Therapeutic/Technology: Therapeutic Approach


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 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect

Therapeutic Candidate or Device KPI-012 is a human bone marrow mesenchymal stem cell secretome formulated as a topical ophthalmic therapeutic Indication Treatment of persistent corneal epithelial defect, a cornea defect refractory to conventional treatments that can lead to blindness Therapeutic Mechanism KPI-012 is intended to restore normal corneal wound healing, providing a curative solution to […]

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 […]

Autologous ARTEMIS® T Cells to Treat Refractory/Relapsed Pediatric Liver Cancer

Therapeutic Candidate or Device ET140203 T cells: engineered T-cell therapy whereby autologous T cells are modified to specifically target and kill AFP+/ HLA-A2+ cancer cells. Indication Pediatric subjects aged ≥ 1 year to ≤ 21 years who are AFP-positive/HLA-A2-positive and have relapsed/refractory (r/r) HB, HCN-NOS, or HCC. Therapeutic Mechanism ET140203 T-cell therapy is designed to […]

Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

Therapeutic Candidate or Device αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191 Indication Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias Therapeutic Mechanism Fanconi anemia (FA) patients undergoing HCT have heightened sensitivity to current conditioning regimens and short and long-term sensitivity to graft versus host disease […]

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 […]

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 […]

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 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 […]