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FTX-PD01
- Development stage
- Preclinical
- Lead developer
- Frida Therapeutics
- Modality
- Patient-derived iPSCs → iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies, Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, CAR-NK Cells → Other Engineered Cells → Adoptive Cell Transfer → Cell Therapies, CAR-Macrophages → Other Engineered Cells → Adoptive Cell Transfer → Cell Therapies, Autologous CAR-T → CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Mesenchymal Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, TCR-Engineered T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Tumor-Infiltrating Lymphocytes (TILs) → Native Immune Cells → Adoptive Cell Transfer → Cell Therapies
- Administration
- Intravenous
01
Overview
FTX-PD01 is a genetically modified autologous bone marrow cell therapy under development by Frida Therapeutics for the treatment of Pompe disease (glycogen storage disease type II). The therapy involves collecting and modifying a patient's own bone marrow cells, with the aim of replacing or repairing defective cells associated with Pompe disease. Its mechanism of action is classified as "cell replacement," targeting the underlying cellular deficiency in this metabolic disorder. As of August 2024, FTX-PD01 remains in preclinical development, with no reported clinical trials initiated yet[1][2][9].
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