Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients

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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,…

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Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting

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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…

Continue ReadingDevelopment of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting

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

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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…

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

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

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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)…

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

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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…

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CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia

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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…

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