Human Stem Cell Use: Adult or Tissue Stem Cell
Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
Therapeutic Candidate or Device CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person Indication Persons with sickle cell disease (adults and adolescents) with repeated, severe painful and lung events are eligible for the clinical trial Therapeutic Mechanism Blood stem cells are harvested […]
A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease.
Therapeutic Candidate or Device ECT-001 graft contains more stem and immune cells than conventional cord blood graft, leading to prompt recovery and better outcomes for patients. Indication Severe Sickle Cell Disease Therapeutic Mechanism Hematopoietic stem cell transplantation is the only cure for severe sickle cell disease. The ECT-001 expanded cord blood cells will replace the […]
Stimulating endogenous muscle stem cells to counter muscle wasting
Evaluation of Gene Therapy Approaches for Autosomal Recessive Hyper IgE Syndrome Due to Mutations in DOCK8
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 […]
RNA-based therapeutics to augment regulatory T cells: a novel approach to treat myocarditis
Research Objective Use human cell therapy insights, specifically CDC-secreted EV analysis, to develop a noncoding RNA chemical entity for myocarditis treatment. Impact Key knowledge gap is how to recruit adaptive immunity to limit inflammation/heart injury in myocarditis. Boosting regulatory T cells is not yet a viable option. Major Proposed Activities Investigate the mechanism(s) by which […]
Vax-CT to promote formation of cancer-specific T memory stem cell for personalized cancer immunotherapy
Research Objective A vaccine booster to induce antigen-specific T memory stem cells that will help enhance the long-term immunity against cancer recurrence Impact Cancer recurrence presents an unmet medical need. Cancer vaccines are promising, but often lack a long-term protection. We will induce T memory stem cells (TMSCs) to boost the long-term immunity. Major Proposed […]
Reversal of dysregulated myelopoiesis in breast cancers and cancer stem cells to boost antitumor immunotherapy
Research Objective A new antiestrogen drug will be developed to stop breast cancer (BC) by direct effects on BC cells including stem cells and indirect action on specific procancer immune cells that surround the cancer. Impact Substantial numbers of patients with localized breast cancer (BC) and essentially all patients with advanced BC become resistant to […]
A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells
Research Objective A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells Impact We aim to develop a novel genome editing based therapy for Artemis-deficient severe combined immunodeficiency that would improve upon prior gene therapies in efficacy, safety, and scalability. Major Proposed Activities Discover optimal approach for nonviral […]
Pharmacological regenerative treatment of idiopathic pulmonary fibrosis targeting the senescent niche of lung progenitor cells.
Research Objective Novel selective pharmacological strategy targeting senescent lung stem cells Impact Idiopathic pulmonary fibrosis along with other interstitial and age-related lung diseases Major Proposed Activities Chracterize senescence cells, including senescent stem and progenitor cells in the lung of patients affected by idiopathic pulmonary fibrosis Screening of a Rubedo Life Science library of senolytic small […]