Human Stem Cell Use: Adult or Tissue Stem Cell

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 […]

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 […]

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 […]

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 […]

A Phase I Study of Chimeric Antigen Receptor Engineered Stem/Memory T Cells for the Treatment of HER2-Positive Brain Metastases

Therapeutic Candidate or Device Autologous naïve-stem/memory T cells engineered with a chimeric antigen receptor targeting the HER2 antigen (HER2BBζ-Tn/mem) Indication HER2-positive brain and/or leptomeningeal metastases, primarily from breast cancer Therapeutic Mechanism The proposed therapy aims to provide a safe and effective treatment option for patients with HER2-positive cancers that have metastasized to the central nervous […]


Therapeutic Candidate or Device The therapeutic candidate is an ex-vivo autologous gene therapy approach for Leukocyte Adhesion Deficiency-I (LAD-I). Indication The target clinical indication is Leukocyte Adhesion Deficiency-I (LAD-I), a pediatric hematological rare disease. Therapeutic Mechanism The therapeutic is based in an ex-vivo, lentiviral-based, patient-specific approach by genetically engineering the patient's own CD34 positively selected […]

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