Therapeutic/Technology: Gene Therapy (All)
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 […]
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, […]
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 […]
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 […]
Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients
Translational Candidate Cytomegalovirus antiviral FD-86: DNA Oligonucleotide Therapy Area of Impact HSCT transplant rejection and childhood cognitive and hearing impairment caused by Cytomegalovirus Mechanism of Action The candidate oligonucleotide therapy, FD-86 disrupts viral IE feedback circuitry and breaks homeostatic control of cytotoxic IE proteins inducing apoptosis only in infected cells. Unmet Medical Need Treatment-resistant CMV […]
Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting
Translational Candidate MZ-1866 is a recombinant AAV9 based gene therapy containing the transgene encoding Transcription Factor 4 (TCF4) Area of Impact Pitt Hopkins Syndrome is a rare genetic neurological disease which causes profound disability and severe health impact Mechanism of Action Patients with Pitt Hopkins Syndrome have heterozygous mutations in the Transcription Factor 4 (TCF4) […]
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. […]
Overcoming resistance to standard CD19-targeted CAR T using a novel triple antigen targeted vector
Translational Candidate A tri-specific chimeric antigen receptor (CAR) T cell product that will prevent relapse since targets 3 different tumor antigens Area of Impact Relapse associated with single or double antigen-targeted CAR T cells Mechanism of Action By being able to target 3 different tumor antigens simultaneously on a single CAR product, there is much […]
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 […]