The FDA\u2019s decision clears an investigational new drug application for Life Bioscience\u2019s ER-100, a gene therapy designed to rejuvenate damaged retinal cells in people with serious, age-related eye diseases. The Boston-based biotech co-founded by Harvard professor Dr David Sinclair, who showed in 2020 that a partial epigenetic reprogramming treatment restored vision in blind mice<\/a>, is now preparing to commence a Phase 1 first-in-human study to show its therapy can be delivered safely in patients with open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION).<\/p>\n Longevity.Technology: This is clearly a huge moment for longevity, and one that has potential implications for many other companies, including the likes of Altos Labs<\/a>, NewLimit<\/a> and Retro Biosciences<\/a>. However, it also represents a pragmatic step forward for the field. While it does not resolve the many questions surrounding human rejuvenation, it does place epigenetic reprogramming on a familiar path of dose finding, safety monitoring and measured clinical validation, and brings the promise of epigenetic rejuvenation a little closer to therapeutic reality. To find out more, we caught up with Life Bio CEO Jerry McLaughlin and CSO Dr Sharon Rosenzweig-Lipson.<\/p>\n While Yamanaka\u2019s discovery that four transcription factors (Oct4, Sox2, Klf4 and c-Myc) could reset a cell to an embryonic-like state, researchers have since sought to adapt the biology in ways that preserve cell identity and reduce cancer risk.<\/p>\n Hailing the IND clearance as \u201ca major inflection point for the longevity and aging biology field,\u201d McLaughlin told us \u201csince Shinya Yamanaka first showed that cellular age could be reset through the reprogramming factors OSKM, the potential of translating that biology into real medicines has been enormous yet has previously remained largely theoretical.\u201d<\/p>\n Primary focus on safety <\/p>\n As a first-in-human trial, Life Bio\u2019s study is primarily focused on safety and tolerability. Instead of using all four Yamanaka factors, ER-100 employs three of the factors (OSK) delivered transiently to reset age-associated epigenetic markers while keeping cells committed to their original function. By excluding c-Myc, a factor associated with uncontrolled growth, the strategy is intended to lower the risk of tumors that has historically concerned regulators and clinicians.<\/p>\n From a safety perspective, Rosenzweig-Lipson told us that the company\u2019s preclinical studies in non-human primates demonstrated that ER-100 was well tolerated in NHPs, with no systemic toxicities.<\/p>\n \u201cThe therapy also uses a doxycycline-inducible system, giving us precise control over when the genes are active and allowing treatment to be paused or stopped if needed,\u201d she added. \u201cIn addition, ER-100 is delivered locally to the eye, limiting systemic exposure. Multiple preclinical animal models have demonstrated controlled gene expression, favorable biodistribution, restoration of epigenetic markers, and improvements in visual function which has collectively provided the foundation for FDA clearance.\u201d<\/p>\n The clinical trial will monitor immune responses and ocular effects while also collecting exploratory data on visual function and retinal health.<\/p>\n \u201cAt this stage, success means demonstrating that ER-100 can be administered safely,\u201d said Rosenzweig-Lipson. \u201cAny signals of improvement in visual parameters would be highly encouraging and would help inform the direction of subsequent efficacy-focused trials.\u201d<\/p>\n A therapeutic platform for age-related disease <\/p>\n If the initial study identifies a safe dose and delivery regimen, Life Bio plans to move into larger trials designed to test whether partial epigenetic reprogramming can meaningfully preserve or restore vision. More broadly, the company views the trial as a test case for an entire therapeutic platform.<\/p>\n \u201cDemonstrating that partial epigenetic reprogramming can be deployed in humans opens the door to applying the same approach to other age-related diseases and organ system,\u201d said Rosenzweig-Lipson.<\/p>\n While the eye is seen as an ideal starting point because it is accessible and allows for localized treatment, Life Bio is already exploring other potential applications for its technology. The company\u2019s near-term vision is to build evidence organ by organ and disease by disease.<\/p>\n \u201cPreclinical data already suggest applicability beyond ophthalmology, including in liver diseases like MASH,\u201d said McLaughlin. \u201cWhile systemic or multi-organ rejuvenation remains a longer-term goal, the current strategy is to establish rigorous clinical proof in specific age-related conditions. Over time, this disciplined approach could support broader applications and new delivery modalities.\u201d<\/p>\n \u201cLife Bio is starting by applying this biology to clearly defined age-related diseases with measurable endpoints. This shift from aspiration to evidence-based clinical development is helping move the whole field forward.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"A milestone moment for longevity as Life Biosciences gets green light to progress cellular rejuvenation trial in eye…\n","protected":false},"author":2,"featured_media":262648,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[34],"tags":[4399,137939,61304,61305,163,521,85,1037,46,137940,137941,11186,43,137942,137943],"class_list":{"0":"post-262647","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-healthcare","8":"tag-clinical-trials","9":"tag-david-sinclair","10":"tag-epigenetic-rejuvenation","11":"tag-epigenetic-reprogramming","12":"tag-health","13":"tag-healthcare","14":"tag-il","15":"tag-interview","16":"tag-israel","17":"tag-jerry-mclaughlin","18":"tag-life-biosciences","19":"tag-longevity","20":"tag-news","21":"tag-partial-cellular-reprogramming","22":"tag-sharon-rosenzweig-lipson"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/262647","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/comments?post=262647"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/262647\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media\/262648"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media?parent=262647"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/categories?post=262647"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/tags?post=262647"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}Jerry McLaughlin is CEO of Life Biosciences <\/p>\n
Sharon Rosenzweig-Lipson is Chief Scientific Officer at Life Biosciences. <\/p>\n