Gene therapy for osteoporosis linked to ALDH2 deficiency in 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-11402253 · Data updated 2026-11-12
A gene therapy approach is being developed to reduce osteoporosis risk in postmenopausal East Asian women who have ALDH2 deficiency and drink alcohol.
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 | 2026 |
| Award (this year) | $791,929 |
| NIH project number | 5R42AA028465-03 |
| Project ID | NIH-11402253 on NIH RePORTER |
Who could benefit from this research
✓Could help
Postmenopausal East Asian women with ALDH2 deficiency who consume alcohol and have osteopenia or osteoporosis risk.
✕Less likely to help
Individuals without ALDH2 deficiency, those not consuming alcohol, or those without osteoporosis risk may see no benefit.
What this research studies
ENYX Therapeutics aims to deliver a one-time IV gene therapy (AAVrh.10hALDH2) to restore normal ALDH2 function and lower fracture risk in alcohol-using postmenopausal East Asian women with ALDH2 deficiency. The project builds on collaborations that showed ethanol-related bone loss in ALDH2-deficient mouse models, and plans translation toward humans. Participants would be part of future clinical development steps to determine safety and potential bone health benefits, guided by careful monitoring of acetaldehyde exposure and bone markers. This grant supports preclinical and early clinical activities over several years to move this targeted therapy toward patients who suffer increased osteoporosis risk due to ALDH2 deficiency and alcohol use.
Why it matters
Potential benefit: If successful, the therapy could reduce osteoporosis risk and fracture events in a high-risk group.
How similar studies have gone: This approach builds on ALDH2 biology and AAV-based gene therapy concepts that have shown promise in preclinical models for metabolic and bone health effects.
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.