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 […]
Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.
Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy patients with reduced immunosuppression regimen and universal recipient eligibility Mechanism of Action The proposed therapeutic candidate comprises inhibitory interneurons that would be delivered in a […]
Targeting pancreatic cancer with Allogeneic Off-the-Shelf PSCA-CAR NK cells
Translational Candidate PSCA-CAR_sIL15 NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with metastatic pancreatic cancer or other cancers that also highly express PSCA Mechanism of Action PSCA-CAR_sIL15 NK cells are umbilical cord blood-derived CD34+ HSCs that are engineered to target PSCA and express soluble IL-15, and then are […]
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 […]
Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND
Translational Candidate Adeno-associated viral vector serotyped for tropism to express guanidinoacetate methyltransferase in hepatocytes and brain cells. Area of Impact Developing a new therapy for Guanidinoacetate Methyltransferase Deficiency, where present day this is minimally effective at best. Mechanism of Action The proposed clinical candidate is a virus that has been altered to carry the gene […]
A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation
Translational Candidate Human pluripotent stem cell expanded and differentiated dopaminergic precursor cells to treat Parkinson’s Disease at high quantity and high quality Area of Impact This candidate uses a 3D cell culture technology that addresses the manufacturing bottleneck and creates a high-quality scalable cell therapy. Mechanism of Action Dopamine producing cells are implanted into the […]
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 […]
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 […]