Therapeutic/Technology: Therapeutic Approach


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 […]

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 […]

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 […]

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 […]

Pluripotent Stem Cells for Tendon Tissue Engineering

Research Objective We propose to develop a bio-tendon engineered from differentiated pluripotent stem cells for the repair of tendon injuries and degeneration. Impact Rotator cuff tears are the most common causes of shoulder pain that require surgery. However, failure rates range from 20% to 90%. A successful tendon repair will have a major impact on […]

Development of novel small molecules against cancer stem cells in solid cancers

Research Objective To study and optimize lead compounds with multi-kinase activity against existing glioma stem cells and radiation-induced phenotype conversion of non-stem glioma cells into induced glioma stem cells. Impact Glioblastoma is a universally deadly disease. While radiotherapy prolongs survival in glioblastoma it has hit a critical barrier. The proposed study aims to improve the […]

Microgel encapsulated iPSC-derived notochordal cells to treat intervertebral disc degeneration and low back pain

Research Objective We aim to discover an injectable, rejuvenating treatment for painful intervertebral disc degeneration using microtissue-encapsulated iPSC-derived notochordal cells (iNCs) using large animal model Impact Our treatment candidate may allow for a non-invasive stem cell therapy, targeting the underlying pathogenesis of intervertebral disc degeneration, the leading cause of chronic back pain in adults. Major […]

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 […]

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 […]