This segment focuses on the development and commercialization of gene therapy for Hemophilia A, a genetic bleeding disorder. Sangamo's lead product, SB-525, is in Phase III clinical trials (AFFINE) and aims to provide a functional cure by delivering a functional copy of the F8 gene. The technology utilizes adeno-associated virus (AAV) vectors to deliver the therapeutic gene. This segment addresses a significant unmet medical need, offering the potential for long-term benefits and reduced reliance on factor replacement therapy. Partnerships with companies like Pfizer are crucial for clinical development and commercialization. Future opportunities include expanding the application of gene therapy to other bleeding disorders and improving the efficacy and safety of AAV-based therapies. Regulatory aspects involve navigating FDA approval processes and demonstrating long-term safety and efficacy in clinical trials.

This segment is dedicated to developing gene therapy for Fabry disease, a rare genetic disorder characterized by the buildup of a specific type of fat in the body's cells. Sangamo's ST-920, a gene therapy, is currently in Phase I/II clinical trials (STAAR) and aims to provide a functional copy of the GLA gene. The technology utilizes AAV vectors to deliver the therapeutic gene. This segment addresses a significant unmet medical need, offering the potential for long-term benefits and reduced reliance on enzyme replacement therapy. The focus is on improving the quality of life for patients by addressing the underlying genetic cause of the disease. Future opportunities include expanding the application of gene therapy to other lysosomal storage disorders and improving the efficacy and safety of AAV-based therapies. Regulatory aspects involve navigating FDA approval processes and demonstrating long-term safety and efficacy in clinical trials.

This segment focuses on developing cell therapy for sickle cell disease (SCD), a genetic blood disorder. Sangamo is developing SAR445136, a cell therapy, which is in Phase I/II PRECIZN-1 clinical trials. The therapy involves modifying a patient's own blood stem cells to produce fetal hemoglobin, which can reduce or eliminate the painful symptoms of SCD. The technology utilizes zinc finger nucleases (ZFNs) for genome editing. This segment addresses a significant unmet medical need, offering the potential for a functional cure for SCD patients. The focus is on improving the quality of life for patients by reducing the frequency and severity of vaso-occlusive crises and other complications. Future opportunities include expanding the application of cell therapy to other genetic blood disorders and improving the efficiency and safety of ZFN-mediated genome editing. Regulatory aspects involve navigating FDA approval processes and demonstrating long-term safety and efficacy in clinical trials.

This segment encompasses research and development efforts focused on neurological disorders, including tauopathies (e.g., Alzheimer's disease) and synucleinopathies (e.g., Parkinson's disease). Sangamo is developing ST-501 for tauopathies and ST-502 for synucleinopathies. The company utilizes its ZFN technology to target and modify genes associated with these diseases. The goal is to develop therapies that can slow or halt disease progression. This segment addresses significant unmet medical needs in the field of neurology. Future opportunities include expanding the pipeline to include other neurological disorders and exploring novel gene editing approaches. Regulatory aspects involve navigating FDA approval processes and demonstrating safety and efficacy in clinical trials. Partnerships with companies like Eli Lilly are crucial for advancing these programs.