Program Type: Clinical
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 […]
Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant
Therapeutic Candidate or Device Mobilized peripheral blood stem cells from allogeneic donors depleted of TCRαβ+ T cells/CD19+ B cells Indication Renal failure due to one of four genetic and/or immunological diseases Therapeutic Mechanism 1. By using haploidentical parents, we will expand the number of potential living donors, and 2. with pre-HSCT immune ablation we can […]
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 […]
Phase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapy
Therapeutic Candidate or Device EBT-101 is a novel genome editing therapeutic targeting integrated HIV-1 genome to achieve sustained virologic reduction to enable functional cure. Indication EBT-101 is intended to deplete the reservoir of integrated HIV-1 proviral DNA in immune reconstituted virally suppressed HIV-1 positive individuals. Therapeutic Mechanism EBT-101 is an in vivo gene therapy product […]
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 […]
A Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade Gliomas
Therapeutic Candidate or Device Neural stem cells that are genetically engineered to express a cancer-killing virus that specifically targets brain tumor cells Indication Recurrent brain tumors in adults: high grade gliomas (HGG), such as glioblastoma (GBM) Therapeutic Mechanism The neural stem cells will act as carriers to deliver a cancer-killing virus to brain tumors (at […]
Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome
Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy X-linked (IPEX) syndrome Therapeutic Mechanism Administration of autologous CD4LVFOXP3 that constitutively and stably express wild-type FOXP3 gene will replace the lack of function regulatory T […]
A Double-Blind Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Progenitor Cells for the Treatment of Dysphagia
Therapeutic Candidate or Device Autologous Muscle Derived Progenitor Cells (AMDC) isolated from skeletal muscle biopsy Indication Subjects with dysphagia (swallowing difficulties) that develops following treatment for head and neck cancer Therapeutic Mechanism Autologous Muscle Derived Progenitor Cells differentiate to form new muscle fibers, engraft into existing myofibers, and have been shown to increase muscle diameter […]
PHASE 1, OPEN LABEL, DOSE-ESCALATION STUDY OF CRX100 IN PATIENTS WITH ADVANCED SOLID TUMORS
Therapeutic Candidate or Device Immune cells loaded with a cancer-killing virus that targets cancer tissue, not healthy tissue. Indication Advanced, refractory solid tumors: CRC, HCC, Osteosarcoma, NNN Breast, Ovarian, Gastric Therapeutic Mechanism The proposed therapy pairs activated immune cells with an adapted oncolytic virus that selectively infects and kills malignant cells. The result of this […]
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 […]