CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia
Grant Award Details
Grant Type:
Grant Number:
TRAN1-12987
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$3,330,801
Status:
Active
Grant Application Details
Application Title:
CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia
Public Abstract:
Translational Candidate
CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies
Area of Impact
Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative options
Mechanism of Action
The proposed candidate functions as Chimeric Antigen Receptor (CAR) T cell. When the CAR-T cell recognizes tumor cell expressing the designed target, CD72, they are activated and lyse the tumor cell. These "living drugs" can potentially multiply and persist within the body to eliminate tumor cells for long periods of time, or even lead to cures in patients with previously dire outcomes.
Unmet Medical Need
Children and young adults with B-cell acute lymphoblastic leukemia who relapse on available therapeutics need new options for survival. Our therapy aims to address this unmet need through a newly-discovered target and new type of cellular design.
Project Objective
Pre-IND meeting
Major Proposed Activities
CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies
Area of Impact
Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative options
Mechanism of Action
The proposed candidate functions as Chimeric Antigen Receptor (CAR) T cell. When the CAR-T cell recognizes tumor cell expressing the designed target, CD72, they are activated and lyse the tumor cell. These "living drugs" can potentially multiply and persist within the body to eliminate tumor cells for long periods of time, or even lead to cures in patients with previously dire outcomes.
Unmet Medical Need
Children and young adults with B-cell acute lymphoblastic leukemia who relapse on available therapeutics need new options for survival. Our therapy aims to address this unmet need through a newly-discovered target and new type of cellular design.
Project Objective
Pre-IND meeting
Major Proposed Activities
- Process development for manufacturing of CD72 nanoCAR T-cells.
- In vitro and in vivo studies to determine preclinical efficacy and safety of the therapeutic candidate.
- Development of the validated CD72 assay, clinical trial protocol, consent form and clinical SOPs.
Statement of Benefit to California:
The goal of our project is to develop a new therapy for pediatric cancer patients who currently have no other good options. We anticipate this therapy would potentially benefit dozens of children per year in California. Furthermore, successful development of this therapy under the goals of this CIRM award could open its use to an even broader set of children and adults with cancers the express our target of interest, potentially impacting hundreds of individuals per year in our State.
Publications
- J Immunother Cancer (2023): Framework humanization optimizes potency of anti-CD72 nanobody CAR-T cells for B-cell malignancies. (PubMed: 38007238)