Sangamo (SGMO) CEO Sandy Macrae opened its presentation stating that over the next year it
will become the leading genomic medicines company active in gene editing, gene and cell
therapy, and gene regulation. The firm chooses what to work on based on high unmet medical
need and matches its technology to certain areas of focus. The pipeline expands across the areas
of metabolic diseases, immunology, CNS, hematology, and cancer. Clinical readouts are expected
in 2019 and 2020 following enrollment progress of the last twelve months. During 2018 Sangamo
partnered with Pfizer and Kite and also acquired TxCell.
Sangamo currently has five programs in the clinic – SB-318 for MPS I, SB-913 for MPS II, SB-525
for hemophilia A, SB-FIX for hemophilia B, and ST-400 for beta-thalassemia. It anticipates moving
another four programs into clinical trials this year, the first being ST-400 for sickle cell disease by
partner Bioverativ. The company also made known that its Fabry candidate ST-920 and Tx200 for
solid organ transplant should move into human studies by year-end. Data from both MPS
programs will be revealed in February at the 2019 WORLD Symposium meeting. The SB-318
presentation is expected to describe preliminary safety and biochemical measurements at up to
four weeks from the first three adult patients enrolled in the EMPOWERS study, while the SB-913
presentation will include interim data on safety and biochemical measurements at up to 24
weeks from six adults enrolled in the three dose cohorts of the CHAMPIONS study.
Sangamo spoke about its gene editing platform that allows for targeted gene knock-out or
therapeutic gene insertion using zinc finger DNA-binding proteins. The ZFN technology can
recognize DNA with great specificity, precision, and efficiency. Through the TxCell acquisition
Sangamo gained CAR-Treg technology and plans to develop products with ZFN multiplex editing.
CAR-Tregs hold significant potential in the autoimmune disease space. Since Tregs are localized to
tissue you don’t need to know antigen causing the disease. Sangamo iterated that it’s excited
about the technology as no one has ever created a CAR-Treg.
will become the leading genomic medicines company active in gene editing, gene and cell
therapy, and gene regulation. The firm chooses what to work on based on high unmet medical
need and matches its technology to certain areas of focus. The pipeline expands across the areas
of metabolic diseases, immunology, CNS, hematology, and cancer. Clinical readouts are expected
in 2019 and 2020 following enrollment progress of the last twelve months. During 2018 Sangamo
partnered with Pfizer and Kite and also acquired TxCell.
Sangamo currently has five programs in the clinic – SB-318 for MPS I, SB-913 for MPS II, SB-525
for hemophilia A, SB-FIX for hemophilia B, and ST-400 for beta-thalassemia. It anticipates moving
another four programs into clinical trials this year, the first being ST-400 for sickle cell disease by
partner Bioverativ. The company also made known that its Fabry candidate ST-920 and Tx200 for
solid organ transplant should move into human studies by year-end. Data from both MPS
programs will be revealed in February at the 2019 WORLD Symposium meeting. The SB-318
presentation is expected to describe preliminary safety and biochemical measurements at up to
four weeks from the first three adult patients enrolled in the EMPOWERS study, while the SB-913
presentation will include interim data on safety and biochemical measurements at up to 24
weeks from six adults enrolled in the three dose cohorts of the CHAMPIONS study.
Sangamo spoke about its gene editing platform that allows for targeted gene knock-out or
therapeutic gene insertion using zinc finger DNA-binding proteins. The ZFN technology can
recognize DNA with great specificity, precision, and efficiency. Through the TxCell acquisition
Sangamo gained CAR-Treg technology and plans to develop products with ZFN multiplex editing.
CAR-Tregs hold significant potential in the autoimmune disease space. Since Tregs are localized to
tissue you don’t need to know antigen causing the disease. Sangamo iterated that it’s excited
about the technology as no one has ever created a CAR-Treg.
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