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Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs
Novel AAV capsid engineered by Sangamo showed robust penetration of blood-brain barrier (BBB) and widespread transgene expression throughout brain in
About this update from Sangamo Therapeutics, Inc.
[{"type":"text","content":"\n\nNovel AAV capsid engineered by Sangamo showed robust penetration of blood-brain barrier (BBB) and widespread transgene expression throughout brain in non-human primates (NHPs) following intravenous administration.\n\n\n\nDemonstrated industry-leading brain tropism and enrichment in NHPs, resulting in 700-fold higher transgene expression than benchmark capsid AAV9.\n\n\n\nCapsid-enabled delivery of zinc finger payloads targeting prion disease and tauopathies resulted in robust and widespread repression of target genes.\n\n\n\nSTAC-BBB capsid could potentially unlock multiple neurology epigenetic regulation programs paused by Sangamo pending identification of suitable capsid and could be advanced either internally or with a collaborator.\n\n\n\nSangamo expects to file up to three neurology Investigational New Drug (IND) submissions and/or Clinical Trial Applications (CTA) by end of 2025.\n\n\n\nSangamo to discuss results in conference call scheduled for Wednesday, March 13 at 4:30pm Eastern Time.\n\n\n\n RICHMOND, Calif.--(BUSINESS WIRE)--\nSangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced preclinical data from its proprietary adeno-associated virus (AAV) capsid variant, STAC-BBB, that demonstrated robust penetration of the blood-brain barrier (BBB) and strong transgene expression throughout the central nervous system (CNS) of NHPs when administered intravenously at clinically-relevant doses, outperforming results obtained by Sangamo for other known neurotropic capsid variants evaluated in the study. Importantly, potent repression of target genes was observed in brain cells expressing the zinc finger cargo, indicating that STAC-BBB could enable development of genomic medicines to potentially treat a wide range of neurological diseases.\n\n\n“The advancement of neurological medicines has long been limited by the inability to achieve widespread CNS delivery, an essential attribute for an effective treatment for devastating neurological disorders. We are extremely encouraged that STAC-BBB, a potentially game-changing capsid variant engineered using our SIFTER capsid platform, demonstrated results that outperformed other known neurotropic capsid variants, achieving widespread brain delivery and transgene expression, desired de-targeting of liver and other peripheral tissues and a favorable safe...