Speeding up gene‑editing to target the C9ORF72 form of ALS
Official title: High-throughput optimization of gene editing systems for treating Amyotrophic lateral sclerosis
Funded at Acrobat Genomics, INC. · NIH-11194384 · Data updated 2026-11-12
This project builds faster, more precise gene‑editing tools aimed at removing the harmful C9ORF72 repeat mutation in adults with ALS.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Acrobat Genomics, INC. NIH-funded |
| Lab location | 1 site (Foster City, UNITED STATES) |
| Project start | September 20, 2024 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $271,981 |
| NIH project number | 5R43AG088777-02 |
| Project ID | NIH-11194384 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with ALS who have a confirmed C9ORF72 repeat expansion would be the most relevant candidates for therapies developed from this work.
✕Less likely to help
People with ALS who do not carry the C9ORF72 repeat expansion, younger patients under typical adult age cutoffs, or those with very advanced respiratory failure are unlikely to benefit from this specific approach.
What this research studies
The team will use high‑throughput lab tests to compare many gene‑editing approaches and identify the ones that best remove the toxic C9ORF72 repeat expansion. They will prioritize methods that can edit the disease mutation precisely, work on human‑relevant cells or samples, and reduce unwanted edits. Safety checks and measures to allow multiplexed targeting are part of the plan so therapies could be both effective and specific. Successful candidates would then move toward further preclinical development that could lead to clinical trials.
Why it matters
Potential benefit: If successful, this work could produce a durable gene‑editing approach that permanently reduces or removes the disease‑causing C9ORF72 mutation and slow or stop disease progression for affected patients.
How similar studies have gone: Related approaches like antisense oligonucleotides and early gene‑editing work have shown promise in models, but permanent, targeted editing of C9ORF72 in patients remains largely unproven.
Where this research is happening
-
Acrobat Genomics, INC.
Foster City, United States Active
Researchers
- Principal investigator: Hughes, Nicholas — Acrobat Genomics, INC.
- Study coordinator: Hughes, Nicholas
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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.