Business
Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results
Data from novel proprietary neurotropic adeno-associated virus (AAV) delivery capsid, STAC-BBB, demonstrated industry-leading blood-brain barrier (BBB)
About this update from Sangamo Therapeutics, Inc.
[{"type":"text","content":"\n\nData from novel proprietary neurotropic adeno-associated virus (AAV) delivery capsid, STAC-BBB, demonstrated industry-leading blood-brain barrier (BBB) penetration in non-human primates (NHPs) following intravenous administration, with capsid-enabled delivery of zinc finger payloads targeting prion disease and tauopathies resulting in potent and widespread repression of target genes.\n\n\n\nChronic neuropathic pain and prion disease preclinical programs advance, with investigational new drug (IND) and clinical trial authorization (CTA) submissions expected in the fourth quarters of 2024 and 2025, respectively.\n\n\n\nAnnounced U.S. Food and Drug Administration (FDA) alignment on an abbreviated pathway to potential approval, and grant by European Medicines Agency (EMA) of priority medicines (PRIME) eligibility for isaralgagene civaparvovec in Fabry disease.\n\n\n\nPfizer anticipates Biologics License Application (BLA) and marketing authorization application (MAA) submissions for Hemophilia A collaboration by early 2025 if the pivotal readout is supportive.\n\n\n\n RICHMOND, Calif.--(BUSINESS WIRE)--\nSangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.\n\n\n“In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo. “With the meaningful preclinical data announced today, we believe our ability to combine potent zinc finger epigenetic regulation payloads with exciting new industry-leading capsid delivery technology could unlock significant potential for the treatment of devastating neurological diseases, indications for which delivery of treatments to the central nervous system has historically proved challenging. In the near-term, we are also seeking to create value by partnering our Fabry disease program, for which we aligned with the FDA on a potentially abbreviated and more cost-effective timeline and received EMA PRIME eligibility. We look forward to advancing our pipeline into the clinic to develop therapies designed to target neurological diseases with high unmet medical needs.”\n\n\nRecent Bu...