

Successful gene modified HSC generate RBC with effective repaired hemoglobin in 30% of transduced RBC

Unmet Medical Need

This improvement in transduction efficiency and potency will allow optimization of the apheresis process (wherein HSCs are harvested), allowing for lower CD34+ cells/kg for transduction and this achieving a better clinical outcome for more patients enrolled in SCD gene therapy trials

Project Objective

IND preparation and submission

Major Proposed Activities

  • Measure in vivo toxicity of lentiviral vector and success of secondary transplant of CD34+ HSC
  • Manufacture of clinical lentiviral vector and perform clinical dry runs
  • IND Preparation and submission
Statement of Benefit to California:
Our protocol is designed to address multiple shortfalls that have hindered the prospects of widespread use of gene therapy for sickle cell disease (SCD). The vector design results in an improved HSC product, & a decrease in the cost of vector & cell manufacturing. Through our collaborations the goal is to bring a cost-effective approach for treatment of SCD, that can be used even in financially distressed communities leading to potential long term benefits for Californians suffering with SCD.