One first-in-class therapy. Multiple diseases. Real impact.
Rebuilding vascular health to change the course of serious disease.
We’re not chasing symptoms. We’re targeting the driver.
Many devastating diseases share a common thread: progressive vascular breakdown - including IPF (idiopathic Pulmonary Fibrosis), Dupuytren’s Contracture
and some forms of Alzheimer’s Disease.
X101 is designed to intervene at that upstream control point, rather than chasing downstream symptoms one by one.
A transformative, single drug solution-built to scale across indications.
We’re starting where the signal is strongest: CADASIL.
Our lead indication is CADASIL, a devastating inherited small-vessel disease of the brain with no FDA-approved disease-modifying therapies.
CADASIL is driven by NOTCH3 mutations that progressively damage vessel structure and blood flow, leading to recurrent strokes and cognitive decline over time.
CADASIL
A platform with broader reach.
X1 Biotech is prioritizing CADASIL to pursue a fast-track path through the FDA, leveraging an orphan-first strategy that can accelerate value creation and inform broader expansion.
Why X101 is different
Upstream control, not downstream patchwork
X101 is designed to modulate a shared upstream regulatory pathway implicated in vascular and fibrotic remodeling across tissues, creating the potential to advance one small molecule across multiple diseases (versus the traditional one-drug/one-indication model).
Preclinical evidence
In multiple animal and pre-clinical models, X101 has shown the potential to slow pathological progression and to partially restore structural features of damaged vasculature, including restored vascular smooth muscle cell coverage, an important marker of vessel stability and function.
X101 has also demonstrated biological activity across several disease-relevant models, including CADASIL, pulmonary fibrosis, Dupuytren’s contracture, eye disease models, and Alzheimer’s disease.
How it works: RUNX1 is the switch. X101 is the control.
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RUNX1 can bind DNA and initiate protein production involved in blood vessel development.
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PsychiatryX101 interacts with CBFβ and together they repress RUNX1 activity.
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RUNX1 is overexpressed in organs tied to target diseases.
Suppressing RUNX1 is associated with repair processes.
Partnerships
We welcome collaboration with:
Pharma/biotech partners in rare disease, fibrosis, neurology, ophthalmology
CADASIL clinical networks/foundations
CRO/CDMO partners for clinical ops, biomarkers, and GMP manufacturing
Strategic investors aligned with capital-efficient, milestone-based development.