Clinical Trial, Phase 1/2 – Page 6

RPESC-RPE-4W Therapy for dry Age-related Macular Degeneration

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Post published:September 28, 2026
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Therapeutic Candidate or Device Retina pigment epithelial stem cell (RPESC)-derived RPE progeny at 4 weeks of differentiation (RPESC-RPE-4W) Indication Dry age-related macular degeneration (dry AMD). Therapeutic Mechanism Dry AMD involves…

The CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele

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Post published:September 28, 2026
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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…

Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A

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Post published:September 28, 2026
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Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation…

The UCLA Delayed Immunological Tolerance after Kidney Transplantation Program

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Post published:September 28, 2026
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Therapeutic Candidate or Device CD34+ hematopoietic stem/progenitor cells (HSPC) and CD3+ cells Indication Phase I/II trial evaluating the safety/efficacy of infusion of donor CD34+ HSPCs and CD3+ T cells into…

Autologous ARTEMIS® T Cells to Treat Refractory/Relapsed Pediatric Liver Cancer

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Post published:September 28, 2026
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Therapeutic Candidate or Device ET140203 T cells: engineered T-cell therapy whereby autologous T cells are modified to specifically target and kill AFP+/ HLA-A2+ cancer cells. Indication Pediatric subjects aged ≥…

Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients

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Post published:September 28, 2026
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Therapeutic Candidate or Device αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191 Indication Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias…

Sequential same donor αβdepleted-HSCT from an HLA-partially matched donor allowing immunosuppression free kidney transplant

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Post published:September 28, 2026
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Therapeutic Candidate or Device Mobilized peripheral blood stem cells from allogeneic donors depleted of TCRαβ+ T cells/CD19+ B cells Indication Renal failure due to one of four genetic and/or immunological…

A Double-Blind Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Progenitor Cells for the Treatment of Dysphagia

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Post published:September 28, 2026
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Therapeutic Candidate or Device Autologous Muscle Derived Progenitor Cells (AMDC) isolated from skeletal muscle biopsy Indication Subjects with dysphagia (swallowing difficulties) that develops following treatment for head and neck cancer…

Anti-HIV duoCAR-T cell therapy for HIV infection

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Post published:September 28, 2026
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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…

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

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Post published:September 28, 2026
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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…