Superior forward-oriented b-globin vector for treating Sickle Cell Disease
Grant Award Details
Grant Type:
Grant Number:
CLIN1-14792
Investigator(s):
Award Value:
$4,619,455
Status:
Pre-Active
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
Indication
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
forward-oriented globin-expressing vector CD34+ HSCs
Indication
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.