Human Stem Cell Use: Somatic Cell


CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia

Translational Candidate CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies Area of Impact Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative options Mechanism of Action The proposed candidate functions as Chimeric Antigen Receptor (CAR) T cell. When the CAR-T cell recognizes tumor cell expressing the designed […]

Development of MyoDys45-55, a gene editing therapy for Duchenne muscular dystrophy

Translational Candidate A gene editing therapy for Duchenne muscular dystrophy that permanently removes a hotspot region of patient mutations to restore dystrophin. Area of Impact Duchenne muscular dystrophy (DMD), a fatal muscle wasting disease with no cure. Mechanism of Action Our therapy uses CRISPR/Cas9 gene editing to permanently remove a hotspot region of DMD patient […]

MPS II: Plasma cell delivery of iduronate sulfatase

Translational Candidate The patient’s own B cells will be engineered to express the therapeutic enzyme needed for care in Mucopolysaccharidosis type II (MPS II) patients Area of Impact MPS II, a rare genetic disease causing multi-organ symptoms and death by age 15, if not treated. Current treatment does not address major symptoms. Mechanism of Action […]

Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma

Translational Candidate Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Area of Impact Glioblastoma is the most common malignant brain tumor, affecting approximately 3 out of 100,000 people/year in the USA with extremely poor prognosis. Mechanism of Action In our proposed system, the first antigen EGFRvIII, which is […]

Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma

Translational Candidate Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Area of Impact Glioblastoma is the most common malignant brain tumor, affecting approximately 3 out of 100,000 people/year in the USA with extremely poor prognosis. Mechanism of Action In our proposed system, the first antigen EGFRvIII, which is […]

Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors

Therapeutic Candidate or Device Adult Stem-Like T cells engineered with chimeric antigen receptor (CAR) to target cancers expressing IL13Ra2, including melanoma. Indication Advanced cancers that express IL13Ra2, including melanoma. Therapeutic Mechanism When administered to a patient, the engineered T cells will circulate through the blood and tissues to 􀁹nd, recognize and kill tumor cells that […]

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication Mucopolysaccharidosis I (MPSI) is a rare disease that affects predominantly children. Untreated, these patients typically die by the age of 10. Therapeutic Mechanism The therapeutic […]

AB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205

Therapeutic Candidate or Device AB-205 is an intravenously infused medicine containing genetically modified human vascular (endothelial) cells intended to repair damaged tissue Indication Treatment to reduce/prevent severe multi-organ complications from curative high-intensity cancer treatments involving stem cell transplantation Therapeutic Mechanism AB-205 acts through paracrine effects from expression of a rich mixture of reparative proteins called […]

A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

Therapeutic Candidate or Device GNSC-001 is an adeno-associated vector (AAV) expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks IL-1 signaling. Indication Osteoarthritis of the knee Therapeutic Mechanism GNSC-001 is an adeno-associated virus (AAV) gene therapy vector that expresses IL-1Ra and confers long-term IL-1 inhibition following a single, local injection into the […]

Treatment of Severe Aplastic Anemia by induction of mixed chimerism using CD4+ T cell depleted haploidentical donor stem cell transplant

Therapeutic Candidate or Device COH-MC-17: A minimally manipulated half-match donor blood stem cell transplant with a low-toxic conditioning regimen of the transplant host Indication Older (>40 yrs) Severe Aplastic Anemia (SAA) patients that are ineligible for the potentially curative standard stem cell transplant Therapeutic Mechanism Allogeneic stem cell transplant from full-match donor is the only […]