Treating GM1 brain disease with a systemically delivered enzyme plus gene therapy
Official title: Treatment of GM1 gangliosidosis CNS manifestations by AAV systemic expression of lectin-enhanced enzyme
Funded at Biostrategies, Lc · NIH-11627834 · Data updated 2026-11-12
A new therapy for GM1 gangliosidosis aims to deliver a corrective enzyme to the brain and body using a one-time AAV-based approach to help children with GM1.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Biostrategies, Lc NIH-funded |
| Lab location | 1 site (State University, United States) |
| Project start | September 15, 2025 |
| Project end | August 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $1,500,000 |
| NIH project number | 5R44NS141514-02 |
| Project ID | NIH-11627834 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children aged 0-11 with GM1 gangliosidosis who have the genetic mutation affecting β-Gal and are eligible for gene-therapy based interventions.
✕Less likely to help
Patients without GM1 gangliosidosis or those not eligible for AAV-based delivery may not benefit.
What this research studies
This project seeks to complete IND-enabling work to move BIOS-GM1 toward clinical testing for GM1 gangliosidosis. It combines a delivery-enhanced enzyme with gene therapy so the CNS and body receive the corrective enzyme, potentially reducing neurologic and motor symptoms and improving survival. Researchers will study how well the system can cross the blood-brain barrier and provide sustained enzyme activity without invasive brain injections. The work is designed to establish safe, scalable delivery and preclinical efficacy to support an early-stage human trial.
Why it matters
Potential benefit: If successful, this therapy could slow or prevent neurological decline and improve life expectancy for children with GM1 gangliosidosis.
How similar studies have gone: Other gene-therapy approaches for GM1 and related lysosomal storage diseases show promise, but this systemic, BBB-crossing strategy is novel.
Where this research is happening
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Biostrategies, Lc
State University, United States Active
Researchers
- Principal investigator: Acosta, Walter — Biostrategies, Lc
- Study coordinator: Acosta, Walter
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.