Gene Therapy (All) – Page 18

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

Post author:
Post published:November 15, 2025
Post category:

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

Post author:
Post published:November 15, 2025
Post category:

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

Post author:
Post published:November 15, 2025
Post category:

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)

Post author:
Post published:November 15, 2025
Post category:

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

Post author:
Post published:November 15, 2025
Post category:

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

Post author:
Post published:November 15, 2025
Post category:

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…

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

Post author:
Post published:November 15, 2025
Post category:

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…

LADICell

Post author:
Post published:November 15, 2025
Post category:

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…

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

Post author:
Post published:November 15, 2025
Post category:

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…

Genetic Modification of Stem Cells and T cells to Activate the Immune System to Target Solid Tumors

Post author:
Post published:November 15, 2025
Post category:

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…

Subscribe to Receive CIRM News