A Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy

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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…

Continue ReadingA Phase 2b, Randomized, Assessor-Masked Clinical Trial to Assess the Safety and Efficacy of the CPCB-RPE1 Implant in Subjects with Geographic Atrophy

Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV

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Therapeutic Candidate or Device Cytomegalovirus (CMV)-specific T cells that express a chimeric antigen receptor (CAR) which targets and eliminates HIV-infected cells Indication Management of human immunodeficiency virus (HIV)/acquired immunodeficiency syndrome…

Continue ReadingEvaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV

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

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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…

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

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

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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…

Continue ReadingA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

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

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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…

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

AB-205-301 Phase 3 Trial and Related Activities to Support Clinical Development of AB-205

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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…

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A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

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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…

Continue ReadingA Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

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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…

Continue ReadingPhase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy