Human Stem Cell Use: Embryonic Stem Cell

Human Embryonic Stem Cell-Derived Natural Killer Cells for Cancer Treatment

Translational Candidate Human embryonic stem cell (hESC)-derived natural killer (NK) cells to target relapsed/refractory Acute Myelogenous Leukemia (AML) Area of Impact hESC-derived NK cells provide a novel and potent approach to treat relapsed or refractory AML that is resistant to current chemotherapy options. Mechanism of Action hESC-derived NK cells provide a standardized, homogeneous, off-the-shelf cellular […]

Pluripotent stem cell-derived chondrocytes for articular cartilage repair

Translational Candidate We propose to develop a universal, off-the-shelf treatment for articular cartilage repair based on pluripotent stem cell (PSC)-derived chondrospheres Area of Impact The proposed therapy could treat the major cartilage lesions present in more than 10% of people under 50; which often result in pain and arthritis Mechanism of Action Untreated cartilage defects […]

Human Embryonic Stem Cell-Derived Neural Stem Cell Transplants in Amyotrophic Lateral Sclerosis

Translational Candidate Spinal cord injections of human embryonic stem cell (hESC)-derived allogeneic neural stem cells (heNSCs) for treatment of ALS Area of Impact Treatment of Amyotrophic Lateral Sclerosis (ALS) Mechanism of Action Although the exact molecular mechanism of action is unknown, extensive research supports the concept that the behavior of defective astrocytes is key to […]

A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy

Therapeutic Candidate or Device A patch comprised of a layer of stem cell-derived retinal pigmented epithelial (RPE) cells on a supporting matrix that is implanted under the retina Indication Geographic atrophy, the late-stage form of age-related macular degeneration Therapeutic Mechanism The healthy RPE cells on the implant replace dying RPE cells in the eye that […]

Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

Therapeutic Candidate or Device NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells. Indication Focal epilepsy; drug-resistant mesial temporal lobe epilepsy. Therapeutic Mechanism NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells (hESCs) for the prospective treatment of drug-resistant focal epilepsy (MTLE). NRTX-1001 is delivered as a […]

A Phase I, Pilot Study of Human Embryonic Stem Cell-Derived Cardiomyocytes in PaTients with ChrOnic Ischemic Left VentRicular Dysfunction (HECTOR)

Therapeutic Candidate or Device The therapeutic candidate is human embryonic stem cell-derived cardiomyocytes (hESC-CMs) as a new therapy for chronic ischemic cardiomyopathy patients Indication hESC-CMs will be indicated for treatment of heart failure (HF) and for preventing progression to HF in patients with chronic ischemic cardiomyopathy. Therapeutic Mechanism There are two commonly accepted mechanisms by […]

Safety and Tolerability Study of Neural Stem Cells (NR1) in Subjects with Chronic Ischemic Subcortical Stroke

Therapeutic Candidate or Device A human embryonic derived, non-genetically modified neural stem cell (NR1), originally derived from the Wi-Cell H-9 line Indication Patients with chronic motor deficits, from 6 to 60 months after stroke. NR1 cells will be injected into the brain near the site of the stroke. Therapeutic Mechanism The proposed therapeutic mechanism of […]

Clinical trial of directly vascularized islet cell replacement therapy for high-risk type 1 diabetes

Therapeutic Candidate or Device pancreatic progenitor cells in a delivery device that allows direct vascularization Indication high-risk type 1 diabetes including "brittle" diabetes and hypoglycemia unawareness Therapeutic Mechanism People with type 1 diabetes have lost their pancreatic cells that make insulin, and therefore have to self-administer insulin. It is very difficult to manage blood sugar […]

Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI)

Therapeutic Candidate or Device Human embryonic stem cell-derived neural stem cells. Indication Spinal cord injury Therapeutic Mechanism The proposed neural stem cell therapy will be transplanted into a spinal cord injury site, providing cell replacement and a new neuronal relay across the injury site in an effort to promote significant functional improvement. Unmet Medical Need […]

Plurocart: a novel stem cell-based implant for articular cartilage restoration

Therapeutic Candidate or Device Plurocart consists of pluripotent stem cell-derived chondrocytes, seeded onto a scaffold; it is intended to treat damaged cartilage in the knee joint. Indication Plurocart is intended to be surgically implanted in the knee and regenerate injured cartilage – relieving pain and improving function of the joint. Therapeutic Mechanism Plurocart is surgically […]