HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma
Grant Award Details
Grant Type:
Grant Number:
TRAN1-12250
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$5,281,199
Status:
Active
Progress Reports
Reporting Period:
Final Operational Milestone #5
Grant Application Details
Application Title:
HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma
Public Abstract:
Translational Candidate
stem cell-based off-the-shelf CAR-iNKT cells
Area of Impact
multiple myeloma (MM)
Mechanism of Action
The proposed therapeutic candidate can directly kill MM tumor cells.
Unmet Medical Need
MM remains an incurable disease, with a high relapse rate. The proposed therapeutic candidate can offer a new treatment opportunity for a broad base of MM patients.
Project Objective
Pre-IND meeting with the FDA
Major Proposed Activities
stem cell-based off-the-shelf CAR-iNKT cells
Area of Impact
multiple myeloma (MM)
Mechanism of Action
The proposed therapeutic candidate can directly kill MM tumor cells.
Unmet Medical Need
MM remains an incurable disease, with a high relapse rate. The proposed therapeutic candidate can offer a new treatment opportunity for a broad base of MM patients.
Project Objective
Pre-IND meeting with the FDA
Major Proposed Activities
- Pharmacology study of the therapeutic candidate
- Chemistry/Manufacturing/Control (CMC) study of the therapeutic candidate
- Safety study of the therapeutic candidate
Statement of Benefit to California:
In 2021 alone, it is estimated that over 3,320 Californians will be diagnosed with MM, and over 1,250 Californians will die of this disease. MM results in devastating economic impacts to the state of California, in addition to the substantial economic and emotional impacts on individual patients and their families. The proposed therapeutic candidate can potentially become a life-saving treatment for MM patients and therefore benefit the state of California.
Publications
- Cell Stem Cell (2023): Advancing cell-based cancer immunotherapy through stem cell engineering. (PubMed: 36948187)
- Nat Commun (2025): Allogeneic CD33-directed CAR-NKT cells for the treatment of bone marrow-resident myeloid malignancies. (PubMed: 39893165)
- Cells (2021): Development of Stem Cell-Derived Immune Cells for Off-the-Shelf Cancer Immunotherapies. (PubMed: 34944002)
- Mol Ther (2024): Engineering allorejection-resistant CAR-NKT cells from hematopoietic stem cells for off-the-shelf cancer immunotherapy. (PubMed: 38584391)
- Cells (2022): An Ex Vivo 3D Tumor Microenvironment-Mimicry Culture to Study TAM Modulation of Cancer Immunotherapy. (PubMed: 35563889)
- Trends Pharmacol Sci (2024): Frontiers in CAR-T cell therapy for autoimmune diseases. (PubMed: 39147651)
- Nat Protoc (2025): Generating allogeneic CAR-NKT cells for off-the-shelf cancer immunotherapy with genetically engineered HSP cells and feeder-free differentiation culture. (PubMed: 39825143)
- Nat Biotechnol (2024): Generation of allogeneic CAR-NKT cells from hematopoietic stem and progenitor cells using a clinically guided culture method. (PubMed: 38744947)
- Int J Mol Sci (2023): Graft-versus-Host Disease Modulation by Innate T Cells. (PubMed: 36835495)
- Int J Mol Sci (2022): Interleukin 15 in Cell-Based Cancer Immunotherapy. (PubMed: 35806311)
- Cancers (Basel) (2022): Targeting Immunosuppressive Tumor-Associated Macrophages Using Innate T Cells for Enhanced Antitumor Reactivity. (PubMed: 35681730)
- Int J Mol Sci (2022): Tumor-Localized Administration of alpha-GalCer to Recruit Invariant Natural Killer T Cells and Enhance Their Antitumor Activity against Solid Tumors. (PubMed: 35886891)
