Development of an Optogenetic Vision Restoration Gene Therapy Using an Engineered Form of Melanopsin
Research Objective The objective of this research proposal is to develop a lead AAV candidate for an optogenetic vision restoration therapy for patients suffering from blindness due to loss of…
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…
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…
CAR T cells targeting abnormal N-glycans for the treatment of refractory/metastatic solid cancers
Research Objective Develop genetically modified chimeric antigen receptor T cells to kill incurable solid cancers by targeting a previously un-targetable tumor associated carbohydrate antigen. Impact Refractory/metastatic solid cancers are almost…
Cardiac Reprogramming Gene Therapy for Post-Myocardial Infarction Heart Failure
Research Objective The candidate is a gene therapy that delivers cardiac reprogramming factors to convert resident cardiac fibroblasts into functioning cardiac muscle. Thus, it is a regenerative cardiac gene therapy.…
Providing a cure for sphingosine phosphate lyase insufficiency syndrome (SPLIS) through adeno-associated viral mediated SGPL1 gene therapy
Research Objective AAV-SPL 2.0 is a gene therapy cure for SPLIS, a lethal childhood disorder of metabolism that causes kidney failure. Our gene therapy may also work in more common…
Generating deeper and more durable BCMA CAR T cell responses in Multiple Myeloma through non-viral knockin/knockout multiplexed genome engineering
Research Objective We will use integrated gene editing techniques to develop a new CAR-T cell therapy for multiple myeloma treatment Impact Develop an improved CAR-T cell therapy for patients with…
Optogenetic Therapy for Treatment of Geographic Atrophy
Translational Candidate Optogenetic gene therapy for patients with geographic atrophy age related macular degeneration. Area of Impact Blindness from geographic atrophy age related macular degeneration Mechanism of Action Gene therapy…
Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma
Translational Candidate SRN-101, an experimental AAV gene therapy for treating glioblastoma Area of Impact Cancer - solid tumors Mechanism of Action Following delivery with an AAV, engineered cytokines are expressed…