Therapeutic/Technology: Immune tolerance/management
Stem cell differentiation to thymic epithelium for inducing tolerance to stem cells
The thymus is an organ that plays a key role in controlling immune responses and immune tolerance. The thymus promotes immune tolerance by deleting and removing self-reactive T cells from the immune system. In addition, the thymus also helps drive the production of important suppressor T cell populations like regulatory T cells that also control […]
Immune cloaking of human stem cell-derived insulin-producing cells for curative cell therapy without immunosuppression
Research Objective The goal of our project is to generate cells for replacement therapy in patients that have reduced ability to trigger the immune response in the recipient and therefore escape rejection. Impact We test a novel molecule that reduces immune activation upon transplantation of allogeneic stem cell products that can improve graft survival and […]
Engineering pluripotent stem cells for universally available, off-the-shelf T cell therapies
Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a range of diseases. Impact We will develop new gene editing approaches to overcome the block to T cell development from iPSC that occurs when key […]
Orthogonal IL2 Receptor Transduced Regulatory T Cells for Clinical Application
Research Objective Genetically modified cells that help control immune reactions Impact Improved treatment for patients undergoing transplantation Major Proposed Activities To identify the optimal source of regulatory T cells for introduction of the novel IL2 receptor To test the impact of a novel IL2 protein on activating the regulatory T cells To determine the optimal […]
A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering
Research Objective We propose to discover genome- and epigenome-edited allogeneic T cells engineered to selectively target JCV as a potentially lifesaving treatment for progressive multifocal leukoencephalopathy (PML). Impact If successful, we would revolutionize treatment for PML patients in whom T cell immunity cannot be restored or boosted and unable to wait for adoptive transfer of […]
Generation of human universal donor iPS cells
Translational Candidate Universal donor cell that is a genetically-engineered iPSC clone and is equipped with a safety switch. Area of Impact Regenerative medicine including replacement therapies affected by immune rejection by host immune cells. Mechanism of Action The universal donor cell mitigates immune rejection by host immune cells and is equipped with a suicide gene […]
Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant
Therapeutic Candidate or Device COH-MC-17: A minimally manipulated half-match donor blood stem cell transplant with a low-toxic conditioning regimen of the transplant host Indication Older (>40 yrs) Severe Aplastic Anemia (SAA) patients that are ineligible for the potentially curative standard stem cell transplant Therapeutic Mechanism Allogeneic stem cell transplant from full-match donor is the only […]
Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant
Therapeutic Candidate or Device Mobilized peripheral blood stem cells from allogeneic donors depleted of TCRαβ+ T cells/CD19+ B cells Indication Renal failure due to one of four genetic and/or immunological diseases Therapeutic Mechanism 1. By using haploidentical parents, we will expand the number of potential living donors, and 2. with pre-HSCT immune ablation we can […]