# Development of KCC2 neuromodulators to treat paralysis after spinal cord injury.

> **NIH NIH R43** · AXONIS THERAPEUTICS, INC. · 2020 · $330,619

## Abstract

Summary
Roughly 17,000 people in the United States will suffer a spinal cord injury (SCI) this year. For the vast
majority of them, the injury will leave them incapable of walking or, if they have suffered a cervical SCI,
entirely dependent upon others for assistance in all of their activities of daily living from feeding to
personal care. More importantly, chances of improvement as a result of therapeutic interventions are
bleak since there are no approved therapies that enhance functional recovery. The cost to these
individuals, and to society as a whole, are significant as SCI often occurs in adults in their peak earning
years. Most therapeutic interventions currently being tested are developed for use in the acute period from
hours to days after injury. We are developing a small molecule that has neuromodulatory activity for treating
both acute and chronic SCI patients. Neuromodulation after injury has the potential to restore functional
recovery, as shown by a number of different investigators. After SCI there is an electrochemical imbalance
that prevents motor neurons from eliciting normal muscle actions that are critical for movement of arms and
legs. This imbalance is partly caused by a decrease in the chloride transporter called KCC2. It has been
shown that by restoring normal levels of this transporter in spared spinal cord neurons, paralyzed mice
regain walking ability. This grant application aims to examine new compounds that have the potency and
properties to be translated towards human clinical studies, by testing them in preclinical models.

## Key facts

- **NIH application ID:** 10154250
- **Project number:** 1R43NS120762-01
- **Recipient organization:** AXONIS THERAPEUTICS, INC.
- **Principal Investigator:** Joanna Stanicka
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $330,619
- **Award type:** 1
- **Project period:** 2020-09-30 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10154250

## Citation

> US National Institutes of Health, RePORTER application 10154250, Development of KCC2 neuromodulators to treat paralysis after spinal cord injury. (1R43NS120762-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10154250. Licensed CC0.

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