Human Stem Cell Use: Somatic Cell
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 […]
Phase I Treatment of Urethral Strictures in Humans
Therapeutic Candidate or Device An engineered urethral segment comprised of autologous urothelial, smooth muscle cells, and progenitor cells within a tubular scaffold. Indication Autologous engineered urethral constructs are for the replacement of urethral strictures that are too long for treatment by conventional methods. Therapeutic Mechanism The autologous engineered urethras are intended to replace the entire […]
Extracellular Vesicles for Ventricular Tachycardia
Therapeutic Candidate or Device CSSHI-2003 (extracellular vesibles derived from cardiohsphere derived cells) Indication Patients afflicted with recurrent fast and irregular heart beating (a.k.a. ventricular tachycardia). Therapeutic Mechanism CSHHI-2003 (CDC exosomes) are lipid vesicles filled with bioactive materials that have anti-inflammatory, anti-fibrotic, and regenerative properties. Decreasing the fibrosis in the heart can decrease the incidence of […]
TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.
Therapeutic Candidate or Device An allogenic, off the shelf, engineered regulatory T cell product that mimics the function of T regulatory Type 1 (Tr1) cells. Indication Prevention of acute and chronic Graft versus Host Disease (GvHD) in patients undergoing mismatched stem cell transplant. Therapeutic Mechanism GvHD is driven by the reaction of allogenic donor T […]
Treatment of the TMJ disc complex
Therapeutic Candidate or Device Tissue implant engineered using expanded, allogeneic chondroprogenitor cells Indication Defects of the temporomandibular joint disc complex Therapeutic Mechanism Though the mechanism has not been established, Hyaleon®️ may initially act as a load bearing structure that fills defects of the temporomandibular joint (TMJ) disc complex. The implant is slowly remodeled allowing for […]
Allogeneic mesenchymal stem cells loaded with oncolytic virus for cancer treatment
Therapeutic Candidate or Device Supernova-1 (SNV1): Allogeneic adipose-derived culture expanded mesenchymal stem cells (AD-MSC) loaded with oncolytic vaccinia virus Indication -Metastatic melanoma -Triple negative breast cancer -Advanced Head & Neck Squamous Cell Carcinoma Therapeutic Mechanism SNV1 will induce direct killing of cancer cells and will modify the tumor microenvironment, converting immunologically "cold" umors into "hot" […]
Genome Editing of Autologous Hematopoietic Stem Cells to Treat Severe Mucopolysaccharidosis type 1 (Hurler Syndrome)
Therapeutic Candidate or Device Autologous blood stem cells edited to restore iduronidase expression Indication Severe Mucopolysaccharidosis Type 1 (MPS1/ Hurler's syndrome) Therapeutic Mechanism Autologous blood stem cells undergo genome editing to restore the production of the missing enzyme. These cells are returned to the patient to replace their bone marrow, where they can secrete functional […]