Transfer of gene therapy SB-525 for hemophilia A to collaboration partner Pfizer (NYSE:PFE) substantially completed. A Phase 3 trial should launch in 2020. Enrollment in a lead-in study is underway.
Phase 1/2 study evaluating gene therapy ST-920 in Fabry patients is in process.
IND for gene therapy ST-101 in PKU to be filed in 2021.
Early data on cell therapy line-up to be presented, including ST-400 beta thalassemia ex vivo gene-edited cell therapy being developed with Sanofi (NASDAQ:SNY).
Updates on CAR-TREG clinical and preclinical programs will be presented as well. CAR-TREGS are regulatory T cells equipped with a chimeric antigen receptor (CAR). TX200 is in development for the prevention of immune-mediated organ rejection in kidney transplant patients.
Other areas: gene editing with zinc finger nuclease in mucopolysaccharidosis type II (MPS II), genome editing candidates in neurodegenerative diseases, including Parkinson’s (ST-502) and Alzheimer’s (ST-501).
Buildout of GMP manufacturing facility in Brisbane, CA almost completed and should be online next year.
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