Long-acting GCase therapy for Gaucher disease using saRNA injections
Official title: Extended In Situ Production of Glucocerebrosidase (GCase) for the Treatment of Gaucher Disease
Funded at Keylicon Biosciences INC · NIH-11314731 · Data updated 2026-11-12
A new injectable therapy aims to deliver the Gaucher disease enzyme GCase every three months using a self-amplifying RNA platform to reduce treatment burden.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Keylicon Biosciences INC NIH-funded |
| Lab location | 1 site (Brookline, United States) |
| Project start | September 1, 2026 |
| Project end | August 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $400,000 |
| NIH project number | 1R43DK146746-01 |
| Project ID | NIH-11314731 on NIH RePORTER |
Who could benefit from this research
✓Could help
Individuals diagnosed with Gaucher disease who would benefit from enzyme replacement therapy and are eligible for intramuscular or subcutaneous dosing approaches.
✕Less likely to help
Patients with Gaucher disease who require alternatives to enzyme delivery or have contraindications to injections may not experience benefit.
What this research studies
Keylicon Biosciences is developing a long-acting glucocerebrosidase therapy for Gaucher disease delivered by quarterly injections. The approach uses self-amplifying RNA to produce the therapeutic enzyme in the body while aiming to minimize immune side effects. Researchers are optimizing the saRNA design to increase protein output, lower dosing needs, and improve safety, with a focus on tolerability and real-world clinic use. The project builds on prior work showing extended expression and reduced inflammatory responses in preclinical models and human cells.
Why it matters
Potential benefit: If successful, patients could receive GCase therapy less frequently, reducing infusion burden and improving quality of life.
How similar studies have gone: Previous saRNA approaches have shown prolonged protein expression and reduced immune responses in models, with ongoing optimization expected to enhance clinical potential.
Where this research is happening
-
Keylicon Biosciences INC
Brookline, United States Active
Researchers
- Principal investigator: Mcgee, Joshua — Keylicon Biosciences INC
- Study coordinator: Mcgee, Joshua
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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.