Drug intelligence / Profile preview

Oct4 + Sox2 + Klf4 gene therapy

Development stage
Preclinical
Lead developer
Life Biosciences
Modality
Gene Therapies
Administration
Intravitreal
01

Overview

OSK gene therapy is an experimental regenerative medicine approach that utilizes three of the four Yamanaka factors—Oct4 (POU5F1), Sox2, and Klf4—to induce partial epigenetic reprogramming in aged or damaged cells. By delivering these transcription factors via a viral vector (typically AAV), the therapy aims to reset the epigenetic clock of cells, restoring youthful gene expression patterns and cellular function without inducing pluripotency or oncogenic transformation, a risk associated with the fourth factor, c-Myc. Developed primarily by Life Biosciences based on research from David Sinclair's laboratory at Harvard Medical School, the therapy is being investigated for its ability to reverse vision loss in conditions such as glaucoma and non-arteritic anterior ischemic optic neuropathy (NAION), as well as for broader applications in age-related systemic diseases.

Other names
OSK gene therapyPartial reprogramming therapyEpigenetic reprogramming therapy
02

Targets

KLF4 (Krüppel-like factor 4)POU5F1 (Octamer-binding transcription factor 4)SOX2 (SRY-box transcription factor 2)

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