Stage of Program: Clinical Trial, Phase 1
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 […]
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 […]
Induction of Tolerance by Combinatorial Therapy w/ Donor Stem Cells and Expanded Recipient Treg cells in HLA-mismatched Kidney Transplant Recipients
Therapeutic Candidate or Device Combined hematopoietic stem cell graft and recipient T regulatory cells Indication Kidney disease requiring kidney transplantation Therapeutic Mechanism The study will determine whether patients treated with TLI and rATG, and given a haploidentical living donor hematopoietic progenitor cell transplant (HSCT) , along with in vitro expanded recipient Treg cells (what we […]
Genetic Modification of Stem Cells and T cells to Activate the Immune System to Target Solid Tumors
Therapeutic Candidate or Device Autologous Peripheral Blood Stem Cells expressing the NY-ESO-1 TCR and a suicide/reporter gene combined with T cells expressing the same TCR Indication Locally advanced (unresectable stage IIIc) or metastatic malignancies (stage IV) that are HLA A2.1 +, NY-ESO-1 +, solid tumors, including sarcomas Therapeutic Mechanism The administration of TCR transduced mature […]
AB-205-001 Phase 1b Trial and Related Activities to Support Clinical Development of AB-205
Therapeutic Candidate or Device AB-205 consists of genetically engineered CD31+ cells derived from Human Umbilical Vein tissue. Indication To ameliorate or accelerate recovery from toxicities related to high-dose chemo followed by HDT-ASCT for the treatment of lymphoma and other cancers. Therapeutic Mechanism E-CEL UVEC cells (the active ingredient in AB-205) work both via the secretion […]
Treatment of sickle cell disease by induction of mixed chimerism and immune tolerance using CD4+ T-depleted haploidentical blood stem cell transplant
Therapeutic Candidate or Device Haploidentical (half-match) T cell depleted blood stem cell transplant with a low-toxic conditioning regimen Indication Adult patients with severe sickle cell disease who are excluded from the potentially curative current standard stem cell transplant. Therapeutic Mechanism The proposed therapy is intended to achieve mixed chimerism and immune tolerance. Mixed chimerism is […]
Phase 1 Study of CD19/CD22 Chimeric Antigen Receptor (CAR) T Cells in Adults with Recurrent or Refractory B Cell Malignancies
Therapeutic Candidate or Device T cells genetically engineered to express as bispecific Chimeric Antigen Receptor (CAR) targeting CD19 and/or CD22 Indication Patients with relapsed and refractory B cell malignancies Therapeutic Mechanism T cells expressing the bispecific CAR will recognize cancer cells expressing one of both of the target antigens. Upon recognition, the T cells will […]
Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells
Therapeutic Candidate or Device Bone marrow stem cells that have been treated by inserting a normal Artemis gene into the DNA using a modified virus called a lentivirus. Indication Children with severe combined immunodeficiency or "bubble baby disease" due to a defective gene that makes a protein called Artemis Therapeutic Mechanism Stem cells from the […]