This would signal an even longer road to approval. Further data on patients who have withdrawn from ERT, due later this year, will also be worth watching, the company claims. Sangamo Therapeutics (formerly Sangamo BioSciences) hopes zinc fingers have the Midas touch when it comes to regulating gene expression. By using this site, you agree that we may store and access cookies on your device. Once bound to the target sequence of DNA, a transcriptional repressor domain attached to the ZFP … For fiscal 2018 and fiscal 2019, Sangamo is expected to generate revenues of $82.73 million and $89.39 million, respectively. 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. JP Morgan day one roundup – disappointments set the tone. 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 … 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. Sangamo Therapeutics' (NASDAQ:SGMO) zinc-finger nuclease approach to gene editing has been heralded as a potential solution for millions of … The two medium-dose patients showed evidence of gene integration, but had no increase in plasma IDS activity. 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. 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. It applies cell and gene therapy to combat haemophilia and other genetic diseases. 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. zealinsider January 7, 2021 0 The company is now putting its hopes on increasing the dosage of SB-913. The Sangamo (NASDAQ: SGMO) tech uses proteins known as zinc finger nucleases that bind DNA to edit genes. 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. Investors, many of whom have kept faith with the company for years, have finally lost patience. The evidence so far suggests that changing the expression of the gene in question has not altered the course of the rare inherited disease in which the project was being tested, mucopolysaccharidosis type II (MPS II). Source: Sangamo presentation, February 7, 2019. We engineer ZFNs for precision, efficiency, and specificity, which we believe are the critical parameters for a therapeutic genome editing technology. Sangamo Therapeutics, Inc. is an American biotechnology company based in Brisbane, California. The latest round of data, released on Thursday at the World Symposium on lysosomal diseases, drove Sangamo shares down 31%, to levels not seen in two years. ZFN has a much longer track record than CRISPR does. Sangamo and Pfizer are also collaborating for the development and commercialization of therapeutics using our zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene. It is little solace to Sangamo investors that the group's lead genome editing project, SB-913, seems to do what it is supposed to – edit a genetic mutation that leads to an enzyme deficiency. Genome editing works by using enzymes called “engineered nucleases” that act like molecular scissors. Higher dose points Sangamo no closer to zinc finger success. 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. Are the stars waning for Pfizer’s haemophilia A gene therapy? 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. Sangamo Therapeutics ; ASH 2020. 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. We engineer ZFNs for precision, efficiency, and specificity, which we believe are the critical parameters for a therapeutic genome editing technology. Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects. In the Champions trial, Sangamo had hoped to show that its zinc finger nuclease technology could insert a working IDS gene. 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. The company contended that, despite the liver toxicity signal, the plasma IDS data from patient six gave hope. Sangamo is not giving up yet. On the question of whether the IDS gene had been integrated, Sangamo reported mixed results. But patient six was the only one to show a jump in plasma IDS, though this faded after liver enzyme elevations were seen. Update for the Alta Study, a Phase 1/2 Gene Therapy Trial of Giroctocogene Fitelparvovec (SB-525) in Adults With Severe Hemophilia A 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. Sangamo Therapeutics is working with Pfizer on a hemophilia A gene therapy that doesn't use zinc-finger proteins. 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, … Snippet roundup: Wins for AcelRx, Foamix and SGLT2 developers. By cutting DNA at a precise spot in the genome, DNA can be removed, added or replaced for therapeutic effect. Our technology team continues to optimize ZFNs for the three key dimensions of genome editing: precision, efficiency, and specificity. 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. More patients began treatment with the 5x1013vg/kg dose at the beginning of 2019. About Sangamo’s Gene Regulation Platform Sangamo's zinc finger protein transcription factor (ZFP-TF) gene regulation technology is designed to either selectively repress (down-regulate) or activate (up-regulate) the expression of a specific gene or gene allele following a single administration. Genome editing makes permanent changes to the genetic code of a cell by correcting, disabling or modifying the DNA. Unfortunately, it might be the case that its older technology is not potent enough to have a therapeutic effect in this or any other disorder. ZFP Technology Platform. Sangamo Therapeutics uses a different approach to gene editing called zinc-finger nuclease (ZFN) technology. 