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The company, GEMMABIO, will pursue a number of rare, large indications.  A new spinout, RareTx, will be dedicated to Orphan diseases. Wilson explained that with respect to RareTx, solvency will depend largely on being self-sufficient.

According to Wilson, he usually waits for pharma to commercialize orphan products but in these ultra rare orphan disease cases the company will be entirely self-sufficient.

Wilson launched RareTx in October 2025. The pipeline contained AAV gene therapies approved for lysosomal storage diseases (LDS) such as Krabbe disease and GM1. They are caused by the buildup of undigested substances. Symptoms vary in accordance with areas affected by the disorder.

Basket Trials

A similar approach where there are low patient numbers would be basket trials. In these trials patients may have different tumor types but share similar genetic mutations.

The IGI

The IGI is a research institute that is focused on gene editing while along with the FDA supports treatments that could be studied in basket trials.

Wilson estimates that the shared infrastructure can generate hundreds of millions of dollars in annual revenue. These are still only moderate returns but sufficient to support an enterprise with a mission.