Disease Focus: Immune Disease

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase – Severe Combined Immunodeficiency (or ADA-SCID) Therapeutic Mechanism This project will lead to a License Application for OTL-101 as a treatment for ADA-SCID. The patient’s own stem cells ("autologous") are […]

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Therapeutic Candidate or Device The therapeutic product candidate is autologous CD34+ hematopoietic stem/progenitor cells (HSPC) transduced with the G1XCGD lentiviral vector. Indication The target indication is for the transplantation of patients with severe X-linked Chronic Granulomatous Disease (XCGD) lacking matched donors. Therapeutic Mechanism Transplantation and engraftment of gene-corrected autologous HSPC after reduced intensity conditioning for […]

Development of OSSM-007, cryopreserved interferon-gamma primed allogeneic MSCs, for treatment of steroid refractory acute graft versus host disease

Therapeutic Candidate or Device OSSM007: cryopreserved, interferon-gamma-primed bone marrow mesenchymal stem cells (BM-MSC) Indication acute Graft versus host disease (aGVHD) resulting from hematopoietic cell transplantation Therapeutic Mechanism Immunomodulation of host-reactive T cells to induce operational tolerance of donor HSC-derived lymphocytes through direct cell-to-cell contact and secreted paracrine factors. Interferon-gamma priming of MSCs enhances therapeutic effects […]

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

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 X-linked (IPEX) syndrome Therapeutic Mechanism Administration of autologous CD4LVFOXP3 that constitutively and stably express wild-type FOXP3 gene will replace the lack of functional regulatory T […]

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)

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 of severe combined immunodeficiency due to defects in the Artemis/DCLRE1C gene. Therapeutic Mechanism Severe combined immunodeficiency (SCID) is characterized by absence of T and B […]

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

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 to form an immune system. If they are exposed to the environment for even a short period of time they can get infections that a […]

Generation of a functional thymus to induce immune tolerance to stem cell derivatives

Stem cell research offers the promise of replacing missing or damaged tissues in the treatment of disease. Stem-cell-derived transplants still face problems with rejection as in traditional organ transplants. Several drugs can prevent rejection but also suppress the immune system, leaving patients vulnerable to infections and cancer. To avoid rejection without using drugs requires re-educating […]

A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

Successful stem cell therapy requires the replacement of diseased or dysfunctional stem cells with healthy ones. These healthy stem cells can come from either a donor or can be stem cells that are modified by gene therapy techniques. One important step in this process of repair and replacement is to eliminate the existing diseased cells […]

Gene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID

Artemis is a chemical in all cells in the body that is essential for the normal development of the immune system and repairing damaged DNA. Artemis deficiency (AD) causes Severe Combined Immunodeficiency (SCID-A), a “bubble baby” syndrome associated with increased sensitivity to radiation and chemotherapy. SCID-A is hard to treat with a bone marrow stem […]

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