Human Stem Cell Use: Adult or Tissue Stem Cell

Extracellular Vesicle-Based Therapy for Corneal Scars

Translational Candidate Extracellular vesicles derived from stem cells for the treatment of corneal scars. Area of Impact The candidate aims to restoring vision by reducing the need for corneal transplant and the associated blinding complications. Mechanism of Action The therapeutic candidate is an extracellular vesicle (EV)-based therapy that could regenerate normal cornea tissue and reduce […]

Novel T cell receptor-STEM T cell immunotherapy in lung cancer

Translational Candidate HLA-A*02 restricted CT83 antigen-specific T cell receptor-engineered T cell cells (CT83TCR-STEM T cells for short). Area of Impact Metastatic lung cancer patients who fail to respond to immune checkpoint therapy or prior treatment Mechanism of Action Despite the impressive clinical response to immune checkpoint inhibitors, the majority of lung cancer patients fail to […]

Specific Targeting Hypoxia Metastatic Breast Tumor with Allogeneic Off-the-Shelf Anti-EGFR CAR NK Cells Expressing an ODD domain of HIF-1α

Translational Candidate EGFR-CAR_sIL15 NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with metastatic breast cancer, especially HER2-low breast cancer Mechanism of Action EGFR-CAR_sIL15 NK cells are umbilical cord blood-derived CD34+ HSCs that are engineered to target EGFR and express soluble IL-15, and then are differentiated into NK cells. […]

CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

Translational Candidate Autologous human CD34+ HSPC of patients with Friedreich’s ataxia, modified ex vivo using CRISPR/Cas9 to remove the GAA expansion mutation in frataxin Area of Impact Friedreich’s ataxia (FRDA) for which there is no effective treatment available Mechanism of Action The proposed therapy intervention is intended to impact the target indication of Friedreich's ataxia […]

Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells for Pulmonary Arterial Hypertension

Translational Candidate Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells Area of Impact Pulmonary Arterial Hypertension (PAH), initially associated with Scleroderma (Systemic Sclerosis -SSC), and then applied to other causes of PAH Mechanism of Action Myeloperoxidase (MPO) protein produced by neutrophils plays a critical role in the development of PAH. Disrupting the MPO gene in […]

A human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injury

Translational Candidate The therapeutic candidate is a central nervous system tissue-derived GMP line developed under DISC2-10753 with an established GMP qualified seed bank. Area of Impact The target is chronic cervical spinal cord injury, which represents approximately 59% of clinical spinal cord injury cases. Mechanism of Action Integration of transplanted human neural stem cells is […]

Hematopoietic Stem Cell Gene Therapy for IPEX Syndrome

Translational Candidate Human hematopoietic stem cells that have been modified to express a functional FOXP3 gene to treat patients with IPEX Syndrome Area of Impact These studies will bring stem cell gene therapy for IPEX closer to the clinic especially for those without an HLA match or disease too severe for HSCT Mechanism of Action […]

Targeting stromal progenitors to prevent the development of heart failure

Translational Candidate Monoclonal antibody targeting Ectonucleotide phosphodiesterase/pyrophosphatase 1 (ENPP1) Area of Impact Heart disease: To prevent the development of heart failure after heart attacks Mechanism of Action After myocardial infarction, myofibroblast progenitors express ENPP1. ENPP1 is a type II transmembrane protein that hydrolyzes extracellular ATP and hydrolytic products generated by ENPP1 initiate an inflammatory cascade […]

CAR-Tnm cell therapy for melanoma targeting TYRP-1

Translational Candidate Autologous naive/memory progenitor T cells genetically modified to express a chimeric antigen receptor targeting the Tyrosinase-related protein 1 Area of Impact Patient with melanoma, without response or with relapse after immune checkpoint blockade therapy and patients with rare melanoma subtypes. Mechanism of Action T cells genetically modified to express the 20D7SL CAR detect […]

HSC-Engineered Off-The-Shelf CAR-iNKT Cell Therapy for Multiple Myeloma

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 […]