Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)
Phase I Study of Chimeric Antigen Receptor Engineered T Cells targeting CD33 for the Treatment of Relapsed/Refractory Acute Myeloid Leukemia
Therapeutic Candidate or Device Immune T cells from a patient’s transplant donor engineered to express chimeric antigen receptors for targeted leukemia killing Indication Relapsed or refractory acute myeloid leukemia Therapeutic Mechanism Upon adoptive transfer, patient specific immune T cells that express chimeric antigen receptors will specifically recognize and directly kill leukemia cells expressing CD33. Unmet […]
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation in the SCN2A gene with both GOF and LOF effects when expressed in cells. nL-SCN2A-002 is designed to specifically bind the pathogenic allele and lower […]
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 […]
Stem-Derived IL13Ra2 Chimeric Antigen Receptor T cells for Patients with Melanoma and Advanced Solid Tumors
Therapeutic Candidate or Device Adult Stem-Like T cells engineered with chimeric antigen receptor (CAR) to target cancers expressing IL13Ra2, including melanoma. Indication Advanced cancers that express IL13Ra2, including melanoma. Therapeutic Mechanism When administered to a patient, the engineered T cells will circulate through the blood and tissues to nd, recognize and kill tumor cells that […]
Evaluation of Safety and Feasibility of Cytomegalovirus-Specific, Anti-HIV Chimeric Antigen Receptor (CMV/HIV-CAR) T Cells in People with HIV
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 (AIDS) Therapeutic Mechanism Persons with HIV (PWH) lose immunity because of decreased T lymphocyte function. We propose to treat them with CAR T cells which […]
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 […]
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 […]
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 […]