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DTSTART;TZID=America/Los_Angeles:20260331T170000
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SUMMARY:BioScience Forum Presents: Precision CRISPR Therapies
DESCRIPTION:BioScience Forum Presents: Precision CRISPR Therapies\nAdvances in biotechnology are transforming the future of medicine\, and one of the most exciting breakthroughs in recent years is the development of CRISPR-based therapies. Scientists around the world are exploring how genome editing can be used to treat complex diseases that previously had limited treatment options. \nThe upcoming BioScience Forum Presents: Precision CRISPR Therapies offers a unique opportunity for professionals\, researchers\, and science enthusiasts to learn about the latest progress in this revolutionary field. The event will feature insights from Aarif Khakoo\, who will discuss how Scribe Therapeutics is applying advanced genome-editing technology to develop therapies for cardiometabolic diseases. \nThe forum will take place at The Alexandria at San Carlos\, bringing together members of the regional bioscience community for an evening of learning\, networking\, and scientific discovery. \nPrecision CRISPR Therapies: Targeting Cardiometabolic Pathways\nThe focus of the event is the cutting-edge topic “Precision CRISPR Therapies: Targeting Cardiometabolic Pathways with In Vivo Genome Editing.” \nDeveloping effective therapeutics requires optimizing several critical factors\, including potency\, specificity\, and deliverability. These factors are especially important when using genome-editing technologies like CRISPR gene editing\, which allow scientists to make precise changes to DNA within living organisms. \nScribe Therapeutics has developed a strategy known as CRISPR by Design™\, an innovative approach that carefully engineers CRISPR tools to maximize safety and effectiveness. This strategy supports the company’s internal research programs targeting cardiometabolic diseases\, while also contributing to collaborations focused on neurological\, neuromuscular\, and genetic conditions such as Sickle Cell Disease. \nA New Starting Point: CasX\nA key innovation behind Scribe’s work is its use of CasX\, a novel CRISPR enzyme. \nCasX offers several advantages compared to other genome-editing systems. It is relatively compact\, easier to deliver into cells\, and potentially safer. By using this enzyme as the foundation for their research\, Scribe scientists have engineered powerful platforms designed to address genetic diseases more effectively. \nThese technologies aim to set new standards for genetic medicine\, opening doors to treatments that target the root causes of disease rather than only managing symptoms. \nTwo Powerful Platforms: ELXR and XE\nScribe has created two groundbreaking platforms designed to deliver precision therapies. \nThe first platform is ELXR\, which stands for Epigenetic Long-Term X-Repressor. This technology allows scientists to silence specific genes without cutting the DNA itself. Instead of editing the genetic code directly\, ELXR modifies how genes are expressed\, providing a safer method of controlling harmful genetic activity. \nThe second platform is XE\, or X-Editor\, which enables direct gene editing with extremely high precision. One of its most promising features is the absence of detectable off-target editing\, meaning it can modify target genes without unintentionally affecting other parts of the genome. \nTogether\, these platforms represent an important step toward developing highly targeted and reliable gene therapies. \nThe STX-1150 Program\nOne of Scribe’s most promising research programs is STX-1150\, which uses the ELXR platform to address high cholesterol levels by targeting the PCSK9 gene in the liver. \nPCSK9 plays a major role in regulating low-density lipoprotein cholesterol\, commonly known as LDL cholesterol. \nIn preclinical studies involving non-human primates\, therapeutic dosing of less than 1 mg per kilogram successfully reduced LDL cholesterol levels. The gene silencing effect was both potent and long-lasting\, suggesting that this therapy could offer a powerful alternative to existing cholesterol-lowering treatments. \nSuch advances could benefit individuals at high risk of Atherosclerotic Cardiovascular Disease\, a major cause of heart attacks and strokes worldwide. \nThe STX-1200 Program\nAnother promising development from Scribe is the STX-1200 program. \nThis therapy uses the XE gene-editing platform to directly target the Lipoprotein(a) gene. \nElevated Lipoprotein(a) levels are linked to increased cardiovascular risk\, but treatment options for reducing these levels remain limited. \nIn preclinical models\, the STX-1200 program demonstrated greater than 90% knockdown of Lipoprotein(a) levels\, a remarkable achievement that highlights the potential of precision genome editing. \nDelivery Strategies for Gene Editing\nAnother key aspect of developing successful CRISPR therapies is delivering the gene-editing tools safely into the body. \nScribe has explored multiple strategies for in vivo delivery\, including: \n\nAdeno-associated virus\nLipid Nanoparticles\n\nBoth technologies have become important tools in modern biotechnology. They allow therapeutic molecules to reach specific tissues within the body\, enabling scientists to target disease at the molecular level. \nInsights from Dr. Aarif Khakoo\nDuring the forum\, Aarif Khakoo will share insights into the technical challenges and opportunities associated with developing these therapies. \nHis presentation will highlight: \n\nThe scientific strategy behind Scribe’s CRISPR platforms\nThe latest preclinical data supporting ongoing programs\nThe next steps toward clinical evaluation of these therapies\n\nAttendees will also have the chance to participate in a Q&A session\, offering an opportunity to engage directly with the speaker and discuss the future of genome editing. \nEvent Agenda\nThe evening will include both networking opportunities and scientific discussion. \n5:00 PM – 6:00 PM | Networking\, Appetizers\, and Drinks\nGuests can connect with local bioscience professionals while enjoying appetizers and refreshments. \n6:00 PM – 7:00 PM | Presentation\nThe scientific presentation will be delivered by the guest speaker and followed by an interactive Q&A session. \nEvent Details\nThe forum will take place on: \nTuesday\, March 31\n5:00 PM (GMT-07:00) \nat The Alexandria at San Carlos. \nA Glimpse into the Future of Medicine\nThe BioScience Forum Presents: Precision CRISPR Therapies provides an exciting opportunity to explore how genome editing is transforming modern healthcare. \nWith the potential to treat complex conditions such as cardiovascular disease\, genetic disorders\, and neurological illnesses\, CRISPR-based technologies are paving the way for a new era of precision medicine. \nBy bringing together scientists\, professionals\, and innovators\, events like this help foster collaboration and inspire the next generation of breakthroughs in biotechnology. \nFor those passionate about the future of science and medicine\, this forum promises an evening filled with knowledge\, inspiration\, and meaningful connections. \n  \n  \n  \n\nSee other interesting events in San Carlos. You may also check the main calendar for the dates of these\, or to save them on your personal calendar. \n\n\nDon't miss out!Subscribe To San Carlos LifeAdd your email here to subscribe to all things San Carlos Invalid email address Give it a try. You can unsubscribe at any time.Thanks for subscribing!\n\nAbout San Carlos\nWhat’s Great about San Carlos? \n \nAbout Mark and Viv\nMark Martinho and Viv Kelvin are the owners of Vabrato Real Estate
URL:https://sancarloslife.com/event/bioscience-forum-therapies/
LOCATION:The Alexandria\, 835 Industrial Road\, San Carlos\, 94070\, United States
CATEGORIES:Adult Activities,Main Events,The Alexandria
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