RICHMOND -
Presentations will focus on the progression of Sangamo's neurology-focused pre-clinical pipeline, including data supporting innovations in zinc finger epigenetic regulation, advances in AAV capsid engineering, and discovery of next-generation integrase technology.
'We are thrilled to be showcasing at ASGCT the strong depth of our neurology pipeline, including various applications for our zinc finger epigenetic regulation platform, exciting advances in our capsid delivery technology, and new, potentially transformative Modular Integrases (MInts), that together reflect our continued commitment to developing potential neurology genomic medicines for patients in need,' said
'These data showcase the deep expertise of our scientific team in engineering DNA-protein interactions and the broad potential reach of our innovative pre-clinical approaches, including exciting new discoveries for site-directed integration of large DNA sequences via engineered MInts,' said
Data presentations at the ASGCT Annual Meeting include three oral presentations, two of which detail Sangamo's potent delivery capabilities developed through its AAV capsid engineering platform SIFTER, and one that will discuss a new breakthrough in programming Bxb1, a large serine recombinase, for the development of MInts to potentially enable large-scale genome engineering. These data will showcase a novel AAV capsid, STAC-BBB, that exhibited robust and widespread central nervous system transduction in cynomolgus macaques after intravenous delivery, as well as another novel AAV capsid, STAC-150, engineered to accelerate the discovery of potent and highly specific epigenetic regulators. Sangamo will also showcase data highlighting the use of MInts, derived from compact Bxb1 variants, to integrate large sequences of DNA into the genome with minimal double-stranded DNA breaks and without the need for assistance from ancillary genome editing or DNA-repair modulating cargo.
Additional poster presentations at the ASGCT Annual Meeting will showcase the use of zinc finger activators and repressors for the potential treatment of neurological diseases such as prion disease, tauopathies, Charcot-Marie-Tooth disease type 1A (CMT1A), Dravet Syndrome, SOD1-mediated amyotrophic lateral sclerosis (ALS), Phelan-McDermid syndrome, Parkinson's disease, Angelman syndrome, and other CNS disorders. Sangamo will also present additional data from its AAV capsid engineering platform SIFTER and its efforts for integrase evolution and utilization.
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Sangamo Forward Looking Statements
This press release contains forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, statements relating to Sangamo's technologies, the presentation of data from various therapeutic and research programs and the potential of these programs to demonstrate therapeutic benefit and transform the lives of patients. These statements are not guarantees of future performance and are subject to certain risks and uncertainties that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the research and development process, including the results of clinical trials; the regulatory approval process for product candidates and the potential for technological developments that obviate technologies used by Sangamo. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business. These risks and uncertainties are described more fully in our
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