Gene-modified, personalized cell therapy – Page 12

Development of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem Cells

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Post published:September 12, 2024
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Therapeutic Candidate or Device TriLeukeVax, an autologous AML vaccine designed to stimulate induction of anti-leukemic cytolytic activity and improve relapse free survival (RFS). Indication Older leukemia patients who achieve remission…

Hematopoietic Stem Cell Gene Therapy for XCGD

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Post published:September 12, 2024
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Therapeutic Candidate or Device Hematopoietic stem and progenitor cells collected from X-CGD patients modified with a highly regulated lentiviral vector Indication X-linked Chronic Granulomatous Disease Therapeutic Mechanism Lentiviral vector (LV)…

Late Stage Pre-Clinical Development of a Cirmtuzumab Based CAR T-cell for the Treatment of ROR1+ Hematological Malignancies

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Post published:September 12, 2024
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Therapeutic Candidate or Device We are developing ROR1 Chimeric Antigen Receptor (CAR) modified T-cells for the treatment of hematological and solid tumor cancers. Indication The target for our therapy are…

Curing Sickle cell Disease with CRISPR-Cas9 genome editing

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Post published:September 12, 2024
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Therapeutic Candidate or Device Blood stem cells collected from individuals with sickle cell disease will have the sickle gene corrected and then given back to the same individual. Indication Sickle…

Late-Stage Preclinical Study of CAR-T Memory Stem Cells Targeting PSMA (P-PSMA-101) for the Treatment of Castrate-Resistant Metastatic Prostate Cancer

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Post published:September 12, 2024
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Therapeutic Candidate or Device Genetically engineered, Centyrin-based, CAR- or CARTyrin-T memory stem cells Indication Castrate-resistant metastatic prostate cancer Therapeutic Mechanism The Centyrin-based chimeric antigen receptor (CARTyrin) cells are cells that…

Ex Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma Patients

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Post published:September 12, 2024
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Therapeutic Candidate or Device Blood stem cells will be genetically engineered to protect them from chemotherapy in glioblastoma patients, producing better patient survival. Indication Patients with newly diagnosed glioblastoma (GBM)…

Genome Editing of Autologous Hematopoietic Stem Cells to Treat Sickle Cell Disease

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Post published:September 12, 2024
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Therapeutic Candidate or Device Autologous blood stem cells edited to correct the sickle cell disease mutation to be given back to the patient as an autologous stem cell transplant Indication…

Ex vivo transduced autologous human CD34+ hematopoietic stem cells for treatment of cystinosis

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Post published:September 12, 2024
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Therapeutic Candidate or Device Transduced Hematopoietic Stem Cells from Peripheral Blood Stem Cells of adults and pediatric patients with cystinosis Indication Autologous hematopoietic stem cell gene therapy for patients with…

Ex Vivo Transduction of the Human Artemis (DCLRE1C) cDNA by Lentiviral Vector AProArt into CD34+ Hematopoietic Cells for Artemis (ART)-Deficient Severe Combined Immunodeficiency (SCID)

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Post published:September 12, 2024
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Therapeutic Candidate or Device Blood-forming stem cells harboring a SCID gene defect, modified to become normal by addition of a correct copy of the Artemis/DCLRE1C DNA repair gene. Indication Treatment…

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