Therapeutic/Technology: Therapeutic Approach


Ex Vivo Transduction of the Human Artemis (DCLRE1C) cDNA by Lentiviral Vector AProArt into CD34+ Hematopoietic Cells for Artemis (ART)-Deficient Severe Combined Immunodeficiency (SCID)

Therapeutic Candidate or Device Blood-forming stem cells harboring a SCID gene defect, modified to become normal by addition of a correct copy of the Artemis/DCLRE1C DNA repair gene. Indication Treatment of severe combined immunodeficiency due to defects in the Artemis/DCLRE1C gene. Therapeutic Mechanism Severe combined immunodeficiency (SCID) is characterized by absence of T and B […]

Development of AB-110: genetically-modified endothelial cells plus expanded cord blood hematopoietic stem cells as a transplantation therapy

Therapeutic Candidate or Device CD34+ Cord-Blood Derived Hematopoietic Stem and Progenitor Cells Co-Cultured and Co-Infused with Genetically Modified Endothelial Cells Indication Life-threatening high-risk hematologic malignancies including leukemia and lymphoma Therapeutic Mechanism Appropriately matched cord-blood derived stem and progenitor cells which has a significant population of both short- and long-term stem cells (expanded by genetically modified […]

Development of a Chondrogenic Drug Candidate Targeting Resident Mesenchymal Stem Cells for the Treatment of Osteoarthritis

Therapeutic Candidate or Device The therapeutic candidate is a drug-like small molecule that promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes. Indication Osteoarthritis and cartilage injury Therapeutic Mechanism The therapeutic candidate, through intra-articular administration, promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes. The newly formed chondrocytes replace the dead chondrocytes, synthesize and secret […]

IND-enabling study of subretinal delivery of human neural progenitor cells for the treatment of retinitis pigmentosa

Therapeutic Candidate or Device human fetal cortex derived neural progenitor cells (CNS10-NPC) Indication To stabilize disease progression and maintain ocular integrity and vision for RP patients. This approach can be applied to more prevalent AMD. Therapeutic Mechanism A single subretinal injection of Human neural progenitor cells (CNS10-NPC) offers dramatic preservation of vision. CNS10-NPC engraft and […]

Development of a Chondrogenic Drug Candidate Targeting Cartilage-residing Mesenchymal Stem Cells for the Treatment of Osteoarthritis

Osteoarthritis (OA) is the most prevalent musculoskeletal disease affecting nearly 27 million people in the United States, and is the leading cause of chronic disability in the United States. Current therapeutic options are limited to pain or symptom-modifying drugs and joint replacement surgery; no disease-modifying drugs are approved for clinical use. OA is characterized by […]

Embryonic Stem Cell-Derived Chondroprogenitor Cells to Repair Osteochondral Defects

Surgical approaches to the treatment of focal cartilage defects can be classified into repair, replacement, and regeneration therapies. Marrow stimulation procedures such as microfracture result in a repair tissue that is predominantly fibrocartilaginous in nature, which is mechanically less durable than articular cartilage and survives on average 7 years before requiring another procedure. Osteochondral grafting […]

Scaffold for dermal regeneration containing pre-conditioned mesenchymal stem cells to heal chronic diabetic wounds

The goal of our CIRM-funded Early Translational (ETA) grant was to engineer a product to improve healing in diabetic foot ulcers, a devastating consequence of diabetes that occurs in about 25% of all diabetic patients and is responsible for most leg or foot amputations. More than 6 million people in the US and up to […]

A hNSC Development Candidate for Huntington’s Disease

Huntington’s disease (HD) is a devastating degenerative brain disease with at least a 1 in 10,000 prevalence that inevitably leads to death. These numbers do not fully reflect the large societal and familial cost of HD, which requires extensive care-giving. HD has no effective treatment or cure and symptoms unstoppably progress for 15-20 years, with […]

Pre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1

Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or “bubble boy disease”) is a devastating genetic disease that results in boys not being able to form an immune system. If they are exposed to the environment for even a short period of time they can get infections that a […]

Placental Stem Cells for the In Utero Treatment of Spina Bifida

Myelomeningocele – also known as spina bifida – is a devastating and costly defect that causes lifelong paralysis as well as bowel and bladder incontinence in newborns. It is one of the most common birth defects worldwide, with four children in the United States born with spina bifida every day. Spina bifida affects the physical, […]