YB002 by YouthBio Therapeutics: status and timeline

Milestones

Next · CMC manufacturing optimization

Company founded and YB002 program initiated2021Complete.
Preclinical mouse studies show memory rescue2023Complete.
FDA INTERACT meeting with positive feedback29 Sep 2025Complete.
CMC manufacturing optimizationTarget 2026Current milestone.
Pilot toxicology studiesTarget 2026Not yet reached.
Pre-IND meeting with FDATarget 2027Not yet reached.
IND application submissionTarget 2028Not yet reached.
First-in-human clinical trialTarget 2028Not yet reached.

Upcoming

2026CMC manufacturing optimization (next)
2026Pilot toxicology studies
2027Pre-IND meeting with FDA
2028IND application submission
2028First-in-human clinical trial

Current obstacles

First human brain reprogramming therapyNo partial reprogramming tested in patients; mouse-to-human translation needs safety studies.
Brain targeting and specificityTherapy must reach right brain regions without off-target epigenetic changes.
Patient selection and endpointsWhich Alzheimer's patients benefit and how to measure cognitive improvement remains uncertain.

Physics limits

Fixed transgene expressionSingle injection sets expression level once; cannot be easily adjusted if reprogramming is too strong or weak.
Brain is hard to reachBlood-brain barrier blocks most molecules. Brain injection is invasive; large-scale use faces practical limits.
Multiple disease mechanismsAlzheimer's involves amyloid, tau, neuroinflammation and metabolism. Reprogramming addresses aging but not all.

How it works

4 parts

Gene delivery

Inducible brain transgene

Injection into the brain delivers OSKM transgene to neurons; it remains inactive until pill.

Activation

Transient factor expression

Oral pill triggers temporary Oct4, Sox2, Klf4, c-Myc expression; stops when pill ends.

Cell effects

Reversal of epigenetic aging

Age-related epigenetic marks reset in brain cells while preserving cell function.

Benefit

Restored neuronal function

Mouse models show memory and learning rescue by reversing neurodegeneration.

Spec sheet

Spec	YB002
Reprogramming approach	Transient OSKM expression^R (reported)
Administration route	Brain injection + oral pill^R (reported)
Primary indication	Alzheimer's disease^R (reported)

^R reported by the company

About YouthBio Therapeutics

The team behind YB002

YouthBio Therapeutics

Partial epigenetic reprogramming, gene therapy, Alzheimer's

YouthBio develops gene therapies using partial epigenetic reprogramming to treat Alzheimer's. Lead program YB002 reverses age-related changes in the brain while preserving cell identity. Positive FDA feedback September 2025.

YB002 is a brain injection plus oral pill to reverse neurodegeneration at the epigenetic level.
FDA confirmed preclinical data and trial plan in September 2025 INTERACT meeting.
Manufacturing optimization and toxicology studies underway; first-in-human trials expected 2028–2029.

youthbiotx.com (opens youthbiotx.com)Company profile →

Founded: 20215 yrs
Headquarters: United States
Status: Private
Valuation: private
Last round: Incubator/AcceleratorJul 2023
Works in: LongevityCellular reprogramming
Coverage: 1 program · 1 updatelatest 29 Sep 2025checked 10 Oct
Lead investors: MassChallenge
Investors: MassChallenge (opens youthbiotx.com)
People: Yuri DeiginCEO and Co-founderViet LyCFO and Co-founderJoão Pedro de Magalhães (opens birmingham.ac.uk)Chief Scientific Officer

Spec

YB002

Reprogramming approach

Transient OSKM expression^R (reported)

Administration route

Brain injection + oral pill^R (reported)

Primary indication

Alzheimer's disease^R (reported)