Therapeutic/Technology: Therapeutic Approach
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 […]
Combating Ovarian Cancer with Stem Cell-Engineered Off-The-Shelf CAR-NKT Cell Therapy
Translational Candidate stem cell-based off-the-shelf CAR-NKT cells Area of Impact ovarian cancer (OC) Mechanism of Action The proposed cell therapy candidate, AlloMCAR-NKT cells, can directly kill OC tumor cells through CAR/NKR dual-targeting mechanisms, and can also modulate OC tumor microenvironment (TME) by depleting immunosuppressive tumor-associated macrophages (TAMs) and myeloid-derived suppressive cells (MDSCs) via iNKT TCR-mediated […]
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 AS-241, an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies
Translational Candidate AS-241, an antisense oligonucleotide Area of Impact Amyotrophic Lateral Sclerosis Mechanism of Action AS-241 targets the cryptic exon (CE) of UNC13A and suppresses CE inclusion during RNA splicing, inhibits nonsense-mediated decay, and increases full length mRNA and protein levels Unmet Medical Need To date, therapeutic options for ALS have been limited, and disease-modifying […]
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 […]
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 […]
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 […]
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 […]