Blood Disorders – Page 7

TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.

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Post published:November 30, 2025
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Therapeutic Candidate or Device An allogenic, off the shelf, engineered regulatory T cell product that mimics the function of T regulatory Type 1 (Tr1) cells. Indication Prevention of acute and…

Superior forward-oriented b-globin vector for treating Sickle Cell Disease

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Post published:November 30, 2025
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Therapeutic Candidate or Device forward-oriented globin-expressing vector CD34+ HSCs Indication Severe Sickle Cell Disease (SCD) Therapeutic Mechanism Successful gene modified HSC generate RBC with effective repaired hemoglobin in 30% of…

Hematopoietic Stem Cell Gene Therapy for XCGD

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Post published:November 30, 2025
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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)…

IND-enabling activities for a Phase 1 Study of Autologous CD4LVFOXP3 T Cells in Subjects with IPEX Syndrome

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Post published:November 30, 2025
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Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy…

Curing Sickle cell Disease with CRISPR-Cas9 genome editing

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Post published:November 30, 2025
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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…

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

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Post published:November 30, 2025
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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 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:November 30, 2025
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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…

Pre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1

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Post published:November 30, 2025
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Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or "bubble boy disease") is a devastating genetic disease that results in boys not being able…

A suite of engineered human pluripotent stem cell lines to facilitate the generation of hematopoietic stem cells

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Post published:November 30, 2025
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Our goal is to develop tools that address major bottlenecks that have prevented the generation of blood forming stem cells in culture for therapeutic use. To help overcome these bottlenecks,…

Small molecule tools and scale-up technologies to expand human umbilical cord blood stem and progenitor cells for clinical and research use

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Post published:November 30, 2025
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Tens of thousands of patients need bone marrow transplants (BMT) every year, some for bone marrow (BM) cancers and some for inherited diseases such as sickle cell anemia and thalassemia,…

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