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NDUFS2 knockout human cell line for Complex I research

Biotech & PharmaLab validatedPatent pending

This is a genetically engineered human cell line in which the NDUFS2 gene has been permanently switched off, preventing the cell from producing a key protein component of mitochondrial Complex I, the main energy-generating machinery in human cells. Because Complex I dysfunction is linked to serious inherited metabolic diseases, researchers need a standardized human cell model where Complex I is reliably broken in a specific way. This cell line provides exactly that: a controlled experimental platform for studying how Complex I assembles and what goes wrong when NDUFS2 is missing. It can also serve as a screening tool to test whether candidate drugs or gene therapies can restore or compensate for lost Complex I activity.

What you could build

A licensed research cell line sold or sublicensed to pharmaceutical and biotech companies running drug-discovery programs targeting mitochondrial Complex I disorders; academic core facilities and CROs focused on rare metabolic disease would also be direct buyers.

Who in Virginia should care

Biotech and rare-disease drug developers in the Northern Virginia/DC corridor, as well as academic medical centers like UVA and VCU with mitochondrial disease research programs, would be natural licensees or collaborators.

Readiness: Lab validated

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
Aloka B. BANDARA, David A. BROWN
Filed
Patent pending — filed February 1, 2022
Status
Application
Publication number
US20220299500A1

Ready to talk?

Virginia Tech Intellectual Properties handles licensing for this technology.

VTIP contact coming shortly

Prosim summaries are generated from public patent text and are not legal advice.