Amid somewhat of a renaissance in drug development for orphan diseases, another gene therapy company announced funding today for a potential treatment for a rare neurodegenerative condition called Friedreich’s ataxia (FRDA).
Agilis Biotherapeutics said it’s raised $8 million and will work with synthetic biology company Intrexon Corp. to develop gene therapies and genetically modified cell therapies for FRDA. Whereas current treatments focus on minimizing symptoms of the disease, the partners expect their drugs to be able to target the underlying disease mechanisms.
FRDA is caused by a genetic defect that results in limited production of frataxin, a protein thought to help assemble clusters of iron and sulfur molecules in cells that are necessary for the function of many other proteins. When cells are deficient of frataxin, they may not function properly, causing damage to the nervous system and problems with movement. Most people with the disease become wheelchair-bound within two decades of diagnosis, and many die early due to weakened heart muscles.
The goal for Agilis and Intrexon is to use Intrexon’s gene switch technology to develop drugs that will repair the defective gene and enable increased production of the frataxin protein. Under the options of the deal, Agilis and Intrexon could add another rare genetic disease to the collaboration.
Ahead of the pair in developing new treatments for the rare disease are ViroPharma, which is in Phase 1 development of a small molecule drug for FA, and Edison Phamaceuticals, which is conducting a Phase 2 study of its drug.
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