Autophagon gene therapy to help brain cells clear harmful Alzheimer’s and Parkinson’s proteins
Official title: Autophagon: an Autophagy-Functionalizing Gene Therapy Tool for Neurodegenerative Diseases
Funded at Brigham and Women's Hospital · NIH-11238545 · Data updated 2026-11-12
A gene therapy that helps brain cells send toxic protein clumps to their natural recycling system for people with Alzheimer’s or Parkinson’s.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Brigham and Women's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | March 15, 2022 |
| Project end | December 31, 2026 |
| Funding year | 2026 |
| Award (this year) | $747,248 |
| NIH project number | 5R01AG074899-06 |
| Project ID | NIH-11238545 on NIH RePORTER |
Who could benefit from this research
✓Could help
Future clinical candidates would likely be people diagnosed with Alzheimer’s disease or Parkinson’s disease, especially those in earlier stages or with genetic forms such as APP/PSEN1 or α‑synuclein mutations.
✕Less likely to help
People without evidence of protein‑aggregation disease, those with very advanced neurodegeneration, or anyone now seeking direct treatment will not benefit from this preclinical work.
What this research studies
Researchers are creating a synthetic gene tool called Autophagon that tags and shuttles toxic amyloid‑β and α‑synuclein aggregates into neurons' autophagy (cellular cleanup) pathway. They will test Autophagon in lab-grown human neurons and 3D brain organoids made from patient-derived stem cells carrying Alzheimer’s or Parkinson’s mutations. The team will also deliver the tool into mouse brains using viral vectors to see whether it lowers protein aggregates and protects nerve cells. Successful lab and animal results would support further development toward human testing.
Why it matters
Potential benefit: If successful, this approach could lower toxic protein buildup in the brain and potentially slow or prevent cognitive and motor decline in Alzheimer’s and Parkinson’s patients.
How similar studies have gone: Related strategies that boost autophagy have cleared toxic proteins in cell and animal models, but gene therapies targeting aggregates have not yet been proven safe and effective in people.
Where this research is happening
-
Brigham and Women's Hospital
Boston, United States Active
Researchers
- Principal investigator: Yakoub, Abraam M. — Brigham and Women's Hospital
- Study coordinator: Yakoub, Abraam M.
Related research
Other NIH-funded projects on the same topics.
-
Developing small molecules to treat TDP-43 related brain diseases
A potential new drug candidate (XL20) for diseases like Alzheimer’s and other dementias linked to TDP-43 protein problems is being developed and refined for people who live with these conditions.
-
Tau filament structure in Alzheimer’s and related dementias
Aims to map the detailed 3D shapes of tau protein filaments found in human brain diseases to help develop better diagnostics and treatments for Alzheimer’s-related dementias.
-
Targeting RNA Splicing Abnormalities in ALS/FTD
A research team aims to understand and correct abnormal RNA splicing in ALS and related brain diseases to help patients in the future.
-
Blood-brain barrier–friendly imaging agents for neurodegenerative diseases
A researcher team will test new imaging agents that can cross the brain barrier to help detect Alzheimer’s and related diseases earlier and more accurately.
-
Brain cell–derived EV markers to differentiate Alzheimer’s and related dementias
A Mayo Clinic team is testing brain-cell–specific tiny particles in blood to tell apart Alzheimer’s disease and similar dementias using advanced machine-learning analysis.
-
Fisetin approach to reduce vulnerability to community infections in aging veterans
A patient-friendly look at a study testing whether the natural compound fisetin can help aging Veterans stay healthier when they face common infections.
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.