# Developing chemoenzymatic strategies, enzymes, and kits for accessible and affordable gangliosides

> **NIH NIH R44** · INTEGRATED MICRO-CHROMATOGRAPHY SYSTEMS, INC. · 2021 · $903,216

## Abstract

Project Summary
Gangliosides, analogous to biological antennae in cells, are involved in critical roles across multiple biological
processes (i.e. pathogen infection, cell-cell communication, inflammation). Neuroprotective and neurorestorative
functions of gangliosides have been explored for their therapeutic application in Parkinson’s disease and other
neurodegenerative diseases. Despite such important role of gangliosides, there is limited access of gangliosides.
Majority of the current commercially available gangliosides are obtained from animal brains, which is likely a
concern for future therapeutic applications. Such limited availability of gangliosides is further beset by the
diversity based on different sugars and fatty acids. The technologies presented here cover an amalgamation of
nearly a decade of research, where complex ganglioside is synthesized in a single reaction vessel followed by
a simple purification technique. This biosynthetic tool opens the availability of critically needed gangliosides in
research and support its therapeutic uses. In this fast track proposal, we will address some major challenges in
transitioning this prototypic one-pot multienzyme (OPME) technology for large scale commercialization. First
challenge is manufacturing of starting materials (glycosyltransferases, supplemental enzymes and
lactosylsphingosine) to reduce the cost to manufacture gangliosides. Additional improvements of existing
glycosyltransferases will further reduce the cost burden to manufacture gangliosides by improving catalytic
efficiencies of the enzymes. Lastly, OPME scaleability will be explored for manufacturing the various
gangliosides. A study will result in several commercial products, ranging from purified enzymes, miniaturized
glycosylation kits and several key gangliosides with high commercial value. GM1 ganglioside has been reported
to delay onset of Parkinson disease, and clinical trials will likely require animal-free gangliosides. A miniaturized
kit will be provided as a tool for researchers to synthesize their own customized gangliosides. The
glycosyltransferases and the OPME kits allow the broad scientific community to access these simple synthetic
tools for other oligosaccharides, glycoconjugates, and glycoproteins. By expanding this OPME and lowering the
cost of these products, additional information on the biological functions of gangliosides will be gained which will
accelerate the development of ganglioside-based diagnostics and therapeutics. A positive feedback loop, where
lower cost will increase purchase volume, which will feed additional studies and will lead to the further growth of
the market for the glycosyltransferases, OPME kits, and highly pure, animal-free gangliosides.

## Key facts

- **NIH application ID:** 10267384
- **Project number:** 4R44GM139441-02
- **Recipient organization:** INTEGRATED MICRO-CHROMATOGRAPHY SYSTEMS, INC.
- **Principal Investigator:** L. Andrew Lee
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $903,216
- **Award type:** 4N
- **Project period:** 2020-08-01 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10267384

## Citation

> US National Institutes of Health, RePORTER application 10267384, Developing chemoenzymatic strategies, enzymes, and kits for accessible and affordable gangliosides (4R44GM139441-02). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10267384. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*