Project Objective: Pre-IND/IDE or Equivalent

COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage

Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit to COVID-19 affected patients. Mechanism of Action Our drug candidate, Berzosertib, works as a treatment against COVID-19 by blocking a critical step in virus replication. […]

A Translating Center designed to expedite the development and delivery of high quality stem cell therapies to patients

A successful partnership, resulting in the creation of a Translating Center recognized as the leader in providing end-to-end preclinical development services (preclinical research, CMC, regulatory) to accelerate the availability of stem cell treatments to patients with unmet medical needs. The proposed Translating Center will benefit the lives of patients in CA with serious unmet medical […]

Living Synthetic Vascular Grafts with Renewable Endothelium

Translational Candidate LXW7 coated ePTFE vascular graft achieves rapid endothelization and improved graft patency by capturing endogenous endothelial progenitor cells Area of Impact This technology will produce long-lasting vascular grafts with self-renewable “living” endothelium and improve dialysis patients’ quality of life Mechanism of Action The arteriovenous ePTFE dialysis graft approach is the most common form […]

Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells

Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells undergo genome editing to restore the deficient enzyme. Reintroducing these edited cells replaces the patient's bone marrow, establishing a lasting enzyme reservoir. The bone marrow […]

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 to deliver optogenetic protein to the targeted cells of the retina to restore vision. Unmet Medical Need Geographic atrophy (GA) age related macular degeneration is […]

Neurogenic hydrogel stimulation of stem cells to regenerate radiation-damaged salivary glands

Translational Candidate Ceviginate is a neuromimetic encapsulated in a hydrogel Area of Impact Dry mouth as a result of injury to the salivary glands by radiation therapy for head and neck cancer Mechanism of Action Regenerate damaged salivary gland tissue through neurogenic stimulation of stem cells Unmet Medical Need Current treatment options for dry mouth/xerostomia, […]

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 from within the tumor to kill the tumor from the inside out, they are then further secreted to stimulate local immune cells to kill remaining […]

Pro-regenerative infusible ECM biomaterial for treating acute myocardial infarction

Translational Candidate Injectable biomaterial derived from the natural scaffolding of pig hearts Area of Impact Improving the quality of life of patients with heart attacks Mechanism of Action The proposed mechanism of action is through recruitment of the body's own stem cells and reducing inflammation to heal the heart. Unmet Medical Need The prevalence of […]

Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis

Translational Candidate Modified synthetic noncoding RNA molecule Area of Impact Systemic Sclerosis Mechanism of Action The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes that alleviate cell stress which, in turn, control inflammation and fibrosis in diseases tissue. Unmet Medical Need Systemic sclerosis is an incurable disease with no […]

Adenine Base Editing for Autologous Hematopoietic Stem Cell Gene Therapy of CD3δ SCID

Translational Candidate The translational candidate is Autologous Hematopoietic Stem and Progenitor Cells from CD3δ SCID Patients Corrected by Adenine Base Editing Area of Impact The candidate will provide treatment for a fatal inborn error of immunity (CD3δ SCID) affecting a genetically-isolated population. Mechanism of Action Autologous Hematopoietic Stem and Progenitor cells from CD3δ SCID Patients […]

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