Gene Therapy (All) – Page 19

A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA

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Post published:December 17, 2025
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Therapeutic Candidate or Device GNSC-001 is an adeno-associated vector (AAV) expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks IL-1 signaling. Indication Osteoarthritis of the knee Therapeutic…

Phase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapy

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Post published:December 17, 2025
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Therapeutic Candidate or Device EBT-101 is a novel genome editing therapeutic targeting integrated HIV-1 genome to achieve sustained virologic reduction to enable functional cure. Indication EBT-101 is intended to deplete…

Phase 1 Study of Autologous CD4LVFOXP3 in Participants with IPEX Syndrome

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Post published:December 17, 2025
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Therapeutic Candidate or Device CD4+ T cells that have undergone lentiviral -mediated gene transfer of Forkhead Box P3 (FOXP3) and acquired regulatory T cell function. Indication Immune dysregulation Polyendocrinopathy Enteropathy…

A Phase I Study of Multiple doses of NSC-Based Oncolytic Virotherapy Administered Intracerebrally to Patients with Recurrent High-Grade Gliomas

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Post published:December 17, 2025
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Therapeutic Candidate or Device Neural stem cells that are genetically engineered to express a cancer-killing virus that specifically targets brain tumor cells Indication Recurrent brain tumors in adults: high grade…

Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor T Cells for Diffuse Intrinsic Pontine Gliomas and Spinal Diffuse Midline Glioma

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Post published:December 17, 2025
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Therapeutic Candidate or Device Autologous T cells genetically engineered to express a Chimeric Antigen Receptor targeting GD2 (GD2-CART) Indication Brain tumors in children and young adults: Diffuse Intrinsic Pontine Gliomas…

CNS10-NPC-GDNF delivered into the motor cortex for the treatment of ALS

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Post published:December 17, 2025
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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…

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

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Post published:December 17, 2025
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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…

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

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Post published:December 17, 2025
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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…

Anti-HIV duoCAR-T cell therapy for HIV infection

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Post published:December 17, 2025
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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…

A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease

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Post published:December 17, 2025
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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…

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