Human Neural Progenitors Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis

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Therapeutic Candidate or Device CNS10-NPC-GDNF - a neural progenitor cell secreting GDNF Indication ALS Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As…

Continue ReadingHuman Neural Progenitors Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis

Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients

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Therapeutic Candidate or Device HSC gene modified by a lentiviral vector which encodes a triple combination of HIV-resistance genes and a pre-selective marker. Indication HIV in AIDS-lymphoma patients. Therapeutic Mechanism…

Continue ReadingStem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells in AIDS lymphoma patients

A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

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Therapeutic Candidate or Device The therapeutic product candidate is autologous CD34+ hematopoietic stem/progenitor cells (HSPC) transduced with the G1XCGD lentiviral vector. Indication The target indication is for the transplantation of…

Continue ReadingA Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy

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Therapeutic Candidate or Device RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21. Indication Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening…

Continue ReadingNovel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy

Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and AYA with Recurrent Solid Tumors

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Therapeutic Candidate or Device Engineered CAR T-cells are enhanced to allow their survival in solid tumors Indication Children, adolescents and young adults with variety of solid tumors but focus on…

Continue ReadingChimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and AYA with Recurrent Solid Tumors

A 1XX-enhanced and fully non-viral BCMA CAR T cell therapy for Relapsed and Refractory Multiple Myeloma (UCCT-BCMA-1)

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Therapeutic Candidate or Device Cryopreserved autologous TRAC locus 1XX BCMA-CAR T cells Indication Relapsed and Refractory Multiple Myeloma Therapeutic Mechanism Engineered T cells target and kill BCMA+ Myeloma cells Unmet…

Continue ReadingA 1XX-enhanced and fully non-viral BCMA CAR T cell therapy for Relapsed and Refractory Multiple Myeloma (UCCT-BCMA-1)