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YB002 is YouthBio's lead candidate, a first-in-class gene therapy for Alzheimer's disease. It is designed to safely and transiently express the OSKM Yamanaka factors (OCT4, SOX2, KLF4, c-MYC) in the brain — a process known as partial reprogramming — to reverse accumulated epigenetic changes while preserving neuronal identity and restoring more youthful gene expression. In September 2025 the company reported a positive FDA INTERACT meeting, with the agency agreeing that existing preclinical data support the candidate's bioactivity and the proposed first-in-human trial, establishing a path toward the clinic for a brain-directed reprogramming therapy.
YouthBio's platform delivers partial cellular reprogramming in a tissue-specific manner, using gene therapy to transiently express reprogramming transcription factors in targeted cells. The transient, controlled expression is intended to reset age-related epigenetic marks and restore youthful function without driving cells to full pluripotency or erasing their identity — a key safety consideration for reprogramming in vivo. The platform stems from Shinya Yamanaka's Nobel-winning reprogramming discovery and is aimed initially at neurodegenerative conditions, with YB002 for Alzheimer's as the lead program and additional brain-disease applications envisioned.
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