Therapeutic/Technology: Gene Therapy (All)
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 […]
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 […]
Reprogramming Somatic Cells into iPSCs Engineered with an Anti-PSCA CAR to Develop Allogeneic Off-the-Shelf Cell Therapy to Treat Pancreatic Cancer
Research Objective Our candidate product PSCA-CAR_s15 uiNK is derived from transduction of iPSCs selected from the most ideal source and episomally reprogrammed from mature NK cells or CD34+ cells. Impact Cell Functionality and Quality; Scale up and Manufacture Major Proposed Activities Development and characterization of induced pluripotent stem cells (iPSCs) via somatic cell reprogramming of […]
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 […]
In Utero Treatment of Duchenne Muscular Dystrophy with Non-viral Gene Editing
Research Objective To develop a lipid nanoparticle/mRNA complex that can safely and efficiently edit muscle stem cells in utero, correct the dystrophin mutation, and develop a treatment for Duchenne muscular dystrophy Impact If successful, we will have developed an effective and low-cost treatment for Duchenne muscular dystrophy and a robust method to safely and efficiently […]
Gene Therapy for SLC6A8 Creatine Transporter Disorder
Research Objective The objective is to define a final therapeutic candidate for an effective gene therapy for mutations of the creatine transporter SLC6A8, a major cause of X-linked intellectual disability (ID). Impact This disorder results in severe ID, autistic-like behavior, seizures, & lack or delay of speech with no treatment. Improving brain transduction is essential […]
A Novel Therapy for Sanfilippo B
Research Objective To develop a Stem Cell therapy for Sanfilippo B syndrome. Impact There is no treatment for Sanfilippo syndrome, and other therapeutic approaches have failed in clinics. This proposal will develop a stem cell based therapy for Sanfilippo syndrome. Major Proposed Activities Generation of universal donor Embryonic Stem Cells (ESC H1) using state of […]
Novel Lipid Nanoparticles for Enhancing eNOS Synthesis for Cardioprotection Post Myocardial Infarction
Research Objective Our therapeutic candidate is a lipid nanoparticle that delivers a therapeutic dose of mRNA to the human heart, which transiently transfects of cells within the heart to improve function after an MI. Impact There is evidence for eNOS therapy as a cardioprotectant post MI; however, the progression of to the clinic has stalled […]
Autologous stem cell-derived interneuron cell therapy for spinal cord injury (SCI)
Research Objective Functional restoration following spinal cord injury using defined excitatory and inhibitory spinal interneuron progenitor cell transplantation Impact The development of a stem cell derived progenitor cell therapy with disease modifying potential for spinal cord injury Major Proposed Activities Define and characterize DI4 GABA and V3 Glut spinal interneuron progenitor cells derived from pluripotent […]
Modified RNA-Based Gene Therapy for Cardiac Regeneration Through Cardiomyocyte Proliferation
Research Objective Efficacious and safe intramyocardial delivery of modified mRNA encoding cell cycle regulators as a gene therapy for cardiac regeneration through resident cardiomyocyte proliferation. Impact This project would provide disease-modifying gene therapy for people with heart failure due to loss of cardiac muscle, a leading cause of deaths in the US, using novel modified […]