Phase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain Tumors

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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…

Continue ReadingPhase I Study of IL13Rα2-Targeting CAR T Cells After Lymphodepletion for Children with Refractory or Recurrent Malignant Brain Tumors

A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)

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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…

Continue ReadingA Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)

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

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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…

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

Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa

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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…

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LADICell

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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…

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Phase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.

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Therapeutic Candidate or Device Autologous Human CD34+ HSC from Mobilized PBSC of Patients with Cystinosis Modified by Ex Vivo Transduction using the pCCL-CTNS Lentiviral Vector Indication Cystinosis - An autosomal…

Continue ReadingPhase 1/2 study for autologous human CD34+ hematopoietic stem cells ex vivo transduced with pCCL-CTNS lentiviral vector for treatment of Cystinosis.