Axovia Therapeutics
Gene therapy company developing disease-transformative medicines for ciliopathies, a group of rare genetic disorders caused by dysfunction of cellular cilia.
Notes
Axovia Therapeutics is a gene therapy company focused on developing transformative treatments for ciliopathies, a class of rare genetic diseases caused by defects in cellular cilia. Cilia are small hair-like structures on cells that play critical roles in various biological processes, and mutations affecting cilia function can cause multi-system disorders affecting the eyes, kidneys, brain, and other organs.
The company is developing gene therapy approaches to address the underlying genetic causes of these devastating diseases, with the potential to provide one-time, curative treatments for patients with limited or no treatment options.
Team
- James McArthur, Ph.D. - CEO
- Jill Milne, Ph.D. - Chief Scientific Officer
Additional Research Findings
- Focus on ciliopathies - diseases caused by cilia dysfunction
- Gene therapy platform for rare genetic diseases
- Targeting multi-system disorders (eyes, kidneys, brain)
- One-time treatment approach for genetic diseases
- Cambridge biotech hub location
- Addressing significant unmet medical need in rare diseases
Sources
Investors
| Name | Location | Type | Stages | Portfolio |
|---|---|---|---|---|
| AlbionVC (Healthcare) | London, UK | biotech-focused | seedseries-a+1 | 17 |