Intrexon will develop DNA-based system for in vivo cell synthesis of PGIS.

Intrexon could end up owning just shy of 30% of Adeona Pharmaceuticals as part of a worldwide collaboration through which Adeona will exploit its partner’s technology to develop a DNA-based treatment for pulmonary arterial hypertension (PAH). The aim is to use Intrexon’s UltraVector® platform and RheoSwitch Therapeutic System® to generate a technology for enabling the controlled, continuous in vivo cellular production of prostaglandin synthase (PGIS), an effector enzyme that regulates the production of prostacyclin, and which is decreased in the lungs of PAH patients.

Under terms of the deal Intrexon will initially receive 9.995% of Adeona’s stock, and then another 9.995% once the first patient has been enrolled in a U.S. Phase II study of a candidate product. In addition Intrexon has the right to purchase up to another 10% of Adeona common stock on the open market.

Intrexon will carry out discovery work and certain aspects of manufacturing, while Adeona will be responsible for preclinical and clinical development, commercialization, and other aspects of manufacturing. The firm will in addition be subject to expense allocations and pay Intrexon 50% of the cumulative net quarterly profits derived from the sale of any products developed through the collaboration.

“Current sales of approved therapies for PAH are an estimated $3 billion per year,” comments James S. Kuo, M.D., Adeona CEO. “We believe that by having the ability to correct what is considered to be a critical pathophysiological defect in PAH, namely the decreased expression of prostaglandin synthase, we may have the opportunity to fundamentally change the course of PAH.”

The firms say continuous central venous catheter infusion of prostacyclin has already been found to increase the survival of primary PAH patients, while animal models of PAH have demonstrated that DNA-based expression of PGIS in vivo increases prostacyclin levels and improves survival.

Synthetic biology firm Intrexon is exploiting its modular DNA control systems to generate Better DNA™ for applications across a range of industries. In October the firm acquired platform human antibodies firm Immunologix. Also in October Intrexon announced the acquisition of GT Life Sciences, and the purchase of assets comprising Cyntellect’s LEAP cell-processing platform, which together form the basis of Intrexon’s new cell-engineering unit. 

Adeona is focused primarily on developing drugs for the treatment of central nervous system diseases. The firm has products in clinical trials for the treatment of multiple sclerosis and multiple sclerosis-related cognitive dysfunction, fibromyalgia, and amyotrophic lateral sclerosis. 

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