One-time gene therapy to prevent alcohol-related bone loss in ALDH2-deficient postmenopausal East Asian women
Official title: Gene Therapy to Treat Ethanol-induced Osteoporosis Associated with Aldehyde Dehydrogenase 2 Deficiency in Post-menopausal East Asian Women
Funded at Enyx Therapeutics, LLC · NIH-11256090 · Data updated 2026-11-12
A single IV gene therapy aims to restore the ALDH2 enzyme to lower toxic acetaldehyde and help prevent alcohol-related bone loss in postmenopausal East Asian women with ALDH2 mutations.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Enyx Therapeutics, LLC NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | July 1, 2020 |
| Project end | August 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $1,253,597 |
| NIH project number | 2R42AA028465-02A1 |
| Project ID | NIH-11256090 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are postmenopausal East Asian women who consume alcohol and have a confirmed ALDH2 deficiency (for example the ALDH2*2 allele) and who meet study health and safety criteria.
✕Less likely to help
People without ALDH2 deficiency, men, younger women, or individuals who do not consume alcohol are unlikely to benefit from this therapy.
What this research studies
I would be offered a single intravenous dose of ENX03, an AAV vector carrying the normal human ALDH2 gene, intended to increase ALDH2 enzyme activity in my body. The therapy is meant to reduce acetaldehyde buildup after drinking alcohol, since acetaldehyde and ethanol together can impair early bone-forming cells and lead to bone loss. The program is based on evidence from mouse models of ALDH2 deficiency that develop osteopenia after chronic alcohol exposure, and the work is being done in collaboration with the Crystal laboratory at Weill Cornell. ENYX Therapeutics is developing the approach in New York and plans to move toward human testing if safety and effectiveness are demonstrated preclinically.
Why it matters
Potential benefit: If successful, this could reduce bone loss and lower the risk of hip fractures in alcohol-consuming, ALDH2-deficient postmenopausal East Asian women.
How similar studies have gone: AAV gene therapies have worked for other inherited enzyme disorders, but using ALDH2 gene transfer to prevent alcohol-related osteoporosis is a novel approach so far supported only by animal-model data.
Where this research is happening
-
Enyx Therapeutics, LLC
New York, United States Active
Researchers
- Principal investigator: Crystal, Ronald G — Enyx Therapeutics, LLC
- Study coordinator: Crystal, Ronald G
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.