# Development of AAV-AXN-007 gene therapy to treat glaucoma.

> **NIH NIH R43** · AXONIS THERAPEUTICS, INC. · 2022 · $298,819

## Abstract

Development of AAV-AXN-007 gene therapy to treat Glaucoma
Abstract:
Glaucoma represents the world’s leading cause of irreversible blindness, with its prevalence increasing as the
population ages. Vision loss in glaucoma is caused by a progressive degeneration of retinal ganglion cells
(RGCs), yet there are no approved therapies that can directly prevent RGC loss. Because elevated intraocular
pressure (IOP) is only known modifiable risk factor, current standard of care involves IOP-lowering treatments
via pharmacological and surgical approaches. This chronic, progressive, age-related neurodegenerative
disorder is an urgent, unmet, global and growing problem, with the number of people worldwide suffering with
glaucoma expected to double to ~120 million by 2040, and the annual medical costs of glaucoma in US
projected treble to >$17 billion by 2050. To address this escalating medical and socioeconomic burden, and to
improve lives of glaucoma patients and their families, novel disease-modifying therapies are needed.
 Progressive RGC degeneration in glaucoma is thought to result from an intrinsic sensitivity of RGCs
that over time succumb to the chronic pathological stresses of this complex neurodegenerative disease.
Intervening during this time period in the remaining RGCs of glaucoma patients could prevent their
degeneration. To identify new therapeutic targets, an unbiased in vivo AAV2-CRISPR/Cas9 screen of >2,000
genes (tested one-by-one) for RGC neuroprotection in the mouse optic nerve crush model was conducted.
This forward genetic screen in an animal model of optic neuropathy discovered a neuroprotective hit gene that,
when targeted with AAV2-sgRNA, could prevent RGCs from degeneration following axon damage.
Additionally, this pathway has been reported to be activated in post-mortem retinal tissue from glaucoma
patients and after optic nerve damage. In this SBIR application, our goal is to validate and test a novel
neuroprotective target and AAV2-AXN-007 gene therapeutic approach for its ability to protect RGCs, their
axons and visual function in the widely-used mouse microbead occlusion model of glaucoma.

## Key facts

- **NIH application ID:** 10547231
- **Project number:** 1R43EY034407-01
- **Recipient organization:** AXONIS THERAPEUTICS, INC.
- **Principal Investigator:** Shane Hegarty
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $298,819
- **Award type:** 1
- **Project period:** 2022-09-01 → 2024-02-29

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10547231

## Citation

> US National Institutes of Health, RePORTER application 10547231, Development of AAV-AXN-007 gene therapy to treat glaucoma. (1R43EY034407-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10547231. Licensed CC0.

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