# Development of Neuromodulating AAV-KCC2 Gene Therapy to Treat Paralysis, Spasticity and Neuropathic Pain after Spinal Cord Injury

> **NIH NIH R43** · AXONIS THERAPEUTICS, INC. · 2022 · $335,668

## Abstract

Summary
There are no approved therapies for spinal cord injury. There are about 300,000 people living with chronic
spinal cord injury in US. For the vast majority of them, the injury left 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. We are developing a neuromodulating gene therapy for treating both
acute and chronic SCI patients that will be delivered as a single injection to spinal cord.
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. Furthermore,
enhancement of KCC2 reduces pain and spasticity in rodent models. This grant application aims to examine
a new therapy that has the potency and properties to be translated towards human clinical studies, by
testing them in preclinical models.

## Key facts

- **NIH application ID:** 10384710
- **Project number:** 1R43NS124434-01A1
- **Recipient organization:** AXONIS THERAPEUTICS, INC.
- **Principal Investigator:** Shane Hegarty
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $335,668
- **Award type:** 1
- **Project period:** 2022-09-24 → 2024-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10384710

## Citation

> US National Institutes of Health, RePORTER application 10384710, Development of Neuromodulating AAV-KCC2 Gene Therapy to Treat Paralysis, Spasticity and Neuropathic Pain after Spinal Cord Injury (1R43NS124434-01A1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10384710. Licensed CC0.

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