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 is also conducting earlier stage research on ZFN gene-editing approaches that may someday help patients with hemophilia B and MPS I, … This technique has broad applicability, namely for the treatment of hemophilia B and other disorders. Aducanumab tops 2021’s biggest potential launches, The pandemic releases its grip on clinical trials, 2020 drug approvals rise despite Covid-19, Merck KGaA’s dual immuno-oncology weapon misfires, Zinc finger protein nuclease associated IDS gene editing, Zinc finger protein nuclease associated factor IX gene editing, Zinc finger protein nuclease associated IDUA gene editing, Zinc finger protein nuclease edited cell therapy. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. As this enzyme usually breaks down glycosaminoglycans (GAGs), these accumulate in MPS II patients, causing tissue and organ damage. 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. By way of comparison, Elaprase won approval with a 96-patient, 52-week trial that used pulmonary function and six-minute walk test as its endpoints. Novartis has agreed to license Sangamo Therapeutics’ zinc finger protein transcription factors (ZFP-TFs) to develop gene regulation therapies … Replace a gene, replace a weekly infusion. 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. But, even if more persuasive biomarker signs emerge, the study might not be sufficient for a regulatory filing. It will be crucial that, when the biopsy results come back, they show the gene to have been successfully integrated. Sangamo should be applauded for producing the first US data with in vivo genome editing. The presentation concerned six subjects, two from each of the three dosing groups. 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. Yesterday's presentation cast doubt on most of those hypotheses. Global Zinc Finger Nuclease Technology Market 2020-2025 (Impact of Covid-19) | Sigma-Aldrich, Sangamo Therapeutics, Labomics, Thermo Fisher Scientific, Gilead, etc. Replace a gene, replace a weekly infusion. The goal of genome editing is to provide a permanent therapeutic solution or cure for genetic diseases. Data from the first patients treated with hemophilia A therapy is expected this year. Sangamo is developing new genomic medicines which may address the underlying mistakes in DNA that lead to some genetic diseases. We use cookies on this website. Sangamo Therapeutics Announces Nature Medicine Publication Detailing the Activity of Disease Allele-Selective Zinc Finger Proteins in Preclinical Models of … But one mid-dose patient resumed ERT after three months when he experienced fatigue and an increase in urinary GAGs. Sangamo Therapeutics, Inc. is a Brisbane, California-headquartered clinical-stage genomics medicines company that is a pioneer in the research and development of zinc finger … Novartis is the latest big drug firm to strike a collaboration with Sangamo Therapeutics. Sangamo Therapeutics: The Promise of Zinc Fingers Real Estate The current standard of care is enzyme replacement therapy (ERT) with Shire's Elaprase, but this requires weekly infusions. Kite will use the ZFN gene editing … 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. In the low-dose group, one patient had no evidence of gene integration and one was not evaluable, being unable to undergo liver biopsy. Sangamo is best known for developing our proprietary gene editing technology, zinc finger nucleases (ZFNs). Ex vivo gene-edited cell therapy is the most straightforward application of ZFN technology. Unlocking the potential for precise genetic cures Since our founding in 1995, Sangamo has been a leader in genomic medicine research and development. 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). 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. Independent, data-driven daily news and analysis on pharma, biotech and medtech. In MPS II, also known as Hunter syndrome, the mutated IDS gene results in an absence of iduronate-2-sulfatase. sangamo therapeutics inc (sgmo) - develops zinc finger dna-binding proteins for the therapeutic regulation of disease-associated genes. Sangamo's been advancing zinc finger gene editing tech for decades without a single late-stage clinical trial success to show for it. Three doses of SB-913 are being tested: 5x1012vg/kg, 1x1013vg/kg, and 5x1013vg/kg. Three patients were taken off ERT, two in the medium-dose group and one in the high-dose. No measurable reduction in urine GAGs was identified in any patients, contrary to an earlier update from the same trial (Zinc fingers get the thumbs down, September 6, 2018). Gilead’s Kite has struck a $3 billion deal to access Sangamo Therapeutics’ zinc finger nuclease (ZFN) technology. Sangamo's ZFP-TF technology involves introducing an engineered zinc finger protein (ZFP) which is designed to identify and bind to a precise sequence of DNA. SCIENTIFIC AND MEDICAL PRESENTATIONS. Putting its hopes on increasing the dosage of SB-913 are being tested 5x1012vg/kg. Gene expression fingers have the Midas touch when it comes to regulating gene expression will also be worth,... A gene therapy, disabling or modifying the DNA enzyme replacement therapy ( ). The treatment of hemophilia B and other disorders a jump in plasma IDS activity of... Called zinc-finger nuclease ( ZFN ) technology or replaced for therapeutic effect of! 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