{"id":373351,"date":"2026-07-02T09:26:04","date_gmt":"2026-07-02T09:26:04","guid":{"rendered":"https:\/\/wolfscientific.com\/?p=373351"},"modified":"2026-07-02T09:26:04","modified_gmt":"2026-07-02T09:26:04","slug":"promoting-genetic-medicine","status":"publish","type":"post","link":"https:\/\/wolfscientific.com\/?p=373351","title":{"rendered":"Promoting Genetic Medicine"},"content":{"rendered":"

\u2018We are now effectively reshaping these highly intriguing DNA-targeting technologies into something that resembles a therapeutic much more,\u2019 states Benjamin Oakes, CEO of Scribe Therapeutics<\/a> \u2013 a genetic medicine company based in the US that is customizing Crispr editing technology to create both genetic and epigenetic therapies.<\/p>\n

Oakes co-established Scribe in 2017 with three former peers from the University of California, Berkeley. Among them were Oakes\u2019 postgraduate mentors \u2013 molecular biologist David Savage and Nobel-laureate Crispr trailblazer Jennifer Doudna \u2013 along with Brett Staahl, a past postdoc from the Doudna lab.<\/p>\n

After previously researching DNA-targeting with zinc finger proteins, Oakes became part of Doudna\u2019s research group in 2014, motivated by the \u2018significant shift\u2019 in genome targeting that her Crispr tools were poised to bring about. Upon finishing his studies, Oakes was committed to discovering methods to utilize the technology within the medical field.<\/p>\n

\u2018The overarching ambition that we had at the inception of Scribe was that genetic medicine ought to be accessible to everyone, as we could all reap its benefits,\u2019 Oakes shares. \u2018The subsequent question is, how do you achieve that?\u2019 Oakes elaborates that the answer would entail years of meticulous effort to gradually enhance the potency and precision of Crispr\u2013Cas genetic editing systems \u2013 converting them from naturally evolved elements of bacterial defense into precisely calibrated therapeutic instruments. \u2018I\u2019m unlikely to publish another Nature Biotech paper, because I enhance the potency of a molecule by 10% every six months for seven years. However, when you accomplish this, you end up with a remarkably different molecule,\u2019 Oakes observes.<\/p>\n

A fundamental aspect of Scribe\u2019s platform is the CasX enzyme family<\/a>. As per Oakes, these \u2018extremely rare, highly intriguing\u2019 proteins perfectly meet the criteria \u2018in terms of being functional, but also exceptionally specific, and small enough to be integrated into any delivery system\u2019. From 2019 to 2021, Scribe dedicated three years to refining the CasX proteins into what it currently refers to as its X-Editor, which Oakes characterizes as \u2018the industry-leading genome editor\u2019 that exhibits high uptake into liver cells while minimizing off-target effects.<\/p>\n

At that juncture, Scribe secured $100 million (\u00a374 million)<\/a> in funding to enhance the X-Editing platform and progress toward its aim of \u2018making genetic medicine available to all\u2019. Focused on impacting as many individuals as possible, the company has shifted its attention toward cardiovascular disease, the leading global cause of mortality.<\/p>\n

This year, the organization is set to initiate a phase 1 human trial of an epigenetic editor designed to target \u2018bad\u2019 low-density lipoprotein cholesterol (LDL-C) \u2013 a significant factor in atherosclerotic cardiovascular disease (ASCVD). In doing so, Scribe joins a limited number of genetic medicine companies that are starting to explore the safety of therapies based on targeted epigenetic editing<\/a> in humans. Instead of making permanent genetic modifications, these epigenetic editors modify chemical tags on DNA that control gene expression \u2013 either increasing or diminishing the rate at which particular genes are converted into proteins.<\/p>\n

Scribe\u2019s STX-1150 candidate therapy aims to epigenetically silence the PCSK9<\/a> gene, thereby diminishing the production of low-density lipoprotein cholesterol (LDL-C). The company initially created a gene editor targeting PCSK9, but subsequently also developed the epigenetic editor, as Oakes explains. \u2018The epigenetic molecule turned out to be not only potentially a more universally applicable molecule, but one that yielded better data.\u2019 Scribe\u2019s editor is enhanced through the integration of an allosteric control loop, detailed in a recent preprint<\/a>, which helps to further improve specificity for the target gene and minimize off-target methylation.<\/p>\n

Preclinical studies demonstrated<\/p>\n","protected":false},"excerpt":{"rendered":"

\u2018We are now effectively reshaping these highly intriguing DNA-targeting technologies into something that resembles a therapeutic much more,\u2019 states Benjamin Oakes, CEO of Scribe Therapeutics \u2013 a genetic medicine company based in the US that is customizing Crispr editing technology to create both genetic and epigenetic therapies. Oakes co-established Scribe in 2017 with three former […]<\/p>\n","protected":false},"author":2,"featured_media":373352,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"Default","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[174],"class_list":["post-373351","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized","tag-source-chemistryworld-com"],"_links":{"self":[{"href":"https:\/\/wolfscientific.com\/index.php?rest_route=\/wp\/v2\/posts\/373351","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/wolfscientific.com\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/wolfscientific.com\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/wolfscientific.com\/index.php?rest_route=\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/wolfscientific.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=373351"}],"version-history":[{"count":0,"href":"https:\/\/wolfscientific.com\/index.php?rest_route=\/wp\/v2\/posts\/373351\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/wolfscientific.com\/index.php?rest_route=\/wp\/v2\/media\/373352"}],"wp:attachment":[{"href":"https:\/\/wolfscientific.com\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=373351"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/wolfscientific.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=373351"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/wolfscientific.com\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=373351"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}