{"id":54805,"date":"2023-03-14T12:07:27","date_gmt":"2023-03-14T11:07:27","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/in-pivotal-demonstration-epic-bio-accomplishes-persistent-gene-activation-with-single-administration\/"},"modified":"2023-03-14T12:07:27","modified_gmt":"2023-03-14T11:07:27","slug":"in-pivotal-demonstration-epic-bio-accomplishes-persistent-gene-activation-with-single-administration","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/in-pivotal-demonstration-epic-bio-accomplishes-persistent-gene-activation-with-single-administration\/","title":{"rendered":"In Pivotal Demonstration, Epic Bio Accomplishes Persistent Gene Activation with Single Administration"},"content":{"rendered":"
\n

\n– New data detail discovery and engineering of hypercompact transcriptional activation domains with greater magnitude and duration of effect than any previously described –<\/i>\n<\/p>\n

\n– Epic Bio is advancing a pipeline of therapies to precisely modulate gene expression to address previously intractable diseases –<\/i>\n<\/p>\n

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Epic Bio<\/a>, a leading epigenome engineering company developing therapies to modulate gene expression using ultracompact, non-cutting dCas proteins, has presented new data demonstrating the achievement of persistent gene activation using its Gene Expression Modulation System (GEMS) platform. The data were presented at the Keystone Symposia\u2019s Chromatin Architecture in Development and Human Health conference, taking place March 12-15, 2023, in British Columbia, Canada.\n<\/p>\n

<\/a><\/p>\n

\n\u201cThese data are a thrilling proof-of-principle for our GEMS platform\u2019s ability to generate new therapeutic constructs that can overcome longstanding challenges within genetic medicine. In this case those challenges were two-fold: no one has yet demonstrated epigenetic gene activators with more than a short-lived effect, and secondly, even those short-lived activators are too large to be packaged within a clinically validated AAV vector, limiting their use as in vivo therapies,\u201d said Dan Hart, Ph.D., head of technology development at Epic Bio. \u201cHere we\u2019ve documented discovery of novel activators that successfully overcome those challenges, producing durable activation that is passed down through cell divisions after just a single, transient exposure to our GEMS construct.\u201d\n<\/p>\n

\nThe novel activators were discovered through the use of a proprietary cell-based reporter platform, and then optimized for greater potency and smaller size using machine learning algorithms through successive rounds of protein engineering and evaluation in cellular models. The activators, when fused to a dCas and targeted to native gene promoters, were observed to have a greater magnitude and duration of effect (up to 80 cell divisions) compared to standard multi-protein domain fusions such as VPR, which is only durable for a dozen cell divisions. Furthermore, the coding length of the entire construct was just 1\/10th<\/sup> that of comparator approaches, allowing it to fit within a single AAV vector.\n<\/p>\n

\n\u201cThis study highlights all the powerful differentiators built into our GEMS platform: non-cutting, precisely targeted modulation of gene expression produced by a smaller payload than has ever before been possible,\u201d said Amber Salzman, Ph.D., chief executive officer of Epic Bio. \u201cThe kind of persistent activation we\u2019ve shown here could be transformative for the treatment of haploinsufficient diseases, by offering a way to durably restore gene expression to normal physiological levels. As we continue to advance our first GEMS candidate toward clinical readiness, we are excited to build upon these findings and to rapidly work to translate them into meaningful therapies for people with previously intractable diseases.\u201d\n<\/p>\n

\nA PDF of the poster may be accessed under the Science<\/a> section of Epic Bio\u2019s website.\n<\/p>\n

\nAbout Epic Bio<\/b>\n<\/p>\n

\nEpic Bio is a leading epigenome engineering company, leveraging the power of CRISPR without cutting DNA. The company is using its proprietary Gene Expression Modulation System (GEMS) to develop therapies. Through the company\u2019s library of the most compact Cas DNA-binding proteins to work on human cells, the company is developing in vivo therapies with delivery via a single AAV vector. Epic Bio has an initial focus on Facioscapulohumeral Muscular Dystrophy (FSHD) and is conducting additional research to address Antitrypsin Deficiency (A1AD), Heterozygous Familial Hypocholesterolemia (HeFH), as well as other indications. The company is financially backed by Horizons Ventures and other leading investors. Visit www.epic-bio.com<\/a> for more information or follow us on Twitter<\/a> and LinkedIn<\/a>.\n<\/p>\n

Contacts<\/b> <\/p>\n

\nInvestor Contact<\/b>
Shawn M. Cox
\n
Manager, Investor Relations, Corporate Communications
\n
Epic Bio
\n
shawn.cox@epic-bio.com<\/a>\n<\/p>\n

\nMedia Contact<\/b>
Wendy Ryan
\n
Ten Bridge Communications
\n
wendy@tenbridgecommunications.com<\/a>
(781) 316-4424\n<\/p>\n<\/div>\n","protected":false},"excerpt":{"rendered":"

– New data detail discovery and engineering of hypercompact transcriptional activation domains with greater magnitude and duration of effect than any previously described – – Epic Bio is advancing a pipeline of therapies to precisely modulate gene expression to address previously intractable diseases – SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Epic Bio, a leading epigenome engineering company … [Read more…]<\/a><\/span><\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[13],"tags":[],"class_list":{"0":"entry","1":"post","2":"publish","3":"author-business","4":"post-54805","6":"format-standard","7":"category-industry"},"yoast_head":"\nIn Pivotal Demonstration, Epic Bio Accomplishes Persistent Gene Activation with Single Administration - Pharma Trend<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/pharma-trend.com\/en\/in-pivotal-demonstration-epic-bio-accomplishes-persistent-gene-activation-with-single-administration\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"In Pivotal Demonstration, Epic Bio Accomplishes Persistent Gene Activation with Single Administration - Pharma Trend\" \/>\n<meta property=\"og:description\" content=\"– New data detail discovery and engineering of hypercompact transcriptional activation domains with greater magnitude and duration of effect than any previously described – – Epic Bio is advancing a pipeline of therapies to precisely modulate gene expression to address previously intractable diseases – SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Epic Bio, a leading epigenome engineering company ... 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