Program Type: Translation
Ex Vivo Modified Hematopoietic Stem Cells to Treat Danon Disease
Translational Candidate The candidate is CD34+ HSPCs transduced ex vivo with a LAMP2 lentiviral vector. Area of Impact Danon Disease, Lysosomal Storage Diseases, Drug Development for Rare Disease Mechanism of Action Engrafted HSPC progeny will supply normal LAMP2B to the heart, liver, muscle, and brain via lysosomal cross-correction. Specifically, macrophages transfer lysosomes containing LAMP2B to […]
Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting
Translational Candidate Adeno-associated viral vector serotyped for hepatic tropism to express Arginase 1 in hepatocytes. Area of Impact Developing a new therapy for Arginase Deficiency, where present day this is minimally effective at best. Mechanism of Action The proposed clinical candidate is a virus that has been altered to carry the gene for & produce […]
Targeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibody
Translational Candidate s15.BsAb.BCMA-CAR NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with multiple myeloma Mechanism of Action s15.BsAb.BCMA-CAR NK cells are umbilical cord blood-derived CD34+ hematopoietic stem cells that are engineered to include two components of the BCMA-CAR and the anti-NKG2D-anti-GPRC5D BsAb. s15.BsAb.BCMA-CAR NK cells can launch dual […]
AAV Gene Therapy for Treating Congenital Hereditary Endothelial Dystrophy associated with Biallelic SLC4A11 Mutations
Translational Candidate Therapeutic candidate rAAV8-EF1α-hSLC4A11 is a recombinant AAV vector with single-stranded cDNA encoding the wild-type human SLC4A11 protein. Area of Impact The candidate is for treatment of congenital hereditary endothelial dystrophy, an orphan disease associated with congenital corneal opacification. Mechanism of Action Therapeutic candidate rAAV8-EF1α-hSLC4A11 introduces normal copies of human SLC4A11 gene into the […]
Hematopoietic Stem Cell Gene Therapy for Wiskott Aldrich Syndrome
Translational Candidate Human hematopoietic stem cells that have been modified to express a functional WAS gene to treat patients with Wiskott Aldrich Syndrome (WAS) Area of Impact These studies will bring stem cell gene therapy for WAS closer to the clinic especially for those without an HLA match or disease too severe for HSCT Mechanism […]
Development of Autologous Cell Replacement Therapy for Parkinson’s Disease: Path to Personalized Treatment
Translational Candidate autologous iPSC-derived dopaminergic progenitor cells Area of Impact Parkinson's Disease Mechanism of Action Autologous iPSC-derived dopaminergic progenitor cells represent a promising strategy to replace the nigrostriatal cells which are lost in Parkinson's Disease (PD). While approaches using fetal tissue / allogeneic stem cells show great promise, they are not sufficiently personalized to provide […]
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 […]
Hematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV Infection
Translational Candidate A blood forming stem cell based therapy to treat HIV infection and enhance HIV immunity. Area of Impact We are seeking to develop a therapy to treat HIV infection to replace standard drug therapy and cure people of the virus. Mechanism of Action We are seeking to develop a gene therapy that modifies […]
Telomerase mRNA for short telomere related pulmonary fibrosis
Translational Candidate Nucleoside-modified mRNA encoding telomerase reverse transcriptase (TERT) encapsulated in a lung-targeting lipid nanoparticle delivered intravenously. Area of Impact 1) Idiopathic pulmonary fibrosis (IPF) and 2) Other diseases and conditions caused or exacerbated by short telomeres. Mechanism of Action TERT mRNA LNPs transiently extend the telomeres of the alveolar epithelial cells of the lung, […]
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 […]