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    ER-100 (Life Biosciences' reprogramming therapy)

    Also known as ER-100, Life Bio ER-100

    ER-100 is an experimental gene therapy from Life Biosciences that delivers three Yamanaka factors (OSK) into the eye. In January 2026 it became the first partial epigenetic reprogramming therapy cleared by the FDA for human testing.[1][2] Early Phase 1 data from three glaucoma patients, released on 8 October 2026, showed no serious adverse events. The trial is designed to test safety, not efficacy.[3][4]

    Editor reviewedStrict sourcingUpdated Aging and longevity biologyLife sciencesHealth and medicine
    Key facts

    What ER-100 is

    ER-100 is a gene therapy that uses controlled expression of three transcription factors, OCT4, SOX2 and KLF4 (OSK).[1] These are three of the four Yamanaka factors, which together can turn adult cells into stem cells.[5] ER-100 is given as a single injection into the eye. Its factors are switched on with daily oral doxycycline for about eight weeks.[6] The genes travel in an AAV-2 viral vector, and patients take a tapering course of the steroid prednisolone to lower the risk of inflammation from an immune reaction to that vector.[7] For how such vectors work, see how gene therapy delivery works.

    The target cells are retinal ganglion cells, the neurons that connect the eye to the brain. They do not regenerate naturally, so damage to them causes permanent vision loss. NAION, one of the two conditions in the trial, is the most common sudden optic nerve disorder in adults over 50 and has no approved treatment, according to the company.[8]

    The approach builds on mouse work. In 2020, OSK expression in retinal nerve cells restored youthful DNA methylation patterns and reversed vision loss in a glaucoma model and in aged mice.[9] Those benefits depended on the DNA demethylases TET1 and TET2, which the authors took as evidence that tissues retain a record of youthful epigenetic information.[10] See partial-reprogramming. Life Biosciences was co-founded by David Sinclair, a Harvard geneticist and an author of that 2020 study.[11] Patents on OSK and on the rtTA4 switch that makes expression inducible have been licensed to the company.[12]

    Regulatory milestones

    • 28 January 2026: Life Biosciences announced FDA clearance of its IND, the first for a partial epigenetic reprogramming therapy.[2]
    • 8 April 2026: The company announced an $80 million Series D financing.[13]
    • 9 June 2026: First patient dosed in the Phase 1 trial (NCT07290244) in open-angle glaucoma and NAION, which evaluates safety and tolerability with additional visual-function endpoints.[14]

    The trial registration lists an estimated 18 participants, a single dose, follow-up of up to five years, and an estimated primary completion date of May 2027.[15]

    The trial targets eye diseases rather than aging; the FDA does not recognize aging as a disease, so aging-targeted therapies are developed through disease-specific pipelines.[16]

    First-in-human data (October 2026)

    On 8 October 2026 the company released data from three open-angle glaucoma participants who received the lowest dose. There were no dose-limiting toxicities, adverse events of special interest or serious adverse events.[3] The company reported that two of the three showed visual-field improvements at day 56.[4] The data safety monitoring board recommended moving to the second dose level, and the trial is ongoing.[17]

    Caveats

    These are company-reported results from three people in an early-stage study designed to assess safety.[3][4] Full reprogramming carries a known tumour risk in mice, which is why long-term follow-up matters for this class of therapy.[18] The trial’s five-year follow-up is designed with that in mind.[15] Whether eye results tell us anything about rejuvenating other tissues is an open question. Whole-body OSK gene therapy has so far been tested only in animals: in a 2024 study, it extended the remaining lifespan of very old male mice.[19]

    Questions readers ask

    Is ER-100 an anti-aging treatment?

    It is being tested for two eye diseases, open-angle glaucoma and NAION, not for aging, which the FDA does not recognize as a disease.[14][16]

    Did ER-100 improve vision?

    The company reported visual-field improvements in two of three patients at day 56, but the Phase 1 trial is designed to assess safety, so these are preliminary observations.[4]

    What happens next?

    The trial's safety board recommended escalating to the second dose level in glaucoma patients, and the trial is ongoing.[17]

    Sources

    Each numbered claim is a statement we checked against the sources listed with it. Status shows how well established it is.

