Gilead’s Kite has struck a $3 billion deal to access Sangamo Therapeutics’ zinc finger nuclease (ZFN) technology. The case for Sangamo Therapeutics Year after year, Sangamo publishes a study that proves its zinc-finger nuclease (ZFN) technology is superior to all other gene editing methods in … Further data on patients who have withdrawn from ERT, due later this year, will also be worth watching, the company claims. Three patients were taken off ERT, two in the medium-dose group and one in the high-dose. The ZFNs make a double stranded break in the DNA in a precise location in the albumin gene, where the transgene is permanently integrated using the cell’s natural repair mechanism. Sangamo Therapeutics: The Promise of Zinc Fingers Real Estate Our zinc finger nuclease (ZFN) technology is based on a naturally occurring class of proteins called zinc finger DNA-binding proteins (ZFPs), which recognize and bind to specific sequences of DNA. Kite will use the ZFN gene editing … The theory was that this would result in an increase in IDS production and a reduction in GAGs, with the ultimate hope that patients could be safely taken off ERT. But, even if more persuasive biomarker signs emerge, the study might not be sufficient for a regulatory filing. Ex vivo gene-edited cell therapy is the most straightforward application of ZFN technology. The goal of genome editing is to provide a permanent therapeutic solution or cure for genetic diseases. It will be crucial that, when the biopsy results come back, they show the gene to have been successfully integrated. The presentation concerned six subjects, two from each of the three dosing groups. Snippet roundup: Wins for AcelRx, Foamix and SGLT2 developers. Sangamo Therapeutics is at the forefront of a new era of genomic medicine that aims to transform the lives of people living with serious diseases. We engineer ZFNs for precision, efficiency, and specificity, which we believe are the critical parameters for a therapeutic genome editing technology. Sangamo’s genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), is currently delivered with AAVs and functions at the DNA level to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect. The contagion was not contained to just Sangamo and its older zinc finger nuclease genome editing technology; names like Crispr Therapeutics, Intellia, Editas Medicine and even Sarepta were also dragged down by this setback. Sangamo's chief executive Sandy Macrae yesterday talked up second-generation gene-editing projects that the company hopes will be more potent and that should enter the clinic later this year. We engineer ZFNs for precision, efficiency, and specificity, which we believe are the critical parameters for a therapeutic genome editing technology. The current standard of care is enzyme replacement therapy (ERT) with Shire's Elaprase, but this requires weekly infusions. Of the high dose group, one patient could not undergo biopsy to assess gene integration, and results from the other, patient six in the trial, are still pending. We use cookies on this website. Update for the Alta Study, a Phase 1/2 Gene Therapy Trial of Giroctocogene Fitelparvovec (SB-525) in Adults With Severe Hemophilia A Sangamo scientists today are presenting data demonstrating that the company’s engineered zinc finger protein transcription factors (ZFP-TFs) specifically and powerfully repress key genes involved in brain diseases including Alzheimer’s, … Three doses of SB-913 are being tested: 5x1012vg/kg, 1x1013vg/kg, and 5x1013vg/kg. BRISBANE, Calif., March 8, 2019 /PRNewswire/ -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, announced today the publication in Nature Communications of improvements to its zinc finger nuclease (ZFN) platform technology, which yield a 64-fold increase in the diversity of ZFNs available for targeting any DNA segment. The company is now putting its hopes on increasing the dosage of SB-913. The company contended that, despite the liver toxicity signal, the plasma IDS data from patient six gave hope. zealinsider January 7, 2021 0 The glimmer of hope here for Sangamo is patient six, whose transient plasma IDS expression might hint at a dose response and the potential for higher dosing. Yesterday's presentation cast doubt on most of those hypotheses. Sangamo Therapeutics is working with Pfizer on a hemophilia A gene therapy that doesn't use zinc-finger proteins. By using this site, you agree that we may store and access cookies on your device. Sangamo Announces Interim Results Of Phase 1/2 EMPOWERS Study Evaluating SB-318 Zinc Finger Nuclease (ZFN) In Vivo Genome Editing Demonstrating Increased Leukocyte IDUA Activity In Patients With MPS I | Sangamo Therapeutics, Inc. Sangamo should be applauded for producing the first US data with in vivo genome editing. This technique has broad applicability, namely for the treatment of hemophilia B and other disorders. The contagion was not contained to just Sangamo and its older zinc finger nuclease genome editing technology; names like Crispr Therapeutics, Intellia, Editas Medicine and even Sarepta were also dragged down by this setback. Our zinc finger nuclease (ZFN) technology is based on a naturally occurring class of proteins called zinc finger DNA-binding proteins (ZFPs), which recognize and bind to specific sequences of DNA. On the question of whether the IDS gene had been integrated, Sangamo reported mixed results. Higher dose points Sangamo no closer to zinc finger success. Sangamo Therapeutics, Inc. is an American biotechnology company based in Brisbane, California. Once bound to the target sequence of DNA, a transcriptional repressor domain attached to the ZFP … Highlighted Sangamo’s zinc finger protein in vivo genome regulation programs and capabilities and research detailing Sangamo’s engineered adeno-associated virus (AAV) ... Net loss attributable to Sangamo Therapeutics, Inc. stockholders $ (35,929 ) $ (30,284 ) $ (78,842 ) $ Sangamo Therapeutics Announces Nature Medicine Publication Detailing the Activity of Disease Allele-Selective Zinc Finger Proteins in Preclinical Models of … ZFN has a much longer track record than CRISPR does. Sangamo Therapeutics is also conducting earlier stage research on ZFN gene-editing approaches that may someday help patients with hemophilia B and MPS I, … The two medium-dose patients showed evidence of gene integration, but had no increase in plasma IDS activity. Sangamo Therapeutics (formerly Sangamo BioSciences) hopes zinc fingers have the Midas touch when it comes to regulating gene expression. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. But patient six was the only one to show a jump in plasma IDS, though this faded after liver enzyme elevations were seen. Sangamo's been advancing zinc finger gene editing tech for decades without a single late-stage clinical trial success to show for it. As this enzyme usually breaks down glycosaminoglycans (GAGs), these accumulate in MPS II patients, causing tissue and organ damage. Instead, Gilead will partner with Sangamo Therapeutics, a Richmond, Calif.-based company that pioneered an older gene-editing tool called the zinc finger nuclease (ZFN). Are the stars waning for Pfizer’s haemophilia A gene therapy? But one mid-dose patient resumed ERT after three months when he experienced fatigue and an increase in urinary GAGs. In MPS II, also known as Hunter syndrome, the mutated IDS gene results in an absence of iduronate-2-sulfatase. Data from the first patients treated with hemophilia A therapy is expected this year. Investors, many of whom have kept faith with the company for years, have finally lost patience. Sangamo Therapeutics recently presented what it characterized as key improvements to its technology platform for engineering highly specific zinc finger nucleases (ZFNs), which are used for targeted editing of the human genome. Sangamo’s zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation approach is designed to either selectively repress (down-regulate) or activate (up-regulate) the expression of a specific gene or DNA sequence with a single administration. sangamo therapeutics inc (sgmo) - develops zinc finger dna-binding proteins for the therapeutic regulation of disease-associated genes. Our broad collection of publications, authored by our own scientists, collaborators, and others, contains original research articles and review articles that are relevant to our ZFP technology and its many applications. Our technology team continues to optimize ZFNs for the three key dimensions of genome editing: precision, efficiency, and specificity. 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