YouthBio Therapeutics has received positive feedback from the U.S. Food and Drug Administration (FDA) following an INTERACT meeting regarding its investigational gene therapy, YB002, aimed at treating Alzheimer’s disease. The FDA’s response indicates that existing preclinical data support the bioactivity of YB002 and the company’s proposed plans for a first-in-human trial.
YB002 is a gene therapy designed to transiently express a set of protein transcription factors known as OSKM (Oct4, Sox2, Klf4, and c-Myc) in the brain. This approach stems from the Nobel Prize-winning discovery by Shinya Yamanaka in 2006 that adult cells could be reverted to an embryonic-like state by exposure to these factors. Rather than fully reprogramming cells back into stem cells, YB002 uses transient expression to reverse age-related epigenetic changes while maintaining cell identity. The goal is to restore youthful gene expression patterns, improve cellular resilience, and address the underlying drivers of Alzheimer’s pathology.
With the FDA’s endorsement of its preclinical data, YouthBio plans to prioritize chemistry, manufacturing, and controls (CMC) activities along with a pilot toxicology study ahead of a pre-IND meeting to finalize designs for IND-enabling studies. The company aims to initiate clinical trials in approximately three years.
This development marks a significant step toward initiating human trials for what is described as a first-of-its-kind gene therapy targeting Alzheimer’s disease.
