Program Type: Clinical
CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS
Therapeutic Candidate or Device CNS10-NPC-GDNF – a neural progenitor cell secreting GDNF Indication Amyotrophic Lateral Sclerosis (ALS) Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this neuroprotective factor to dying motor neurons in the motor […]
Safety and Tolerability Study of Neural Stem Cells (NR1) in Subjects with Chronic Ischemic Subcortical Stroke
Therapeutic Candidate or Device A human embryonic derived, non-genetically modified neural stem cell (NR1), originally derived from the Wi-Cell H-9 line Indication Patients with chronic motor deficits, from 6 to 60 months after stroke. NR1 cells will be injected into the brain near the site of the stroke. Therapeutic Mechanism The proposed therapeutic mechanism of […]
Phase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain Tumors
Therapeutic Candidate or Device Autologous chimeric antigen receptor T cells derived from naive/memory T cells and engineered to target IL13Rα2 on pediatric malignant brain tumors. Indication Recurrent/refractory malignant pediatric brain tumors that express the tumor-associated antigen IL13Rα2. Therapeutic Mechanism Naive and memory T cells are harvested from patients and reprogrammed to express chimeric antigen receptors […]
Phase 1 Clinical Development of IO-202, A First-in-Class Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMML
Therapeutic Candidate or Device IO-202, a first-in-class antibody targeting leukocyte immunoglobulin-like receptor B4 (LILRB4), an immune inhibitory receptor Indication Acute myeloid leukemia (AML) with monocytic differentiation and chronic myelomonocytic leukemia (CMML) Therapeutic Mechanism IO-202 is the first T-cell activator for AML. Preclinical studies showed that IO-202 can convert a “don’t kill me” to “kill me” […]
The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair
Therapeutic Candidate or Device Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM) Indication Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenatally Therapeutic Mechanism Placenta-derived mesenchymal stem cells (PMSCs) act by a paracrine mechanism, secreting a variety of growth factors, cytokines, and extracellular vesicles. This secretory profile is unique to PMSCs […]
A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)
Therapeutic Candidate or Device The therapeutic candidate is an ex-vivo autologous gene therapy approach for Infantile Malignant Osteopetrosis (IMO). Indication The target clinical indication is Infantile Malignant Osteopetrosis (IMO), a pediatric, autosomal recessive rare disease. Therapeutic Mechanism The therapeutic mechanism is based on an ex-vivo, lentiviral-based, patient-specific approach by genetically engineering the patient's own CD34+ […]
Anti-HIV duoCAR-T cell therapy for HIV infection
Therapeutic Candidate or Device HIV-specific CAR-T cells Indication Management of HIV infection Therapeutic Mechanism We will modify T cells such that they are able to directly control HIV in the absence of therapy. Should this work, these cells will result in long-term control of HIV in absence of any ongoing treatment, a version of a […]
A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease
Therapeutic Candidate or Device AAV2-GDNF is a gene therapy product encoding Glial cell line-Derived Neurotrophic Factor (GDNF) Indication Parkinson's disease Therapeutic Mechanism AAV2-GDNF will be delivered into the putamen. GDNF is a growth factor expected to act by stimulating regeneration of the terminals of dopamine producing neurons that are progressively lost in PD. This is […]
Safety and Feasibility of Cultivated Autologous Limbal Stem Cells for Limbal Stem Cell Deficiency
Therapeutic Candidate or Device Autologous cultivated limbal stem cells (cLSC) Indication Limbal stem cell deficiency Therapeutic Mechanism Restoration of a normal corneal surface using cLSC might be achieved by replenishing the LSC population and/or providing trophic factors to stimulate residual LSCs. Unmet Medical Need Therapy using cultivated LSC, which achieves the best clinical outcomes is […]
Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa
Therapeutic Candidate or Device CNS10-NPC – a human neural progenitor cell line Indication Retinitis Pigmentosa Therapeutic Mechanism 1. Phagocytosis of photoreceptor outer segment debris. 2. The release of pro-survival factors that have localized diffusion to inhibit retinal photoreceptor cell death. 3. Immunomodulation resulting in markedly fewer host inflammatory cells at the site of CNS10-NPC engraftment […]