Mitochondrial antioxidant therapy for CASK-linked
This technology uses a class of compounds called SkQ molecules — mitochondria-targeted antioxidants — to treat CASK-linked neurodevelopmental disorders, a group of rare genetic conditions caused by mutations in the CASK gene that lead to severe intellectual disability, microcephaly, and other neurological impairments. The core insight is that CASK gene dysfunction disrupts mitochondrial health, generating damaging oxidative stress in neurons, and that SkQ compounds can be directed specifically into mitochondria to neutralize that damage. By administering these compounds to affected patients, the method aims to reduce or slow neurological symptoms at their biochemical root cause. SkQ1 (one known SkQ compound) has existing safety data from other research contexts, which may shorten the path to clinical evaluation.
What you could build
A pharmaceutical drug (oral or injectable SkQ compound formulation) targeting CASK-linked disorders, sold to rare-disease specialty pharma companies or licensed to orphan-drug developers; buyers would be biotech firms focused on rare pediatric neurological conditions seeking orphan drug designation and its associated regulatory incentives.
Who in Virginia should care
Virginia-based rare disease advocacy networks, pediatric neurology programs at academic medical centers like UVA or VCU, or biotech startups pursuing orphan drug pipelines in the mid-Atlantic region.
Readiness: Concept
Concept — described but not yet demonstrated. Lab validated — supported by experimental results in the patent. Prototype likely — the text describes a built, working embodiment.
Readiness is inferred from the patent text, not from a lab visit.
The record
- Inventors
- Konark MUKHERJEE, Sarika SRIVASTVA
- Filed
- Patent pending — filed November 13, 2024
- Status
- Application
- Publication number
- US20250312360A1
Ready to talk?
Virginia Tech Intellectual Properties handles licensing for this technology.
Prosim summaries are generated from public patent text and are not legal advice.