Therapeutic/Technology: Therapeutic Approach
Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor T Cells for Diffuse Intrinsic Pontine Gliomas and Spinal Diffuse Midline Glioma
Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting GD2 (GD2-CART) Indication Brain tumors in children and young adults: Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG) Therapeutic Mechanism Progenitor GD2-CART cells will recognize DIPG/DMG cancer cells expressing GD2, become activated, divide, and kill the […]
Phase 1/1b study of T-allo10 infusion after HLA-partially matched abdepleted-HSCT in children and young adults with hematologic malignancies.
Therapeutic Candidate or Device An immunotherapy cell product, T-allo10, that is enriched for specialized immune cells called type I regulatory T (Tr1) cells Indication Children and young adults with relapse/refractory acute leukemia receiving a specialized stem cell transplant, αβdepleted-HSCT Therapeutic Mechanism αβdepleted-HSCT has increased the number of patients who can safely receive transplants, however this […]
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 […]
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 […]
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 […]