    1. [1]

      ER-100 is a gene therapy that uses controlled expression of three transcription factors, OCT4, SOX2 and KLF4 (OSK). confirmedas of 2026-06-09

    2. [2]

      On 28 January 2026 Life Biosciences announced FDA clearance of its investigational new drug (IND) application for ER-100 in optic neuropathies, the first partial epigenetic reprogramming therapy cleared for human testing. confirmedas of 2026-01-28

    3. [3]

      In first-in-human data released on 8 October 2026, three open-angle glaucoma participants given the lowest ER-100 dose had no dose-limiting toxicities or serious adverse events. confirmedas of 2026-10-08

    4. [4]

      The company reported visual-field improvements in two of the three participants at day 56, in a Phase 1 study designed to assess safety rather than efficacy. reportedas of 2026-10-08

    5. [5]

      In 2006 researchers showed that adult mouse cells can be turned into induced pluripotent stem (iPS) cells by introducing four factors, Oct3/4, Sox2, c-Myc and Klf4. confirmedas of 2006-08-25

    6. [6]

      ER-100's factors are switched on with the oral antibiotic doxycycline, taken daily for about eight weeks after a single injection into the eye. confirmedas of 2026-10-08

    7. [7]

      ER-100 delivers OSK in an AAV-2 vector by a single injection into the eye; doxycycline switches expression on for 56 days, and patients take a tapering course of oral prednisolone to reduce the risk of inflammation from an immune response to the vector. confirmedas of 2026-10-08

    8. [8]

      Optic neuropathies damage retinal ganglion cells, the neurons connecting the eye to the brain, which do not naturally regenerate; NAION, the most common acute optic neuropathy in adults over 50, has no approved treatment, according to Life Biosciences. confirmedas of 2026-10-08

    9. [9]

      In 2020, expressing three factors (Oct4, Sox2 and Klf4, or OSK) in mouse retinal ganglion cells restored youthful DNA methylation patterns, promoted nerve regeneration and reversed vision loss in a glaucoma model and in aged mice. confirmedas of 2020-12-02

    10. [10]

      The vision and nerve-regeneration benefits of OSK reprogramming in mice required the DNA demethylases TET1 and TET2, suggesting cells retain a record of youthful epigenetic information. confirmedas of 2020-12-02

    11. [11]

      Life Biosciences was co-founded by Harvard genetics professor David Sinclair. confirmedas of 2026-01-30

    12. [12]

      Patents on OSK and the rtTA4 switch used for inducible expression have been licensed to Life Biosciences, according to a 2024 paper's conflict-of-interest statement. confirmedas of 2024-02-01

    13. [13]

      Life Biosciences announced an $80 million Series D financing on 8 April 2026. confirmedas of 2026-04-08

    14. [14]

      On 9 June 2026 Life Biosciences announced the first patient dosed in its Phase 1 trial of ER-100 (NCT07290244) in open-angle glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION), which evaluates safety and tolerability with additional visual-function endpoints. confirmedas of 2026-06-09

    15. [15]

      The ER-100 Phase 1 trial (NCT07290244) is recruiting an estimated 18 adults with open-angle glaucoma or NAION, gives a single dose, and follows participants for up to five years; its estimated primary completion date is May 2027. confirmedas of 2026-10-10

    16. [16]

      Papers published in 2025 in peer-reviewed journals state that the FDA does not recognize aging as a disease and that no regulatory framework exists for developing or approving therapies that target aging, so such therapies are developed through disease-specific pipelines. confirmedas of 2025-08-04

    17. [17]

      The trial's data safety monitoring board recommended escalating to the second dose level in glaucoma participants, and the trial is ongoing. confirmedas of 2026-10-08

    18. [18]

      Transient induction of the four Yamanaka factors inside living mice caused teratomas to emerge from multiple organs. confirmedas of 2013-09-11

    19. [19]

      In a 2024 study by researchers at Rejuvenate Bio, systemically delivered AAV vectors carrying inducible OSK extended the median remaining lifespan of 124-week-old male mice by 109% over controls and improved frailty scores. confirmedas of 2024-02-01

    20. [20]

      Altos Labs says its mission is to reverse disease, injury and disability by restoring cell health and resilience through cell rejuvenation. confirmedas of 2026-10-10

    Revision history (2)
    1. Page created.
    2. Refresh: added vector and steroid details, the target cells, trial size and follow-up, the founding and licensing background, and whole-body mouse data; replaced superscript excerpts with verbatim text.

    Created Oct 10, 2026. Last reviewed by an editor on Oct 10, 2026. Next scheduled review: Jan 10, 2027.

    Cite this page

    "ER-100 (Life Biosciences' reprogramming therapy)." ContentLora, updated Oct 10, 2026. https://contentlora.com/wiki/er-100

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