Human Stem Cell Use: Adult or Tissue Stem Cell
Allogenic human adipose-derived mesenchymal stem cells for the treatment of knee osteoarthritis
Therapeutic Candidate or Device Intra-articularly injected allogeneic culture-expanded human adipose derived mesenchymal progenitor cells Indication Knee osteoarthritis Therapeutic Mechanism Cartilage regeneration (as determined by cartilage volume increase), immunomodulatory effects Unmet Medical Need There is no approved disease modification therapy for osteoarthritis (OA), and OA is a leading cause of both hospitalization and joint replacement surgery. […]
Ex vivo transduced autologous human CD34+ hematopoietic stem cells for treatment of cystinosis
Therapeutic Candidate or Device Transduced Hematopoietic Stem Cells from Peripheral Blood Stem Cells of adults and pediatric patients with cystinosis Indication Autologous hematopoietic stem cell gene therapy for patients with cystinosis Therapeutic Mechanism Direct transfer of proteins from interstitial macrophages to host cells via long tubular protrusions called tunneling nanotubes, transplantion of autologous HSC modified […]
Regeneration of a Normal Corneal Surface by Limbal Stem Cell Therapy
Therapeutic Candidate or Device cultivated patient-specific corneal epithelial stem cells (limblal stem cells, LSC) Indication Corneal blindness from inability to heal due to corneal epithelial stem cell deficiency as a results of injury Therapeutic Mechanism Limbal stem cell deficiency (LSCD) leads to inability to heal. The most desired treatment is to replace the necessary amount […]
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 […]
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 […]
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 […]
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, […]