The startup develops gene therapies utilizing Adeno-Associated Virus (AAV) technology to target ciliopathies, which are diseases caused by cilia dysfunction. Their approach enables early identification and treatment of these conditions by addressing the underlying genetic causes.
Funding
$45M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
ARFounders
Product
Problem
Ciliopathies, a group of rare genetic diseases affecting cilia function, lack effective disease-modifying therapies, leading to a significant disease burden for affected individuals. Patients often endure a prolonged diagnostic journey, and the absence of targeted treatments results in a range of severe, life-threatening conditions, including blindness and obesity.
Solution
Axovia Therapeutics is developing novel AAV-based gene therapies to address the underlying genetic causes of ciliopathies, with a primary focus on Bardet-Biedl Syndrome (BBS). Their lead program, AXV-101, utilizes an AAV9 vector to deliver a functional copy of the BBS1 gene to key tissues, aiming to halt retinal degeneration and reverse obesity. By targeting the genetic root of the disease, Axovia seeks to prevent blindness and correct metabolic dysfunction associated with BBS1 mutations. A subretinal study is planned to prevent further retinal degeneration, and a CNS delivery program will address hyperphagia and obesity.
Target Audience
The primary target audience includes individuals with ciliopathies, particularly Bardet-Biedl Syndrome (BBS) and those with MC4R mutations causing genetic obesity, as well as their families and caregivers.
Features
- AAV9-based gene therapy platform for targeted delivery of functional genes
- Lead program (AXV-101) focused on addressing BBS1 mutations
- Therapeutic approach designed to halt photoreceptor cell death and prevent retinal degeneration
- CNS delivery program (AXV-102) to address hyperphagia and obesity associated with BBS
- Second program (AXV-201) in development for genetic obesity caused by MC4R mutations
- AXV-101 has received U.S. Food and Drug Administration Orphan Drug Designation and Rare Pediatric Disease Designation